ReferenceCitation: Blom DJ, Harada-Shiba M, Rubba P, Gaudet D, Kastelein JJP, Charng MJ, Pordy R, Donahue S, Ali S, Dong Y, Khilla N, Banerjee P, Baccara-Dinet M, Rosenson RS. Efficacy and Safety of Alirocumab in Adults With Homozygous Familial Hypercholesterolemia: The ODYSSEY HoFH Trial. J Am Coll Cardiol. 2020 Jul 14;76(2):131-142. doi: 10.1016/j.jacc.2020.05.027.

family: Blom

full_name: Dirk J. Blom

given: Dirk J.

family: Harada-Shiba

full_name: Mariko Harada-Shiba

given: Mariko

family: Rubba

full_name: Paolo Rubba

given: Paolo

family: Kastelein

full_name: John J.P. Kastelein

given: John J.P.

family: Charng

full_name: Min-Ji Charng

given: Min-Ji

family: Donahue

full_name: Stephen Donahue

given: Stephen

family: Banerjee

full_name: Poulabi Banerjee

given: Poulabi

family: Baccara-Dinet

full_name: Marie Baccara-Dinet

given: Marie

journal_name: Journal of the American College of Cardiology

location_country:
Italy
Austria
South Africa
Germany
France
Czechia
Greece
Japan
Turkey
United States
Canada
Taiwan
Ukraine

acronym: ODYSSEY HoFH

location_facility:
Regeneron Study Site
Regeneron Research Site

title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia

summary:
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment.

The secondary objectives of the study are:

To evaluate the effect of alirocumab Q2W on other lipid parameters (ie, apolipoprotein [Apo] A-1 and B, non-high-density lipoprotein cholesterol [non-HDL-C], total-cholesterol [TC], proportion of participants with 15%, 30%, and 50% LDL-C reductions, Lp(a), HDL-C, triglycerides [TG]) in participants with HoFH
To evaluate the safety and tolerability of alirocumab SC Q2W in participants with HoFH
To assess the pharmacokinetics of alirocumab SC Q2W in participants with HoFH
To assess the potential development of anti-drug (alirocumab) antibodies


title: A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects With Light Chain (AL) Amyloidosis Who Were Previously Enrolled in Study NEOD001-201 (PRONTO)

summary: The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who have completed Study NEOD001-201.

study_start_date_type: Actual

status: Terminated

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Actual

ipd_sharing: Undecided

lead_sponsor: Prothena Biosciences Ltd.

location_country:
Italy
Austria
Germany
France
Greece
Israel
Australia
Spain
United States
United Kingdom

location_facility: Hospital of the University of Pennsylvania

location_facility: Hospital Universitario Puerta de Hierro - Majadahonda

location_facility: The University of Texas MD Anderson Cancer Center

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Unknown


title: Multi-Center Clinical Evaluation of the ATTUNE® Revision System in Revision Total Knee Arthroplasty

summary: Five-year, Prospective, multi-center, non-randomized, non-controlled study of the ATTUNE® Revision total knee prostheses, utilizing the fixed bearing (FB) and rotating platform (RP) tibial component with the posterior stabilizing (PS) femoral component in revision total knee arthroplasty.

status: Recruiting

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: DePuy Orthopaedics

location_country:
Italy
Austria
United States
Switzerland
Netherlands
Germany
France
Ireland
Australia
Belgium
New Zealand
Canada
United Kingdom

location_facility: Klinik und Poliklinik für Orthopädie und Sportorthopädie am Klinikum rechts der Isar der Technischen Universität Münche

location_facility: Kantonsspital Baselland (Bruderholz, Liestal, Laufen), Department of Orthopaedic Surgery and Traumatology

location_facility: Hampshire Hospitals NHS Foundation Trust

location_facility: The Gold Coast Centre for Bone and Joint Surgery

location_facility: University of Mississippi Medical Center

location_facility: Centre Hospitalier Universitaire de Rennes

location_facility: Krankenhaus der Barmherzigen Schwestern Linz Betriebsgesellschaft m.b.H.

location_facility: The Ohio State University Wexner Medical Center

location_facility: The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust

location_facility: London Health Sciences Centre University Hospital

location_facility: CLINICHE GAVAZZENI SPA aka Humanitas Castelli

location_facility: Asklepios Orthopädische Klinik Lindenlohe

intervention_type: Device

status_simplified: Ongoing


design_allocation: Non-Randomized

lead_sponsor: Rainier Therapeutics

location_country:
Italy
Sweden
Serbia
Netherlands
Germany
Moldova, Republic of
France
Poland
Hungary
Belgium
Russian Federation
Spain
Turkey
Denmark
United States
Korea, Republic of
Ukraine

location_facility: Washington University School of Medicine

type: EudraCT Number

source: euctr

title: A Multi-Center, Open-Label Phase 1b/2 Study of a Novel FGFR3 Inhibitor (B-701) Combined With Pembrolizumab in Subjects With Locally Advanced or Metastatic Urothelial Carcinoma Who Have Progressed Following Platinum-based Chemotherapy

summary: This is a Phase 1b/2 multi-center, open-label study to establish the initial safety and to determine a recommended Phase 2 dose of B-701 in combination with pembrolizumab, and to determine safety, tolerability and efficacy of B-701 (vofatamab) plus pembrolizumab in the treatment of subjects with locally advanced or metastatic UCC, who have progressed following platinum-based chemotherapy and who have not received prior immune checkpoint inhibitor therapy.


title: A Phase 1 Multicenter, Open-label, Dose-escalation and Dose-expansion Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Immunogenicity and Antitumor Activity of MEDI7247 in Patients With Selected Relapsed/Refractory Hematological Malignancies

summary: To assess safety and tolerability, describe the dose-limiting toxicities, determine the maximum tolerated dose (MTD) or the highest protocol-defined dose (maximum administered dose) in the absence of establishing the MTD, and a recommended dose for further evaluation of MEDI7247 in patients with selected hematological malignancies who have relapsed after, or are refractory to prior standard therapy, and for whom there is no standard salvage regimen available.

status: Completed

lead_sponsor: MedImmune LLC

location_country:
United States
Korea, Republic of
France

status_simplified: Completed


status: Active, not recruiting

lead_sponsor: Kite, A Gilead Company

location_country:
United States
France

location_facility: University of Rochester Medical Center (URMC)

location_facility: Georgetown Lombardi Comprehensive Cancer Center

location_facility: University of Miami Hospital and Clinics

location_facility: Centre Hospitalier Régional Universitaire de Lille

location_facility: Hackensack University Medical Center - John Theurer Cancer Center

title: A Phase 2 Multicenter Study of Axicabtagene Ciloleucel in Subjects With Relapsed/Refractory Indolent Non-Hodgkin Lymphoma (iNHL)

summary: This study will enroll approximately 160 adult subjects who have relapsed or refractory (r/r) iNHL to be infused with the study treatment, axicabtagene ciloleucel, to see if their disease responds to this experimental product and if this product is safe. Axicabtagene ciloleucel is made from the subjects own white blood cells which are genetically modified and grown to fight cancer. An objective response rate of 70% is targeted.


title: A Phase II Randomized, Open-label, Multi-center Study of the Safety and Efficacy of IMCgp100 Compared With Investigator Choice in HLA-A*0201 Positive Patients With Previously Untreated Advanced Uveal Melanoma

summary: To evaluate the overall survival of HLA-A*0201 positive adult patients with previously untreated advanced UM receiving IMCgp100 compared to Investigator's Choice of dacarbazine, ipilimumab, or pembrolizumab.

design_allocation: Randomized

ReferenceType: derived

ReferenceCitation: Nathan P, Hassel JC, Rutkowski P, Baurain JF, Butler MO, Schlaak M, Sullivan RJ, Ochsenreither S, Dummer R, Kirkwood JM, Joshua AM, Sacco JJ, Shoushtari AN, Orloff M, Piulats JM, Milhem M, Salama AKS, Curti B, Demidov L, Gastaud L, Mauch C, Yushak M, Carvajal RD, Hamid O, Abdullah SE, Holland C, Goodall H, Piperno-Neumann S; IMCgp100-202 Investigators. Overall Survival Benefit with Tebentafusp in Metastatic Uveal Melanoma. N Engl J Med. 2021 Sep 23;385(13):1196-1206. doi: 10.1056/NEJMoa2103485.

name: From Mount Vernon Cancer Centre, Northwood (P.N.), the Clatterbridge Cancer Centre NHS Foundation Trust, Wirral (J.J.S.), the University of Liverpool, Liverpool (J.J.S.), and Immunocore, Abingdon (S.E.A., C.H., H.G.) — all in the United Kingdom; the Department of Dermatology and the National Center for Tumor Diseases, University Hospital Heidelberg, Heidelberg (J.C.H.), the Department of Dermatology and Allergy, University Hospital, Ludwig Maximilian University of Munich, Munich (M.S.), the Department of...

family: Nathan

full_name: Paul Nathan

given: Paul

sequence: first

family: Hassel

full_name: Jessica C. Hassel

given: Jessica C.

sequence: additional

family: Rutkowski

full_name: Piotr Rutkowski

given: Piotr

family: Baurain

full_name: Jean-Francois Baurain

given: Jean-Francois

family: Butler

full_name: Marcus O. Butler

given: Marcus O.

family: Schlaak

full_name: Max Schlaak

given: Max

family: Sullivan

full_name: Ryan J. Sullivan

given: Ryan J.

family: Ochsenreither

full_name: Sebastian Ochsenreither

given: Sebastian

family: Dummer

full_name: Reinhard Dummer

given: Reinhard

family: Kirkwood

full_name: John M. Kirkwood

given: John M.

family: Joshua

full_name: Anthony M. Joshua

given: Anthony M.

family: Sacco

full_name: Joseph J. Sacco

given: Joseph J.

family: Shoushtari

full_name: Alexander N. Shoushtari

given: Alexander N.

family: Orloff

full_name: Marlana Orloff

given: Marlana

family: Piulats

full_name: Josep M. Piulats

given: Josep M.

family: Milhem

full_name: Mohammed Milhem

given: Mohammed

family: Salama

full_name: April K.S. Salama

given: April K.S.

family: Curti

full_name: Brendan Curti

given: Brendan

family: Demidov

full_name: Lev Demidov

given: Lev

family: Gastaud

full_name: Lauris Gastaud

given: Lauris

family: Mauch

full_name: Cornelia Mauch

given: Cornelia

family: Yushak

full_name: Melinda Yushak

given: Melinda

family: Carvajal

full_name: Richard D. Carvajal

given: Richard D.

repository_institution: University of Zurich - Zurich Open Repository and Archive

location_country:
Italy
United States
Switzerland
Netherlands
Germany
France
Poland
Belgium
Australia
Russian Federation
Spain
Canada
United Kingdom
Ukraine

location_facility: Universitaetsklinikum Koeln Dermatologie und Venerologie

location_facility: Federal State Budgetary Institution N.N. Blokhin National Medical Research Center of Oncology

location_facility: University of Miami - Sylvester Comprehensive Cancer Center

location_facility: Federal State Budget Institution National Medical Research Center of Oncology

location_facility: Hospital Clínico Universitario de Santiago de Compostela

location_facility: Istituto Nazionale Tumori - IRCCS Fondazione "G. Pascale" - UOC Melanoma, Immunoterapia Oncologica e Terapie Innovative

location_facility: Hospital Universitario General de Valencia

location_facility: Nationales Centrum für Tumorerkrankungen

location_facility: The Angeles Clinic and Research Institute

location_facility: Winship Cancer Institute of Emory University

location_facility: Central Adelaide Local Health Network, Royal Adelaide Hospital Cancer Center

location_facility: Institut Roi Albert II Cliniques Universitaires St-Luc

location_facility: Klinik und Poliklinik für Dermatologie und Allergologie

location_facility: Byers Eye Institute, Stanford University

location_facility: Centrum Onkologii - Instytut im. Marii Sklodowskiej-Curie

location_facility: Institut Catala d'Oncologia (ICO) - L'Hospitalet


title: A Multicenter Phase 1/2, Open-Label Study of DCC-3014 to Assess the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics in Patients With Advanced Tumors and Tenosynovial Giant Cell Tumor

summary: This is a multicenter, open-label Phase 1/2 study of DCC-3014 in patients with malignant solid tumors and tenosynovial giant cell tumor (TGCT). There will be 2 distinct parts in this study: Dose Escalation (Phase 1) and Expansion (Phase 2). Phase 1 will enroll both malignant solid tumor and TGCT patients. Phase 2 will comprise two cohorts (Cohort A and Cohort B) and will only enroll TGCT patients.

lead_sponsor: Deciphera Pharmaceuticals LLC

location_country:
Italy
United States
Netherlands
France
Poland
Australia
Spain
Canada
United Kingdom

location_facility: Fondazione IRCCS Istituto Nazionale Dei Tumori

location_facility: Gustave Roussy Cancer Campus Grand Paris

location_facility: Hospital Universitario Virgen del Rocío, Sevilla

location_facility: M. Sklodowska-Curie Memorial Cancer Center


title: Double Blind, Randomised, Vehicle Controlled, Phase III, Efficacy and Safety Study With 24-month Open-label Follow up of Oleogel-S10 in Patients With Inherited Epidermolysis Bullosa

acronym: EASE

summary:
This is a Phase III, Efficacy and Safety Study of Oleogel-S10 in Participants with Inherited Epidermolysis Bullosa (EB).

EB is a rare group of genetic skin fragility disorders characterised by blistering of the skin in response to minor injury. In most cases, onset of EB is at birth or shortly after. All participants affected by any type of EB share the main characteristic of repeatedly developing painful wounds that take days to months to heal. Current treatment of EB is primarily preventative and supportive including protection from mechanical forces by avoiding rubbing, early treatment of wounds to prevent infections, and protection of the wound with adequate non-adhesive dressings to enable healing.

Oleogel-S10 was authorised in 2016 in the European Union for treatment of partial thickness wounds in adults under the brand name Episalvan®. The active pharmaceutical ingredient in Oleogel-S10 is a refined birch bark extract, quantified to 72 to 88% betulin.

This clinical study of Oleogel-S10 in patients with inherited EB has been initiated to investigate whether Oleogel-S10 is effective for treatment of EB wounds and safe in the long term use.

Oleogel-S10 will be compared to a vehicle gel placebo. The placebo is an identical looking sunflower oil gel that does not contain any active substance. The participant will receive either Oleogel-S10 or vehicle gel for a double-blind study phase of 90 days. The probability that the participant will receive Oleogel-S10 is 50%, which means that they have a 1 in 2 chance of receiving Oleogel-S10. However, in the follow-up phase of the study all participants will be treated with Oleogel S10 for a period of 24 months.

This clinical study will be performed in several countries; in total, about 250 participants are expected to participate.

lead_sponsor: Amryt Research Limited

location_country:
Greece
Romania
Australia
Russian Federation
Denmark
Ukraine
France
Argentina
Colombia
Chile
Hong Kong
Switzerland
Singapore
Hungary
Ireland
Germany
United States
United Kingdom
Italy
Austria
Brazil
Serbia
Czechia
Israel
Georgia
Spain

location_facility: Consutorios Medicos (Instituto de Neumonologia y Dermatologia)

location_facility: University Hospital Brno, Children´s Hospital

location_facility: Hospital of Skin and Veneral Diseases "A. Syggros"

location_facility: Istituto Dermopatico dell'Immacolata IDI-IRCCS

location_facility: Kandang Kerbau (KK) Women's and Children's Hospital

location_facility: Prince of Wales Hospital, The Chinese University of Hong Kong

location_facility: Kinder- und Jugendkrankenhaus AUF DER BULT

location_facility: University of Belgrade, School of Medicine

location_facility: National Children Specialized Hospital "Ohmatdyt" of Ministry of Health of Ukraine

location_facility: Murdoch Childrens Research Institute Royal Children's Hospital

location_facility: S/R National Center of Dermatology and Venerology

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Birmingham Children's Hospital NHS Trust

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: Centro de investigaciones Metabolicas, CINME

location_facility: State Scientific Center for Dermatovenerology and Cosmetology

location_facility: Hospital Universitari de la Vall d'Hebron

location_facility: Semmelweis University, Faculty of Medicine


title: Phase Ib, Multicenter, Open Label Study of PDR001 in Combination With Platinum Doublet Chemotherapy and Other Immunooncology Agents in PD-L1 Unselected, Metastatic NSCLS Patients (ElevatION:NSCLC-101 Trial)

summary: The primary purpose of this study is to establish the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) of PDR001 when administered in combination with platinum-doublet chemotherapy and other immunooncology agent(s) in treatment naive patients with PD-L1 unselected, advanced NSCLC, and to estimate the preliminary anti-tumor activity in this patient population.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
United States
Hong Kong
Netherlands
Germany
France
Czechia
Singapore
Belgium
Spain
Canada
Korea, Republic of

location_facility: UCLA Santa Monica Hematology / Oncology SC-2

location_facility: Washington University School of Medicine SC


title: A Post-approval Study of the Medtronic Resolute Onyx™ Zotarolimus-Eluting Coronary Stent System

acronym: ONYX PAS

summary: To observe the continued performance of the Medtronic Resolute Onyx™ Zotarolimus-Eluting Coronary Stent System in a real-world more-comer population.

study_type: Observational

time_perspective: Prospective

lead_sponsor: Medtronic Vascular

location_country:
Slovakia
United States
Belgium
France

location_facility: Centre Hospitalier Universitaire Besancon - Hôpital Jean Minjoz

location_facility: University of Michigan Health System - University Hospital

location_facility: Columbia University Medical Center/NewYork Presbyterian Hospital

location_facility: University Hospitals Cleveland Medical Center

location_facility: Stredoslovensky Ustav Srdcovych a Cievnych Chorob a.s

location_facility: Ziekenhuis Oost Limburg - Campus Sint-Jan


title: Evaluation of Ablation Index and VISITAG™ Use for Pulmonary Vein Isolation (PVI) in Patients With Paroxysmal Atrial Fibrillation (PAF)

acronym: VISTAX

summary: The purpose of this study is to assess the safety, acute and long term effectiveness, during standard RF ablation procedures while using Ablation Index and VISITAG™ software in combination with a Thermocool SmartTouch® (ST) or SmartTouch Surroundflow® (STSF) catheter. Furthermore, the role of Ablation Index and VISITAG™ workflow in creating contiguous ablation lines is assessed. The study is a prospective, non-randomized, post-market clinical evaluation. Up to 330 patients will be included in this study. All patients who qualify based on the study specific requirements will be invited to participate. The total duration of the study is estimated to be about 24 months (12 months enrollment period and 12 months of follow up). The clinical investigation population include subjects undergoing RF ablation for treatment of drug resistant symptomatic paroxysmal AF. Prior to enrollment in the clinical investigation, all subjects must meet the inclusion/exclusion criteria and are suitable candidates for enrollment in a clinical investigation in the opinion of the investigator. Subjects must have failed at least one antiarrhythmic drug (AAD) (Type I or III, including β-blocker) as evidenced by recurrent or are intolerant of the AAD.

ReferenceCitation: Gupta D, Vijgen J, Potter T, Scherr D, Van Herendael H, Knecht S, Kobza R, Berte B, Sandgaard N, Albenque JP, Széplaki G, Stevenhagen Y, Taghji P, Wright M, Duytschaever M. Quality of life and healthcare utilisation improvements after atrial fibrillation ablation. Heart. 2021 Aug;107(16):1296-1302. doi: 10.1136/heartjnl-2020-318676. Epub 2021 May 5.

family: Gupta

full_name: Dhiraj Gupta

given: Dhiraj

family: Vijgen

full_name: Johan Vijgen

given: Johan

family: Potter

full_name: Tom De Potter

given: Tom De

family: Scherr

full_name: Daniel Scherr

given: Daniel

family: Van Herendael

full_name: Hugo Van Herendael

given: Hugo

family: Knecht

full_name: Sebastien Knecht

given: Sebastien

family: Kobza

full_name: Richard Kobza

given: Richard

family: Berte

full_name: Benjamin Berte

given: Benjamin

family: Sandgaard

full_name: Niels Sandgaard

given: Niels

family: Albenque

full_name: Jean-Paul Albenque

given: Jean-Paul

family: Széplaki

full_name: Gábor Széplaki

given: Gábor

family: Stevenhagen

full_name: Yorick Stevenhagen

given: Yorick

family: Taghji

full_name: Philippe Taghji

given: Philippe

family: Wright

full_name: Matt Wright

given: Matt

family: Duytschaever

full_name: Mattias Duytschaever

given: Mattias

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Heart

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via page says license)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

evidence: oa repository (via pmcid lookup)

host_type: repository

license_normalized: no license

repository_normalized: PubMed Central

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

ReferenceCitation: Duytschaever M, Vijgen J, De Potter T, Scherr D, Van Herendael H, Knecht S, Kobza R, Berte B, Sandgaard N, Albenque JP, Szeplaki G, Stevenhagen YJ, Taghji P, Wright M, Macours N, Gupta D. Standardized pulmonary vein isolation workflow to enclose veins with contiguous lesions: the multicentre VISTAX trial. Europace. 2020 Nov 1;22(11):1645-1652. doi: 10.1093/europace/euaa157.

name: Department of Cardiology, AZ Sint-Jan Hospital, Bruges, Belgium

name: Division of Electrophysiology, Hasselt Heart Center, Jessa Hospital, Hasselt, Belgium

name: Department of Cardiology, OLV Hospital, Aalst, Belgium

family: De Potter

name: Division of Cardiology, Medical University Graz, Austria

name: Department of Cardiology, Ziekenhuis Oost – Limburg, Genk, Belgium

name: Department of Cardiology, Heart Center, Luzerner Kantonsspital, Luzern, Switzerland

name: Department of Cardiology, Odense University Hospital, Odense, Denmark

name: Department of Cardiology, Clinique Pasteur, Toulouse, France

name: Department of Cardiac Electrophysiology, Mater Private Hospital, Dublin, Ireland

name: Department of Cardiology, Thoraxcentrum, Medisch Spectrum Twente, Enschede, The Netherlands

name: Department of Cardiology, Private Hospital Clairval Marseille, Marseille, France

name: Department of Cardiology, St. Thomas' Hospital London, London, UK

name: Department of Clinical Research, Biosense Webster, Inc., Diegem, Belgium

name: Department of Cardiology, Liverpool Heart and Chest Hospital, Liverpool, UK

location_country:
Italy
Austria
Switzerland
Netherlands
France
Ireland
Belgium
Denmark
United Kingdom

location_facility:
Mater Private Heart & Vascular Centre
St. Thomas' Hospital
Aarhus Universitetshospital Skejby
Ospedaliera Universitaria Pisana
Clinique Clairval Marseille
OLV Aalst
Odense University Hospital
Clinique Pasteur
Policlinico Caisilino Roma
AZ Sint-Jan
Medisch Spectrum Twente
Liverpool Heart and Chest Hospital
LKH Univ. Klinikum Graz
Herzzentrum, Luzerner Kantonsspital
ZOL Genk
Jessa Ziekenhuis
Gentofte Hospital


ReferenceCitation: Zeng R, Liu F, Fang C, Yang J, Luo L, Yue P, Gao B, Dong Y, Xiang Y. PIV and PILE Score at Baseline Predict Clinical Outcome of Anti-PD-1/PD-L1 Inhibitor Combined With Chemotherapy in Extensive-Stage Small Cell Lung Cancer Patients. Front Immunol. 2021 Oct 29;12:724443. doi: 10.3389/fimmu.2021.724443. eCollection 2021.

family: Zeng

full_name: Ran Zeng

given: Ran

family: Liu

full_name: Fang Liu

given: Fang

family: Fang

full_name: Chen Fang

given: Chen

family: Yang

full_name: Jin Yang

given: Jin

family: Luo

full_name: Lifeng Luo

given: Lifeng

family: Yue

full_name: Ping Yue

given: Ping

family: Gao

full_name: Beili Gao

given: Beili

family: Dong

full_name: Yuchao Dong

given: Yuchao

family: Xiang

full_name: Yi Xiang

given: Yi

currency_apc_doaj: USD

journal_name: Frontiers in Immunology

oa_host_type: publisher

evidence: oa journal (via doaj)

publisher: Frontiers Media SA

publisher_dissemination: Frontiers

publisher_group: Frontiers

publisher_normalized: Frontiers

ReferenceCitation: Hussein M, Maglakelidze M, Richards DA, Sabatini M, Gersten TA, Lerro K, Sinielnikov I, Spira A, Pritchett Y, Antal JM, Malik R, Beck JT. Myeloprotective Effects of Trilaciclib Among Patients with Small Cell Lung Cancer at Increased Risk of Chemotherapy-Induced Myelosuppression: Pooled Results from Three Phase 2, Randomized, Double-Blind, Placebo-Controlled Studies. Cancer Manag Res. 2021 Aug 9;13:6207-6218. doi: 10.2147/CMAR.S313045. eCollection 2021.

family: Hussein

full_name: Maen Hussein

given: Maen

family: Maglakelidze

full_name: Marina Maglakelidze

given: Marina

family: Richards

full_name: Donald A Richards

given: Donald A

family: Sabatini

full_name: Marielle Sabatini

given: Marielle

family: Gersten

full_name: Todd A Gersten

given: Todd A

family: Lerro

full_name: Keith Lerro

given: Keith

family: Sinielnikov

full_name: Ivan Sinielnikov

given: Ivan

family: Spira

full_name: Alexander Spira

given: Alexander

family: Pritchett

full_name: Yili Pritchett

given: Yili

family: Antal

full_name: Joyce M Antal

given: Joyce M

family: Malik

full_name: Rajesh Malik

given: Rajesh

family: Beck

full_name: J Thaddeus Beck

ReferenceCitation: Ferrarotto R, Anderson I, Medgyasszay B, García-Campelo MR, Edenfield W, Feinstein TM, Johnson JM, Kalmadi S, Lammers PE, Sanchez-Hernandez A, Pritchett Y, Morris SR, Malik RK, Csőszi T. Trilaciclib prior to chemotherapy reduces the usage of supportive care interventions for chemotherapy-induced myelosuppression in patients with small cell lung cancer: Pooled analysis of three randomized phase 2 trials. Cancer Med. 2021 Sep;10(17):5748-5756. doi: 10.1002/cam4.4089. Epub 2021 Aug 18.

name: University of Texas MD Anderson Cancer Center Houston TX USA

name: St Joseph Heritage Healthcare Santa Rosa CA USA

name: Veszprém County Lung Medicine Institute Veszprém Hungary

name: University Hospital A Coruña A Coruña Spain

name: Prisma Health Cancer Institute Greenville SC USA

name: Piedmont Cancer Institute Atlanta GA USA

name: Thomas Jefferson University Hospital Philadelphia PA USA

name: Ironwood Cancer & Research Center Chandler AZ USA

name: Hospital Provincial de Castellon Castelló Spain

name: G1 Therapeutics, Inc. Research Triangle Park NC USA

title: Trilaciclib prior to chemotherapy reduces the usage of supportive care interventions for chemotherapy‐induced myelosuppression in patients with small cell lung cancer: Pooled analysis of three randomized phase 2 trials

location_country:
France
Bulgaria
Spain
Estonia
United States
Latvia
Ukraine

location_facility: Hospital Clinic de Barcelona- Servicio de Oncología Médica

location_facility: The Alvin J. Siteman Cancer Center - Center for Advanced Med

location_facility: Oklahoma University - Peggy and Charles Stephenson Cancer Center

location_facility: Multiprofile Hospital for Active Treatment "Serdika", Sofia

location_facility: Redwood Regional Medical Group (RRMG) - Fountain Grove

location_facility: St. Louis Cancer Care, LLP, North County

location_facility: East Tallinn Central Hospital Ltd., Clinic of Internal Medicine, Center for Oncology

location_facility: UCLA Medical Center - Santa Monica Hematology And Oncology

location_facility: Daugavpils Regional Hospital, Department of Oncology

location_facility: Lviv State Regional Treatment and Diagnostics Oncology Center

location_facility: Pauls Stradiņš Clinical University Hospital, Oncology Clinic

location_facility: Chernivtsi Regional Clinical Oncology Center

location_facility: H. Donostia, Hospital Donostia- Servicio de Oncología

location_facility: Dnipropetrovsk City Multispecialty Clinical Hospital #4

location_facility: Northside Hospital - Georgia Cancer Specialists

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Hospital Universitario Ntra. Sra. de Valme

location_facility: Trinity Health - Trinity CancerCare Center

location_facility: H.U. Quirón Dexeus, Hospital Universitario

title: Phase 2 Study of Carboplatin, Etoposide, and Atezolizumab With or Without Trilaciclib in Patients With Untreated Extensive Stage Small Cell Lung Cancer

summary:
This is a study to investigate the potential clinical benefit of trilaciclib (G1T28) in preserving the bone marrow and the immune system, and enhancing antitumor efficacy when administered with carboplatin, etoposide, and atezolizumab (E/P/A) therapy in first line treatment for patients with newly diagnosed extensive-stage SCLC.

The study is a randomized, double-blinded, placebo-controlled design. The study will include 3 study phases: Screening Phase,Treatment Phase (induction part + maintenance part), and Survival Follow-up Phase. The Treatment Phase begins on the day of first dose with study treatment and completes at the Post-Treatment Visit. Approximately, 100 patients will be enrolled in the study.


title: A Multi-center, Non-randomized Study to Evaluate the Safety and Effectiveness of the Abre Venous Self-expanding Stent System in Patients With Symptomatic Iliofemoral Venous Outflow Obstruction

summary: Evaluate the safety and effectiveness of the Abre venous self-expanding stent system for treatment of symptomatic iliofemoral venous outflow obstruction in patients with venous occlusive disease.

lead_sponsor: Medtronic Endovascular

location_country:
Italy
United States
France
Ireland
Germany
United Kingdom

location_facility: Guy's & St Thomas' NHS Foundation Trust - St Thomas' Hospital

location_facility: Galway University Hospitals - University Hospital Galway

location_facility: Assistance Publique - Hôpitaux de Marseille - Hôpital Nord

location_facility: University College London Hospitals NHS Foundation Trust - University College London Hospitals


title: Persona Partial Knee Clinical Outcomes Study

summary: The primary objective of this study is to obtain implant survivorship and clinical outcomes data for the commercially available Persona Partial Knee System.

lead_sponsor: Zimmer Biomet

location_country:
Italy
Austria
Sweden
Switzerland
Germany
France
Belgium
Spain
United States
United Kingdom

location_facility: Henry County Orthopedics and Sports Medicine

location_facility: Avon Orthopaedic Centre, Southmead Hospital

location_facility: Harrogate and District NHS Foundation Trust


ReferenceCitation: Bogan RK, Thorpy MJ, Dauvilliers Y, Partinen M, Del Rio Villegas R, Foldvary-Schaefer N, Skowronski R, Tang L, Skobieranda F, Šonka K. Efficacy and safety of calcium, magnesium, potassium, and sodium oxybates (lower-sodium oxybate [LXB]; JZP-258) in a placebo-controlled, double-blind, randomized withdrawal study in adults with narcolepsy with cataplexy. Sleep. 2021 Mar 12;44(3). pii: zsaa206. doi: 10.1093/sleep/zsaa206. Erratum in: Sleep. 2021 Jul 9;44(7):. Sleep. 2021 Nov 12;44(11):.

name: School of Medicine, University of South Carolina, Columbia, SC

family: Bogan

full_name: Richard K Bogan

given: Richard K

name: Department of Neurology, Albert Einstein College of Medicine, Bronx, NY

family: Thorpy

full_name: Michael J Thorpy

given: Michael J

name: Sleep and Wake Disorders Centre, Department of Neurology, Gui de Chauliac Hospital, Montpellier, France

name: Department of Neurology, University of Montpellier, INSERM U1061, Montpellier, France

family: Dauvilliers

full_name: Yves Dauvilliers

given: Yves

name: Helsinki Sleep Clinic, Vitalmed Research Center, Helsinki, Finland

family: Partinen

full_name: Markku Partinen

given: Markku

name: Neurophysiology and Sleep Disorders Unit, Hospital Vithas Nuestra Señora de America, Madrid, Spain

family: Del Rio Villegas

full_name: Rafael Del Rio Villegas

given: Rafael

name: Sleep Disorders Center, Cleveland Clinic, Cleveland, OH

family: Foldvary-Schaefer

full_name: Nancy Foldvary-Schaefer

given: Nancy

name: Global Clinical Development, Jazz Pharmaceuticals, Inc., Palo Alto, CA

family: Skowronski

full_name: Roman Skowronski

given: Roman

name: Biostatistics, Jazz Pharmaceuticals, Inc., Palo Alto, CA

family: Tang

full_name: Lihua Tang

given: Lihua

family: Skobieranda

full_name: Franck Skobieranda

given: Franck

name: Department of Neurology, First Faculty of Medicine, Charles University and General University Hospital, Prague, Czech Republic

family: Šonka

full_name: Karel Šonka

given: Karel

journal_name: Sleep

evidence: open (via crossref license)

license: cc-by-nc

license_normalized: cc-by-nc

publisher: Oxford University Press (OUP)

publisher_dissemination: Oxford University Press

publisher_group: Oxford University Press

publisher_normalized: Oxford University Press

intervention_type: Other

lead_sponsor: Jazz Pharmaceuticals

location_country:
Finland
France
Czechia
Belgium
Spain
United States

acronym: JZP-258 Efficacy Study

location_facility: Hospital Clinic i Provincial de Barcelona

title: A Double-Blind, Placebo-Controlled, Randomized-Withdrawal, Multicenter Study of the Efficacy and Safety of JZP-258 in Subjects With Narcolepsy With Cataplexy

summary: This is a double-blind, placebo-controlled, randomized-withdrawal, multicenter study of the efficacy and safety of JZP-258.


type: U.S. NIH Grant/Contract

title: Craniosynostosis Network

summary: Craniosynostosis (CS) is a common malformation occurring in ~4 per 10,000 live births in which the sutures between skull bones close too early, causing long-term problems with brain and skull growth. Infants with CS typically require extensive surgical treatment and may experience many perioperative complications, including hemorrhage and re-synostosis. Even with successful surgery, children can experience developmental and learning disabilities or vision problems. Most often, CS appears as isolated nonsyndromic CS (NSC). Of the several subtypes of CS, unilateral or bilateral fusion of the coronal suture is the second most common form of CS accounting for 20-30% of all NSC cases. The etiology of coronal NSC (cNSC) is not well understood, although the published literature suggests that it is a multifactorial condition. About 5-14% of coronal craniosynostosis patients have a positive family history, with a specific genetic etiology identified in >25% of cNSC cases, suggesting a strong genetic component in the pathogenesis of this birth defect. The causes for cNSC and its phenotypic heterogeneity remain largely unknown. An international team of investigators will generate large genomic and gene expression datasets on samples from patients with cNSC. State-of-the-art imaging, genetic, and developmental and systems biology approaches will be used to quantitatively model novel pathways and networks involved in the development of cNSC. Novel variant-, gene- and network-level analyses will be performed on the genomic data obtained from cNSC cases, their relatives, and controls to identify novel variants and genetic regions associated with cNCS. Quantitative, analytical, and functional validations of these predictions will provide insights into the etiology and possible therapeutic targets for CS and potentially other bone-related disorders.

time_perspective: Other

ReferenceType: background

ReferenceCitation: Flaherty K, Singh N, Richtsmeier JT. Understanding craniosynostosis as a growth disorder. Wiley Interdiscip Rev Dev Biol. 2016 Jul;5(4):429-59. doi: 10.1002/wdev.227. Epub 2016 Mar 22. Review.

ReferenceCitation: Ye X, Guilmatre A, Reva B, Peter I, Heuzé Y, Richtsmeier JT, Fox DJ, Goedken RJ, Jabs EW, Romitti PA. Mutation Screening of Candidate Genes in Patients with Nonsyndromic Sagittal Craniosynostosis. Plast Reconstr Surg. 2016 Mar;137(3):952-961. doi: 10.1097/01.prs.0000479978.75545.ee.

ReferenceCitation: Heuzé Y, Holmes G, Peter I, Richtsmeier JT, Jabs EW. Closing the Gap: Genetic and Genomic Continuum from Syndromic to Nonsyndromic Craniosynostoses. Curr Genet Med Rep. 2014 Sep 1;2(3):135-145.

ReferenceCitation: Heuzé Y, Singh N, Basilico C, Jabs EW, Holmes G, Richtsmeier JT. Morphological comparison of the craniofacial phenotypes of mouse models expressing the Apert FGFR2 S252W mutation in neural crest- or mesoderm-derived tissues. Bone. 2014 Jun;63:101-9. doi: 10.1016/j.bone.2014.03.003. Epub 2014 Mar 13.

ReferenceCitation: Heuzé Y, Martínez-Abadías N, Stella JM, Arnaud E, Collet C, García Fructuoso G, Alamar M, Lo LJ, Boyadjiev SA, Di Rocco F, Richtsmeier JT. Quantification of facial skeletal shape variation in fibroblast growth factor receptor-related craniosynostosis syndromes. Birth Defects Res A Clin Mol Teratol. 2014 Apr;100(4):250-9. doi: 10.1002/bdra.23228. Epub 2014 Feb 27.

ReferenceCitation: Di Rocco F, Biosse Duplan M, Heuzé Y, Kaci N, Komla-Ebri D, Munnich A, Mugniery E, Benoist-Lasselin C, Legeai-Mallet L. FGFR3 mutation causes abnormal membranous ossification in achondroplasia. Hum Mol Genet. 2014 Jun 1;23(11):2914-25. doi: 10.1093/hmg/ddu004. Epub 2014 Jan 12.

ReferenceCitation: Justice CM, Yagnik G, Kim Y, Peter I, Jabs EW, Erazo M, Ye X, Ainehsazan E, Shi L, Cunningham ML, Kimonis V, Roscioli T, Wall SA, Wilkie AO, Stoler J, Richtsmeier JT, Heuzé Y, Sanchez-Lara PA, Buckley MF, Druschel CM, Mills JL, Caggana M, Romitti PA, Kay DM, Senders C, Taub PJ, Klein OD, Boggan J, Zwienenberg-Lee M, Naydenov C, Kim J, Wilson AF, Boyadjiev SA. A genome-wide association study identifies susceptibility loci for nonsyndromic sagittal craniosynostosis near BMP2 and within BBS9. Nat Genet. 2012 Dec;44(12):1360-4. doi: 10.1038/ng.2463. Epub 2012 Nov 18.

ReferenceCitation: Heuzé Y, Martínez-Abadías N, Stella JM, Senders CW, Boyadjiev SA, Lo LJ, Richtsmeier JT. Unilateral and bilateral expression of a quantitative trait: asymmetry and symmetry in coronal craniosynostosis. J Exp Zool B Mol Dev Evol. 2012 Mar;318(2):109-22. doi: 10.1002/jezb.21449.

ReferenceCitation: Martínez-Abadías N, Heuzé Y, Wang Y, Jabs EW, Aldridge K, Richtsmeier JT. FGF/FGFR signaling coordinates skull development by modulating magnitude of morphological integration: evidence from Apert syndrome mouse models. PLoS One. 2011;6(10):e26425. doi: 10.1371/journal.pone.0026425. Epub 2011 Oct 28.

ReferenceCitation: Heuzé Y, Boyadjiev SA, Marsh JL, Kane AA, Cherkez E, Boggan JE, Richtsmeier JT. New insights into the relationship between suture closure and craniofacial dysmorphology in sagittal nonsyndromic craniosynostosis. J Anat. 2010 Aug;217(2):85-96. doi: 10.1111/j.1469-7580.2010.01258.x. Epub 2010 Jun 22.

ReferenceCitation: Wang Y, Sun M, Uhlhorn VL, Zhou X, Peter I, Martinez-Abadias N, Hill CA, Percival CJ, Richtsmeier JT, Huso DL, Jabs EW. Activation of p38 MAPK pathway in the skull abnormalities of Apert syndrome Fgfr2(+P253R) mice. BMC Dev Biol. 2010 Feb 22;10:22. doi: 10.1186/1471-213X-10-22.

ReferenceCitation: Musy M, Flaherty K, Raspopovic J, Robert-Moreno A, Richtsmeier JT, Sharpe J. A quantitative method for staging mouse embryos based on limb morphometry. Development. 2018 Apr 5;145(7). pii: dev154856. doi: 10.1242/dev.154856.

ReferenceCitation: Heuzé Y, Kawasaki K, Schwarz T, Schoenebeck JJ, Richtsmeier JT. Developmental and Evolutionary Significance of the Zygomatic Bone. Anat Rec (Hoboken). 2016 Dec;299(12):1616-1630. doi: 10.1002/ar.23449.

ReferenceCitation: Percival CJ, Kawasaki K, Huang Y, Weiss KM, Jabs EW, Li R, Richtsmeier JT. Building Bones. Percival CJ, Richtsmeier JT, editors. Cambridge. Cambridge University Press; 2017. Chapter 2, The contribution of angiogenesis to variation in bone development and evolution; 26-51p.

ReferenceCitation: Kawasaki K, Richtsmeier JT. Building Bones. Percival CJ, Richtsmeier JT, editors. Cambridge. Cambridge University Press; 2017. Chapter 3, Association of the chondrocranium and dermatocranium in early skull development; 52-78p.

ReferenceCitation: Kawasaki K, Richtsmeier J. Building Bones. Percival CJ, Richtsmeier JT, editors. Cambridge. Cambridge University Press; 2017. Chapter Appendix, Appendix to Chapter 3 ; 303-315p.

ReferenceCitation: Motch Perrine SM, Stecko T, Neuberger T, Jabs EW, Ryan TM, Richtsmeier JT. Integration of Brain and Skull in Prenatal Mouse Models of Apert and Crouzon Syndromes. Front Hum Neurosci. 2017 Jul 25;11:369. doi: 10.3389/fnhum.2017.00369. eCollection 2017.

ReferenceCitation: Lee C, Richtsmeier JT, Kraft RH. A COMPUTATIONAL ANALYSIS OF BONE FORMATION IN THE CRANIAL VAULT USING A COUPLED REACTION-DIFFUSION-STRAIN MODEL. J Mech Med Biol. 2017 Jun;17(4). pii: 1750073. doi: 10.1142/S0219519417500737. Epub 2017 May 29.

ReferenceCitation: Lesciotto KM, Heuzé Y, Jabs EW, Bernstein JM, Richtsmeier JT. Choanal Atresia and Craniosynostosis: Development and Disease. Plast Reconstr Surg. 2018 Jan;141(1):156-168. doi: 10.1097/PRS.0000000000003928. Review.

ReferenceCitation: Motch Perrine SM, Wu M, Stephens NB, Kriti D, van Bakel H, Jabs EW, Richtsmeier JT. Mandibular dysmorphology due to abnormal embryonic osteogenesis in FGFR2-related craniosynostosis mice. Dis Model Mech. 2019 May 30;12(5). pii: dmm038513. doi: 10.1242/dmm.038513.

ReferenceCitation: Norwood JN, Zhang Q, Card D, Craine A, Ryan TM, Drew PJ. Anatomical basis and physiological role of cerebrospinal fluid transport through the murine cribriform plate. Elife. 2019 May 7;8. pii: e44278. doi: 10.7554/eLife.44278.

ReferenceCitation: Lee C, Richtsmeier JT, Kraft RH. A coupled reaction-diffusion-strain model predicts cranial vault formation in development and disease. Biomech Model Mechanobiol. 2019 Aug;18(4):1197-1211. doi: 10.1007/s10237-019-01139-z. Epub 2019 Apr 20.

ReferenceCitation: Sewda A, White SR, Erazo M, Hao K, García-Fructuoso G, Fernández-Rodriguez I, Heuzé Y, Richtsmeier JT, Romitti PA, Reva B, Jabs EW, Peter I. Nonsyndromic craniosynostosis: novel coding variants. Pediatr Res. 2019 Mar;85(4):463-468. doi: 10.1038/s41390-019-0274-2. Epub 2019 Jan 14.

ReferenceCitation: Lesciotto KM, Richtsmeier JT. Craniofacial skeletal response to encephalization: How do we know what we think we know? Am J Phys Anthropol. 2019 Jan;168 Suppl 67:27-46. doi: 10.1002/ajpa.23766.

ReferenceCitation: Flaherty K, Richtsmeier JT. It's about Time: Ossification Center Formation in C57BL/6 Mice from E12-E16. J Dev Biol. 2018 Dec 15;6(4). pii: E31. doi: 10.3390/jdb6040031.

ReferenceCitation: Holmes G, O'Rourke C, Motch Perrine SM, Lu N, van Bakel H, Richtsmeier JT, Jabs EW. Midface and upper airway dysgenesis in FGFR2-related craniosynostosis involves multiple tissue-specific and cell cycle effects. Development. 2018 Oct 5;145(19). pii: dev166488. doi: 10.1242/dev.166488.

ReferenceCitation: Martínez-Abadías N, Mateu Estivill R, Sastre Tomas J, Motch Perrine S, Yoon M, Robert-Moreno A, Swoger J, Russo L, Kawasaki K, Richtsmeier J, Sharpe J. Quantification of gene expression patterns to reveal the origins of abnormal morphogenesis. Elife. 2018 Sep 20;7. pii: e36405. doi: 10.7554/eLife.36405.

ReferenceCitation: Holmes G, Zhang L, Rivera J, Murphy R, Assouline C, Sullivan L, Oppeneer T, Jabs EW. C-type natriuretic peptide analog treatment of craniosynostosis in a Crouzon syndrome mouse model. PLoS One. 2018 Jul 26;13(7):e0201492. doi: 10.1371/journal.pone.0201492. eCollection 2018.

ReferenceCitation: Richtsmeier JT. A century of development. Am J Phys Anthropol. 2018 Apr;165(4):726-740. doi: 10.1002/ajpa.23379. Review.

ReferenceCitation: Starbuck JM, Cole TM 3rd, Reeves RH, Richtsmeier JT. The Influence of trisomy 21 on facial form and variability. Am J Med Genet A. 2017 Nov;173(11):2861-2872. doi: 10.1002/ajmg.a.38464. Epub 2017 Sep 21.

ReferenceCitation: Singh N, Dutka T, Reeves RH, Richtsmeier JT. Chronic up-regulation of sonic hedgehog has little effect on postnatal craniofacial morphology of euploid and trisomic mice. Dev Dyn. 2016 Feb;245(2):114-22. doi: 10.1002/dvdy.24361. Epub 2015 Dec 6.

ReferenceCitation: Weiss K, Buchanan A, Richtsmeier J. How are we made?: Even well-controlled experiments show the complexity of our traits. Evol Anthropol. 2015 Jul-Aug;24(4):130-6. doi: 10.1002/evan.21454.

ReferenceCitation: Trainor PA, Richtsmeier JT. Facing up to the challenges of advancing Craniofacial Research. Am J Med Genet A. 2015 Jul;167(7):1451-4. doi: 10.1002/ajmg.a.37065. Epub 2015 Mar 28.

ReferenceCitation: Lee C, Richtsmeier JT, Kraft RH. A computational analysis of bone formation in the cranial vault in the mouse. Front Bioeng Biotechnol. 2015 Mar 19;3:24. doi: 10.3389/fbioe.2015.00024. eCollection 2015.

ipd_sharing: Yes

ipd_sharing_description: Clinical results from chromosomal analysis and DNA mutation analysis performed in CLIA approved laboratories with standard clinical genetic counseling will be given to the patient upon request. Individual research results will be made available to the individual from the PI or their primary care physician (PCP) who has obtained preliminary results from the PI from our non-CLIA laboratory, as long as the PCP can confirm and validate the lab results through a CLIA approved lab before the results are given to the subjects.

lead_sponsor: Icahn School of Medicine at Mount Sinai

location_country:
United States
France
Spain
Germany
United Kingdom

location_facility: National Birth Defects Prevention Study at University of Iowa

location_facility: Birth Defect Registries of New York State

location_facility: The International Craniosynostosis Consortium at University of California at Davis

location_facility: Pennsylvania State Milton S. Hershey Medical Center

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago


ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

lead_sponsor: Amgen

location_country:
United States
France
Spain
Germany
United Kingdom
Canada

title: 20150288 A Phase 2 Open-label Study Investigating the Safety and Efficacy of Blinatumomab After Frontline R-Chemotherapy in Adult Subjects With Newly Diagnosed High-risk Diffuse Large B-Cell Lymphoma (DLBCL)

summary: A phase 2, multicenter, open-label, single arm clinical trial in adults with newly diagnosed aggressive high-risk DLBCL.


title: Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity

summary: The purpose of the study is to determine the effect of setmelanotide (RM-493) on weight, hunger assessments and other factors in patients with rare genetic disorders of obesity.

lead_sponsor: Rhythm Pharmaceuticals, Inc.

location_country:
United States
Netherlands
France
Greece
Israel
Spain
Germany
United Kingdom
Canada

location_facility: Synexus Clinical Research US, Inc. - Akron

location_facility: Synexus Clinical Research US, Inc. - Primary Care Associates, PC

location_facility: Hospital Infantil Universitario Niño Jesús

location_facility: Division of Endocrinology and Diabetes Children's National Hospital

location_facility: Synexus Clinical Research US, Inc. - Central Arizona Medical Associates, PC

location_facility: Synexus Clinical Research US, Inc. - Phoenix Southeast

location_facility: Synexus Clinical Research US, Inc. - Simon Williamson Clinic, PC

location_facility: Synexus Clinical Research US, Inc. - St. Petersburg

location_facility: University of Florida College of Medicine

location_facility: Edmond and Lily Safra Children's Hospital

location_facility: Hopital Trousseau - Nutrition et Gastroentérologie

location_facility: Service de pédiatrie CHU de la Réunion - Hôpital Félix Guyon

location_facility: Synexus Clinical Research US, Inc. - Chicago

location_facility: Synexus Clinical Research US, Inc. - Columbus

location_facility: Precision Medicine for Obesity Research: Gastroenterology & Hepatology Mayo Clinic

location_facility: Synexus Clinical Research US, Inc. - Plano

location_facility: Vanderbilt University School of Medicine

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Synexus Clinical Research US, Inc. - Cincinnati

location_facility: Anschutz Health and Wellness Center University of Colorado Anschutz Medical Campus

location_facility: Synexus Clinical Research US, Inc. - San Antonio


title: A Phase I, Open-Label, Dose-Escalation Study Evaluating the Safety, Tolerability, and Pharmacokinetics of GDC-0077 as a Single Agent in Patients With Locally Advanced or Metastatic PIK3CA-Mutant Solid Tumors and in Combination With Endocrine and Targeted Therapies in Patients With Locally Advanced or Metastatic PIK3CA-Mutant Breast Cancer

summary: This is an open-label, multicenter, Phase I study designed to evaluate the safety, tolerability, and pharmacokinetics of inavolisib administered orally as a single agent in patients with locally advanced or metastatic PIK3CA-mutant solid tumors, including breast cancer, and in combination with standard-of-care endocrine and/or targeted therapies for the treatment of locally advanced or metastatic PIK3CA-mutant breast cancer. Participants will be enrolled in two stages: a dose-escalation stage (Stage I) and an expansion stage (Stage II). Participants will be assigned to one of seven regimens: inavolisib as a single agent (Arm A), inavolisib in combination with palbociclib and letrozole (Arm B), inavolisib in combination with letrozole (Arm C), inavolisib in combination with fulvestrant (Arm D), inavolisib in combination with palbociclib and fulvestrant (Arm E), inavolisib in combination with palbociclib, fulvestrant, and metformin (Arm F), and inavolisib in combination with trastuzumab and pertuzumab (and letrozole or fulvestrant, if applicable (Arm G)).

lead_sponsor: Genentech, Inc.

location_country:
France
Spain
United States
United Kingdom
Canada

location_facility: Hospital Clinico Universitario de Valencia


ipd_sharing_description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

lead_sponsor: Shire

location_country:
Italy
Austria
United States
Peru
India
South Africa
Germany
France
Poland
Israel
Hungary
Australia
Spain
Estonia
Canada
United Kingdom

location_facility: DCT-Shah Research, LLC dba Discovery Clinical Trials

location_facility: S. P. Medical College & Associated Group of Hospitals

location_facility: Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont

location_facility: Centrum Medyczne Uno-Med (Private Practice)

location_facility: The Eye Center at Southern College of Optometry

location_facility: Centrum Diagnostyki i Mikrochirurgii Oka LENS

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: The Regents of the University of Michigan

location_facility: The Ottawa Hospital - General Campus, University of Ottawa Eye Institute

location_facility: Emerson Clinical Research Institute, LLC

location_facility: Hospital Universitari de Girona Dr Josep Trueta

location_facility: McGill University Health Centre/Glen Site / Royal Victoria Hospital

location_facility: Vienna Institute for Research in Ocular Surgery

location_facility: University of the Sunshine Coast Clinical Trials Centre

location_facility: Klinisches Studienzentrum der Augenklinik

location_facility: Pretoria Eye Institute Research Foundation

location_facility: M. S. Ramaiah Medical College and Hospital

location_facility: Complejo Asistencial Universitario de Burgos

location_facility: Nelson R Mandela School of Medicine Ophthalmology Department

location_facility: K.L.E. Society's Dr. Prabhakar Kore Hospital and Medical Research Centre

location_facility: University of Waterloo School of Optometry and Vision Science

location_facility: Andrew Gardner Logan, MD / dba Logan Ophthalmic Research, LLC

location_facility: MediSphere Medical Research Center, an AMR affiliate

location_facility: ICARE Eye Hospital and Post Graduate Institute

location_facility: Hospital Clinico Universitario Lozano Blesa

title: A Phase 3, Multi-center, Randomized, Double-Masked Study to Evaluate the Clinical Efficacy and Safety of SHP640 (PVP-Iodine 0.6% and Dexamethasone 0.1%) Ophthalmic Suspension Compared to PVP-Iodine and Placebo in the Treatment of Adenoviral Conjunctivitis

summary: The purpose of this study is to determine if an investigational treatment is effective compared with placebo and PVP-Iodine in the treatment of adults and children with adenoviral conjunctivitis.


publications_result: Flamm S, Lawitz E, Borg B, Charlton M, Landis C, Reddy R, et al. High Efficacy and Improvement in CPT Class With Sofosbuvir/Velpatasvir Plus Ribavirin for 12 Weeks in Patients With CPT C Decompensated Cirrhosis [Poster THU-138]. EASL: The International Liver Congress; 2019 10-14 April; Vienna, Austria.

ReferenceType: result

ipd_sharing_description: Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at https://www.gilead.com/science-and-medicine/research/clinical-trials-transparency-and-data-sharing-policy.

lead_sponsor: Gilead Sciences

location_facility: University of Washington/ Harborview Medical Center

location_facility: Southern Therapy and Advanced Research LLC

location_facility: Hospital of the University of Pennsylvania

location_facility: American Research Corporation at Texas Liver Institute

location_facility: Bon Secours St. Mary's Hospital of Richmond, Inc. d/b/a Bon Secours Liver Institute of Virginia

location_facility: Northwestern Memorial Hospital; Clinical Research Unit

location_facility: Intermountain Liver Disease and Transplant Center

title: A Phase 2, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Sofosbuvir/Velpatasvir Fixed-Dose Combination and Ribavirin for 12 Weeks in Subjects With Chronic HCV Infection and Child-Pugh-Turcotte Class C Cirrhosis

summary: The primary objectives of this study are to evaluate the efficacy, safety, and tolerability of the treatment with sofosbuvir velpatasvir (SOF/VEL) fixed-dose combination (FDC) with ribavirin (RBV) for 12 weeks in participants with chronic hepatitis C virus (HCV) infection and Child-Pugh-Turcotte (CPT) Class C cirrhosis.


title: Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers

summary: This is an open-label, single arm, multi-center, pilot study of Nivolumab in pediatric patients with recurrent or refractory hypermutant malignancies aged 12 months to 18 years of age. This study is to assess clinical and radiological benefits of treatment with Nivolumab in children with hypermutated cancers, including those with bMMRD syndrome. It is our expectation that patients with bMMRD syndrome will account for the majority of patients enrolled on this study.

ReferenceCitation: Rittberg R, Harlos C, Rothenmund H, Das A, Tabori U, Sinha N, Singh H, Chodirker B, Kim CA. Immune Checkpoint Inhibition as Primary Adjuvant Therapy for an IDH1-Mutant Anaplastic Astrocytoma in a Patient with CMMRD: A Case Report-Usage of Immune Checkpoint Inhibition in CMMRD. Curr Oncol. 2021 Feb 1;28(1):757-766. doi: 10.3390/curroncol28010074.

family: Rittberg

full_name: Rebekah Rittberg

given: Rebekah

family: Harlos

full_name: Craig Harlos

given: Craig

family: Rothenmund

full_name: Heidi Rothenmund

given: Heidi

family: Das

full_name: Anirban Das

given: Anirban

family: Tabori

full_name: Uri Tabori

given: Uri

family: Sinha

full_name: Namita Sinha

given: Namita

family: Singh

full_name: Harminder Singh

given: Harminder

family: Chodirker

full_name: Bernie Chodirker

given: Bernie

family: Kim

full_name: Christina A. Kim

given: Christina A.

currency_apc_doaj: CHF

journal_name: Current Oncology

oa_colors: green gold

publisher: MDPI AG

publisher_dissemination: MDPI

publisher_group: MDPI

publisher_normalized: MDPI

title: Immune Checkpoint Inhibition as Primary Adjuvant Therapy for an IDH1-Mutant Anaplastic Astrocytoma in a Patient with CMMRD: A Case Report—Usage of Immune Checkpoint Inhibition in CMMRD

lead_sponsor: The Hospital for Sick Children

location_country:
United States
France
Israel
Australia
Canada

location_facility: Children's & Women's Health Centre of British Columbia

lead_sponsor_type: academique


lead_sponsor: Janssen Research & Development, LLC

location_country:
United States
Germany
France
Poland
Belgium
Canada

acronym: STELARA

location_facility: Center For Digestive Health Systems-Greenville

location_facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu

location_facility: Children's Center For Digestive Healthcare, Llc

location_facility: Universitair Kinderziekenhuis Koningin Fabiola

type: Other Identifier

title: A Randomized Double-blind Pharmacokinetic Study of Ustekinumab in Pediatric Subjects With Moderately to Severely Active Crohn's Disease

summary: The purpose of this study is to evaluate the pharmacokinetics (PK) of ustekinumab in subjects from 2 through less than (<) 18 years old in the USA, or 6 through less than (<) 18 years old in other countries and determine if it is similar to that observed in adults with moderately to severely active Crohn's disease (CD). Also to assess the safety, immunogenicity and efficacy of ustekinumab in the treatment of moderately to severely active CD. The main part of the study continues to Week 16, at which point all subjects who are receiving benefit from ustekinumab maintenance therapy (as determined by the investigator) are eligible to enter the long-term extension (LTE) and continue to receive ustekinumab. The study extension ends at Week 268 or upon availability of the LTE basket study (CNTO1275ISD3001) whichever occurs first. If participants do not consent/assent to the LTE basket study, they will continue safety follow-up for approximately 20 weeks after the last study agent administration.


ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

lead_sponsor: Eli Lilly and Company

location_country:
Brazil
India
Saudi Arabia
Mexico
Germany
France
Hungary
Puerto Rico
China
Turkey
United States
United Kingdom

acronym: AWARD-PEDS

location_facility: Centro de Diabetes y Endocrinologia Pediatrica de PR

location_facility: No.2 Hospital Affiliated to Jilin University

location_facility: Hospital da Clinicas da Faculdade de Medicina da USP

location_facility: The Second Affiliated Hospital of Xian Jiaotong University

location_facility: Seattle Children's Hospital Research Foundation

location_facility: Instituto Estadual de Diabetes e Endocrinologia

location_facility: Childrens Hospital of Suzhou Medical College

location_facility: Cincinnati Childrens Hospital Medical Center

location_facility: Dr Jivraj Mehta Smarak Health Foundation Bakeri Medical Research Centre

location_facility: Ondokuz Mayis University Medical Faculty

location_facility: Beijing Peking Union Medical College Hospital

location_facility: King Salman bin Abdulaziz Hospital - Diabetic Center

location_facility: Center of Excellence in Diabetes & Endocrinology

location_facility: Division of Endocrinology, Diabetes, and Metabolism

location_facility: Health Pharma Professional Research, S.A. de C.V.

location_facility: Florida Center for Endocrinology & Metabolism

location_facility: Deenanath Mangeshkar Hospital & Research Centre

location_facility: Hôpitaux Universitaires Paris Sud - Hôpital Bicêtre

location_facility: Post Graduate Institute of Medical Education & Research

title: A Randomized, Double-Blind Study With an Open-Label Extension Comparing the Effect of Once-Weekly Dulaglutide With Placebo in Pediatric Patients With Type 2 Diabetes Mellitus (AWARD-PEDS: Assessment of Weekly AdministRation of LY2189265 in Diabetes-PEDiatric Study)

summary: The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of the study drug dulaglutide compared to placebo in pediatric participants with type 2 diabetes. The study duration is approximately 60 weeks.


title: A Single Arm, Open-Label, Phase 2 Study of Melflufen in Combination With Dexamethasone in Patients With Relapsed Refractory Multiple Myeloma Who Are Refractory to Pomalidomide and/or an Anti-CD38 Monoclonal Antibody

acronym: HORIZON

summary: This study will evaluate melflufen in combination with dexamethasone in the treatment of relapsed refractory multiple myeloma in adult patients with disease refractory to pomalidomide and/or an anti-CD38 monoclonal antibody. All patients in the study will be treated with melflufen on Day 1 and dexamethasone on Days 1, 8, 15 and 22 of each 28-day cycle.

ReferenceCitation: Richardson PG, Oriol A, Larocca A, Bladé J, Cavo M, Rodriguez-Otero P, Leleu X, Nadeem O, Hiemenz JW, Hassoun H, Touzeau C, Alegre A, Paner A, Maisel C, Mazumder A, Raptis A, Moreb JS, Anderson KC, Laubach JP, Thuresson S, Thuresson M, Byrne C, Harmenberg J, Bakker NA, Mateos MV; HORIZON (OP-106) Investigators. Melflufen and Dexamethasone in Heavily Pretreated Relapsed and Refractory Multiple Myeloma. J Clin Oncol. 2021 Mar 1;39(7):757-767. doi: 10.1200/JCO.20.02259. Epub 2020 Dec 9.

name: Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA

family: Richardson

full_name: Paul G. Richardson

given: Paul G.

name: Institut Català d'Oncologia and Josep Carreras Research Institute, Hospital Germans Trias i Pujol, Badalona, Spain

family: Oriol

full_name: Albert Oriol

given: Albert

name: Myeloma Unit, Division of Hematology, University of Torino, Azienda Ospedaliero-Universitaria Città della Salute e della Scienza, Torino, Italy

family: Larocca

full_name: Alessandra Larocca

given: Alessandra

name: Hematology Department, IDIBAPS, Hospital Clinic, Barcelona, Spain

family: Bladé

full_name: Joan Bladé

given: Joan

name: Seràgnoli Institute of Hematology, Bologna University School of Medicine, Bologna, Italy

family: Cavo

full_name: Michele Cavo

given: Michele

name: Clínica Universidad de Navarra, Pamplona, Spain

family: Rodriguez-Otero

full_name: Paula Rodriguez-Otero

given: Paula

family: Leleu

full_name: Xavier Leleu

given: Xavier

family: Nadeem

full_name: Omar Nadeem

given: Omar

name: Division of Hematology-Oncology, Department of Medicine, University of Florida, Gainesville, FL

family: Hiemenz

full_name: John W. Hiemenz

given: John W.

name: Myeloma Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY

family: Hassoun

full_name: Hani Hassoun

given: Hani

name: Centre de Recherche en Cancérologie et Immunologie Nantes-Angers (CRCINA), Institut National de la Santé et de la Recherche Médicale (INSERM), Centre National de la Recherche Scientifique (CNRS), Université d'Angers, Université de Nantes, Nantes, France

name: Site de Recherche Intégrée sur le Cancer (SIRIC), Imaging and Longitudinal Investigations to Ameliorate Decision-making (ILIAD), Nantes, France

name: Service d'hématologie Clinique, Centre Hospitalier Universitaire, Place Alexis Ricordeau, Nantes, France

family: Touzeau

full_name: Cyrille Touzeau

given: Cyrille

name: Hospital Universitario La Princesa and Hospital Universitario Quironsalud, Madrid, Spain

family: Alegre

full_name: Adrián Alegre

given: Adrián

name: Rush University Medical Center, Chicago, IL

family: Paner

full_name: Agne Paner

given: Agne

name: Baylor Scott & White Charles A. Sammons Cancer Center, Dallas, TX

family: Maisel

full_name: Christopher Maisel

given: Christopher

name: The Oncology Institute of Hope and Innovation, Glendale, CA

family: Mazumder

full_name: Amitabha Mazumder

given: Amitabha

name: Division of Hematology-Oncology, Department of Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA

family: Raptis

full_name: Anastasios Raptis

given: Anastasios

name: Novant Health Hematology, Novant Health Forsyth Medical Center, Winston-Salem, NC

family: Moreb

full_name: Jan S. Moreb

given: Jan S.

family: Anderson

full_name: Kenneth C. Anderson

given: Kenneth C.

family: Laubach

full_name: Jacob P. Laubach

given: Jacob P.

family: Thuresson

full_name: Sara Thuresson

given: Sara

full_name: Marcus Thuresson

given: Marcus

family: Byrne

full_name: Catriona Byrne

given: Catriona

family: Harmenberg

full_name: Johan Harmenberg

given: Johan

name: Hospital Clínico Universitario de Salamanca/IBSAL/CIC, Salamanca, Spain

name: on behalf of the HORIZON (OP-106) Investigators

repository_institution: University of Turin - Institutional Research Information System University of Turin

evidence: oa repository (via OAI-PMH title and first author match)

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Diderot

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inserm

publisher: American Society of Clinical Oncology (ASCO)

location_facility: Hospital Clinic i Provincial de Barcelona

location_facility: Innovative Clinical Research Institute (ICRI)

location_facility: Institut Català d'Oncología (ICO) Badalona

location_facility: Hospital Universitario Fundación Jiménez Díaz


title: Study of Safety and Efficacy of Multiple VAY736 Doses in Patients With Moderate to Severe Primary Sjogren's Syndrome (pSS)

summary: This study will determine the dose-response relationship of VAY736 for key efficacy and safety parameters

ReferenceCitation: Bowman SJ, Fox R, Dörner T, Mariette X, Papas A, Grader-Beck T, Fisher BA, Barcelos F, De Vita S, Schulze-Koops H, Moots RJ, Junge G, Woznicki JN, Sopala MA, Luo WL, Hueber W. Safety and efficacy of subcutaneous ianalumab (VAY736) in patients with primary Sjögren's syndrome: a randomised, double-blind, placebo-controlled, phase 2b dose-finding trial. Lancet. 2021 Nov 30. pii: S0140-6736(21)02251-0. doi: 10.1016/S0140-6736(21)02251-0. [Epub ahead of print]

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

location_country:
Italy
Austria
Chile
Netherlands
Spain
Germany
France
Japan
Poland
Israel
Romania
Hungary
Argentina
Belgium
Russian Federation
Portugal
United States
United Kingdom
Taiwan


title: A Phase 1b Open-Label Study Investigating the Safety and Pharmacokinetics of Administration of Subcutaneous Blinatumomab for the Treatment of Relapsed/Refractory Indolent Non-Hodgkin's Lymphoma

summary:
Primary Objective:

• To evaluate the safety and tolerability of subcutaneous (SC) blinatumomab dose administrations

Secondary Objectives:

To determine pharmacokinetics (PK) with continuous intravenous (cIV) and SC administrations
To estimate the maximum tolerated dose (MTD) tested for blinatumomab administered subcutaneously
To determine the incidence of anti-blinatumomab antibody formation following SC administration
To evaluate efficacy response following treatment with SC blinatumomab administration

Exploratory Objective:

To determine the pharmacodynamics (PD) time profiles for B-and T-lymphocytes as well as cytokine profiles during SC administration
To evaluate efficacy response following treatment with SC blinatumomab administration using Lugano criteria if positron emission tomography-computed tomography (PET/CT) is used for evaluation

ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

location_country:
Italy
United States
France
Australia
Germany
United Kingdom

location_facility: Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia

location_facility: Azienda Ospedaliera Universitaria di Bologna Policlinico S Orsola Malpighi

location_facility: Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII


title: A 5-year Longitudinal Observational Study of the Natural History and Management of Patients With Hepatocellular Carcinoma (HCC)

summary: TARGET-HCC is a longitudinal, observational study of patients being managed for HCC in usual clinical practice. TARGET-HCC will create a research registry of participants with HCC within academic and community real-world practices in order to assess the safety and effectiveness of the entire spectrum of current and future therapies across diverse populations.

ReferenceCitation: Cabrera R, Singal AG, Colombo M, Kelley RK, Lee H, Mospan AR, Meyer T, Newell P, Parikh ND, Sangro B, Reddy KR, Watkins S, Zink RC, Di Bisceglie AM. A Real-World Observational Cohort of Patients with Hepatocellular Carcinoma: Design and Rationale for TARGET-HCC. Hepatol Commun. 2020 Dec 21;5(3):538-547. doi: 10.1002/hep4.1652. eCollection 2021 Mar.

name: University of Florida Gainesville FL USA

family: Cabrera

full_name: Roniel Cabrera

given: Roniel

name: University of Texas Southwestern Medical Center Dallas TX USA

family: Singal

full_name: Amit G. Singal

given: Amit G.

name: Center for Translational Research in Liver Disease Humanitas Hospital Rozzano Italy

family: Colombo

full_name: Massimo Colombo

given: Massimo

name: University of California San Francisco San Francisco CA USA

family: Kelley

full_name: R. Kate Kelley

given: R. Kate

name: Division of Gastroenterology, Hepatology and Nutrition Virginia Commonwealth University Richmond VA USA

family: Lee

full_name: Hannah Lee

given: Hannah

family: Mospan

full_name: Andrea R. Mospan

given: Andrea R.

name: Royal Free Hospital and University College London Cancer InstituteUniversity College London London United Kingdom

family: Meyer

full_name: Tim Meyer

given: Tim

family: Newell

full_name: Pippa Newell

given: Pippa

name: Division of Internal Medicine University of Michigan Ann Arbor MI USA

family: Parikh

full_name: Neehar D. Parikh

given: Neehar D.

name: Department of Internal Medicine Clinica Universidad de Navarra Madrid Spain

family: Sangro

full_name: Bruno Sangro

given: Bruno

name: Department of Medicine University of Pennsylvania Philadelphia PA USA

family: Reddy

full_name: K. Rajender Reddy

given: K. Rajender

family: Watkins

full_name: Stephanie Watkins

given: Stephanie

family: Zink

full_name: Richard C. Zink

given: Richard C.

name: Division of Gastroenterology and Hepatology St. Louis University St. Louis MO USA

family: Di Bisceglie

full_name: Adrian M. Di Bisceglie

given: Adrian M.

journal_name: Hepatology Communications

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

repositories:
PubMed Central
UCL Discovery

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: A Real‐World Observational Cohort of Patients with Hepatocellular Carcinoma: Design and Rationale for TARGET‐HCC

lead_sponsor: Target PharmaSolutions, Inc.

location_country:
Italy
United States
France
Spain
Germany

location_facility: Northwell Health - Center for Liver Diseases

location_facility: Northwestern University Division of Gastroenterology and Hepatology

location_facility: Center for Liver Disease and Transplant at CMC

location_facility: University of North Carolina at Chapel Hill

location_facility: Hospital Universitario Marques de Valdecilla

location_facility: University of Southern California/Norris Comprehensive Cancer Center

location_facility: Complejo Hospitalario Universitario A Coruña

location_facility: University of Florida Health Gastroenterology

location_facility: University of Iowa Hospitals and Clinics

location_facility: Southern Therapy and Advanced Research (STAR) LLC

location_facility: Rutgers-Robert Wood Johnson Medical School

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: Providence Health & Services Cancer Clinical Trials

location_facility: University of Florida Hepatology Research at CTRB

location_facility: Clinical Research Institute@ Methodist Dallas Medical Center

location_facility: Schiff Center for Liver Diseases/University of Miami

location_facility: Vanderbilt University Medical Center - GI Research Office


title: Saline Hypertonic in Preschoolers With Cystic Fibrosis and Lung Structure as Measured by Computed Tomography (CT)

acronym: SHIP-CT

summary: The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice daily for 48 weeks reduces structural lung disease as assessed by computed tomography (CT) in comparison with inhalation of 0.9% isotonic saline (IS) in preschool children (ages 3 to 6) with cystic fibrosis.

ReferenceCitation: Rosenow T, Oudraad MC, Murray CP, Turkovic L, Kuo W, de Bruijne M, Ranganathan SC, Tiddens HA, Stick SM; Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF). PRAGMA-CF. A Quantitative Structural Lung Disease Computed Tomography Outcome in Young Children with Cystic Fibrosis. Am J Respir Crit Care Med. 2015 May 15;191(10):1158-65. doi: 10.1164/rccm.201501-0061OC.

ReferenceCitation: Ramsey KA, Rosenow T, Turkovic L, Skoric B, Banton G, Adams AM, Simpson SJ, Murray C, Ranganathan SC, Stick SM, Hall GL; AREST CF. Lung Clearance Index and Structural Lung Disease on Computed Tomography in Early Cystic Fibrosis. Am J Respir Crit Care Med. 2016 Jan 1;193(1):60-7. doi: 10.1164/rccm.201507-1409OC.

lead_sponsor: University of Washington, the Collaborative Health Studies Coordinating Center

location_country:
Italy
United States
Netherlands
France
Belgium
Australia
Spain
Denmark
Canada

location_facility: Universitair Ziekenhuis Children's Hospital

location_facility: Washington University School of Medicine

location_facility: Copenhagen University Hospital Rigshospitalet

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Sophia Children's Hospital at Erasmus Medical Centre


title: A Phase 1b Dose Escalation Study Evaluating the Safety and Pharmacokinetics of Venetoclax in Combination With Azacitidine in Subjects With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

summary: This is a Phase 1b, open-label, non-randomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in participants with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.

lead_sponsor: AbbVie

location_country:
Italy
United States
Germany
France
Australia
Canada
United Kingdom

location_facility: Universitaetsklinikum Leipzig /ID# 153142

location_facility: Weill Cornell Medical College /ID# 155524

location_facility: Norfolk and Norwich Univ Hosp /ID# 156492

location_facility: Univ Maryland School Medicine /ID# 153669

location_facility: UT MD Anderson Cancer Center /ID# 153809

location_facility: St. Vincent's Hospital, Darlinghurst /ID# 222846

location_facility: Columbia University Medical Center /ID# 153661

location_facility: Oregon Health and Science University /ID# 152734

location_facility: Washington University-School of Medicine /ID# 153671

location_facility: Universitaetsklinikum Carl Gus /ID# 153958

location_facility: Tennessee Oncology-Nashville Centennial /ID# 222769

location_facility: A.O.U. Policlinico S.Orsola-Malpighi /ID# 153763

location_facility: Azienda Policlinico Umberto I /ID# 153764

location_facility: Universitaetsklinikum Halle (S /ID# 153760

location_facility: Concord Repatriation & Gen Hos /ID# 154958

location_facility: Dana-Farber Cancer Institute /ID# 152735

location_facility: Heart of England NHS Foundation Trust /ID# 158810

location_facility: University of Arizona Cancer Center - North Campus /ID# 154155

location_facility: Universitatsklinikum Mannheim /ID# 153140


lead_sponsor: Novo Nordisk A/S

location_country:
Netherlands
Germany
France
Spain
Denmark
United States

title: This Trial is Conducted in Europe and the United States of America. The Aim of This Trial is to Investigate Single Dose Pharmacokinetics (the Exposure of the Trial Drug in the Body) and Safety of Turoctocog Alfa Pegol From the Pivotal Process and Turoctocog Alfa Pegol From the Commercial Process in Patients With Severe Haemophilia A.

summary: Investigating single dose pharmacokinetics and safety of turoctocog alfa pegol from the pivotal process and turoctocog alfa pegol from the commercial process in patients with severe haemophilia A


title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart Failure

summary: This is a phase 2 randomized, double-blind, placebo-controlled study to evaluate the cardiac and renal effects of short term treatment with elamipretide in patients hospitalized with congestion due to heart failure

lead_sponsor: Stealth BioTherapeutics Inc.

location_country:
Serbia
United Kingdom
Netherlands
France
Bulgaria
Poland
Hungary
Belgium
Spain
Latvia

location_facility: Clinic of Internal Diseases, "Multiprofile Hospital for Active Treatment, "Sveta Anna Sofia" AD

location_facility: Department of Cardiology, "Multiprofile Regional Hospital for Active Treatement Dr. Stefan Cherkezov" AD

location_facility: Riga East Clinical University Hospital, Clinic Gailezers

location_facility: Pauls Stradins Clinical University Hospital, Latvian Centre of Cardiology

location_facility: Hospital Clínico Universitario de Valencia

location_facility: Clinic of Cardiology, "City Clinic University Multiprofile Hospital for Active Treatment" EOOD

location_facility: Clinic of Cardiology, "Second Multiprofile Hospital for Active Treatement - Sofia" EAD

location_facility: Institute for Cardiovascular Diseases of Vojvodina

location_facility: Department of Internal Diseases, "Multiprofile Hospital for Active Treatment Sveta Ekaterina - Dimitrovgrad" EOOD

location_facility: Complexo Hospitalario Universitario de A Coruña

location_facility: Hospital Universitari i Politècnic La Fe

location_facility: Clinical Centre of Serbia, Institute for Cardiovascular Diseases

location_facility: Clinic of Cardiology, Multiprofile Hospital for Active Treatment National Heart Hospital" EAD

location_facility: Clinical Centre of Serbia, Emergency Centre

location_facility: Hungarian Institute Of Cardiology, Department of Adult Cardiology

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: Semmelweis University Heart and Vascular Center

location_facility: Clinical Hospital Centre Bezanijska kosa

location_facility: Consorci Hospital General Universitari de Valencia

location_facility: Bieganski Provincial Specialist Hospital, Department of Cardiology, Clinic of Cardiology UM

location_facility: Cardinal Stefan Wyszynski, Institute of Cardiology, Clinic Heart Failure and Transplantation

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Cardinal Stefan Wyszynski, Institute of Cardiology, Clinic of Coronary Heart Disease and Structural Heart Disease

location_facility: County St. George University Teaching Hospital, Department of Internal Medicine Cardiology

location_facility: Zala County Hospital Cardiology Department


ReferenceCitation: Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Xiong H, Zanoteli E, Baranello G, Bruno C, Vlodavets D, Wang Y, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Fontoura P, Servais L; FIREFISH Working Group. Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls. N Engl J Med. 2021 Jul 29;385(5):427-435. doi: 10.1056/NEJMoa2102047.

name: From the Department of Neurology, Boston Children’s Hospital, Harvard Medical School, Boston (B.T.D.); the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (R.M., G.B.), and the Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa (C.B.) — both in Italy; the Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland (M.M.-B.); the Paediatric Gait Analysis Service of New South Wales, the Children’s Hospital...

family: Darras

full_name: Basil T. Darras

given: Basil T.

family: Masson

full_name: Riccardo Masson

given: Riccardo

family: Mazurkiewicz-Bełdzińska

full_name: Maria Mazurkiewicz-Bełdzińska

given: Maria

family: Rose

full_name: Kristy Rose

given: Kristy

family: Xiong

full_name: Hui Xiong

given: Hui

family: Zanoteli

full_name: Edmar Zanoteli

given: Edmar

family: Baranello

full_name: Giovanni Baranello

given: Giovanni

family: Bruno

full_name: Claudio Bruno

given: Claudio

family: Vlodavets

full_name: Dmitry Vlodavets

given: Dmitry

family: Wang

full_name: Yi Wang

given: Yi

family: El-Khairi

full_name: Muna El-Khairi

given: Muna

family: Gerber

full_name: Marianne Gerber

given: Marianne

family: Gorni

full_name: Ksenija Gorni

given: Ksenija

family: Khwaja

full_name: Omar Khwaja

given: Omar

family: Kletzl

full_name: Heidemarie Kletzl

given: Heidemarie

family: Scalco

full_name: Renata S. Scalco

given: Renata S.

family: Fontoura

full_name: Paulo Fontoura

given: Paulo

family: Servais

full_name: Laurent Servais

given: Laurent

journal_name: New England Journal of Medicine

publisher: Massachusetts Medical Society

publisher_dissemination: Massachusetts Medical Society

publisher_group: Massachusetts Medical Society

publisher_normalized: Massachusetts Medical Society

ReferenceCitation: Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, Mercuri E, Rose K, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Seabrook T, Fontoura P, Servais L; FIREFISH Working Group. Risdiplam in Type 1 Spinal Muscular Atrophy. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24.

name: From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) — all in the United Kingdom; the Developmental Neurology Unit, Fondazione...

family: Day

full_name: John W. Day

given: John W.

family: Deconinck

full_name: Nicolas Deconinck

given: Nicolas

family: Klein

repositories_institution: University College London - UCL Discovery

ipd_sharing_description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

location_country:
Italy
Brazil
Serbia
Saudi Arabia
Switzerland
Spain
Croatia
France
Japan
Poland
Belgium
Russian Federation
China
Turkey
United States
Ukraine

location_facility: Fondazione IRCCS Istituto Neurologico "Carlo Besta"; UO di Neurologia dello Sviluppo

location_facility: The Children's Memorial Health Institute Department of Neurology and Epileptology

location_facility: IRCCS Ospedale Pediatrico Bambino Gesù; U.O. Malattie Neuromuscolari e Neurodegenerative

location_facility: Columbia University Medical Center; The Neurological Institute of New York

location_facility: Hospital Yeditepe University Kozyatagi; Pediatry

location_facility: Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie

location_facility: Hospital Universitari Vall d'Hebron; Area Genética Clínica y Molecular

location_facility: Hospital das Clinicas - FMUSP_X; Neurologia

location_facility: Instituto de Puericultura E Pediatria Martagão Gesteira

location_facility: Hacettepe University, School of Medicine; Pediatrics Department; Pediatrics Child Neurology Unit

location_facility: Hopital Femme Mere Enfant; Medecine Physique et Readaptation Pediatrique - L'ESCALE

location_facility: Institute for Mother and Child Dr. Vukan Cupic

location_facility: Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile

location_facility: Ams of Ukraine; Inst. of Neurology, Psychiatry & Narcology

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Unità Operativa Complessa di Neurologia

location_facility: IRCCS Istituto Giannina Gaslini; U.O.S.D. Centro di Miologia e Patologie Neurodegenerative

location_facility: Szpital Gdanskiego Uniwersytetu Medycznego; Clinic of developmental neurology

location_facility: Russian Children Neuromuscular Center of Veltischev

location_facility: Clinical Medical Center Zagreb; University Hospital Rebro Department of Paediatrics

location_facility: Ann and Robert H. Lurie Children Hospital of Chicago

location_facility: King Faisal Specialist Hospital and Research Centre Building

title: A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy

summary: Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate Risdiplam (RO7034067) for 24-months at the dose selected in Part 1.


title: A Prospective, Randomized, International, Multicenter, Double-arm, Controlled, Open-label Study of Riociguat in Patients With Pulmonary Arterial Hypertension (PAH) Who Are on a Stable Dose of Phosphodiesterase-5 Inhibitors (PDE-5i) With or Without Endothelin Receptor Antagonist (ERA), But Not at Treatment Goal

acronym: REPLACE

summary: To demonstrate the effectiveness of riociguat as replacement of phosphodiesterase-5 inhibitors (PDE-5i) therapy in pulmonary arterial hypertension (PAH) patients

ReferenceCitation: Hoeper MM, Al-Hiti H, Benza RL, Chang SA, Corris PA, Gibbs JSR, Grünig E, Jansa P, Klinger JR, Langleben D, McLaughlin VV, Meyer GMB, Ota-Arakaki J, Peacock AJ, Pulido T, Rosenkranz S, Vizza CD, Vonk-Noordegraaf A, White RJ, Chang M, Kleinjung F, Meier C, Paraschin K, Ghofrani HA, Simonneau G; REPLACE investigators. Switching to riociguat versus maintenance therapy with phosphodiesterase-5 inhibitors in patients with pulmonary arterial hypertension (REPLACE): a multicentre, open-label, randomised controlled trial. Lancet Respir Med. 2021 Jun;9(6):573-584. doi: 10.1016/S2213-2600(20)30532-4. Epub 2021 Mar 24.

family: Hoeper

full_name: Marius M Hoeper

given: Marius M

family: Al-Hiti

full_name: Hikmet Al-Hiti

given: Hikmet

family: Benza

full_name: Raymond L Benza

given: Raymond L

family: Chang

full_name: Sung-A Chang

given: Sung-A

family: Corris

full_name: Paul A Corris

given: Paul A

family: Gibbs

full_name: J Simon R Gibbs

given: J Simon R

family: Grünig

full_name: Ekkehard Grünig

given: Ekkehard

family: Jansa

full_name: Pavel Jansa

given: Pavel

family: Klinger

full_name: James R Klinger

given: James R

family: Langleben

full_name: David Langleben

given: David

family: McLaughlin

full_name: Vallerie V McLaughlin

given: Vallerie V

family: Meyer

full_name: Gisela M B Meyer

given: Gisela M B

family: Ota-Arakaki

full_name: Jaquelina Ota-Arakaki

given: Jaquelina

family: Peacock

full_name: Andrew J Peacock

given: Andrew J

family: Pulido

full_name: Tomás Pulido

given: Tomás

family: Rosenkranz

full_name: Stephan Rosenkranz

given: Stephan

family: Vizza

full_name: Carmine Dario Vizza

given: Carmine Dario

family: Vonk-Noordegraaf

full_name: Anton Vonk-Noordegraaf

given: Anton

family: White

full_name: R James White

given: R James

full_name: Mikyung Chang

given: Mikyung

family: Kleinjung

full_name: Frank Kleinjung

given: Frank

family: Meier

full_name: Christian Meier

given: Christian

family: Paraschin

full_name: Karen Paraschin

location_country:
Greece
Belgium
Denmark
Taiwan
France
Japan
Poland
Canada
Korea, Republic of
Netherlands
Portugal
United States
Germany
United Kingdom
Italy
Austria
Brazil
Mexico
Czechia
Spain
Turkey


title: A Multicenter, Randomized, Double-blind, Active-controlled Study to Evaluate the Effects of LCZ696 Compared to Valsartan on Cognitive Function in Patients With Chronic Heart Failure and Preserved Ejection Fraction

acronym: PERSPECTIVE

summary: The purpose of this study is to evaluate the effect of LCZ696 compared to valsartan on cognitive function in patients with chronic heart failure and preserved ejection fraction. Cognitive function will be assessed using a comprehensive battery of tests with an evaluation of longitudinal change of cognitive domains including memory, executive function, and attention.

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
Switzerland
Netherlands
Spain
Germany
Croatia
Bulgaria
France
Korea, Republic of
Lithuania
Poland
Belgium
Argentina
Australia
Russian Federation
Turkey
United States
United Kingdom
Canada
Taiwan


title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Efficacy, Pharmacodynamics, and Pharmacokinetics of Lademirsen (SAR339375) for Subcutaneous Injection Administered Every Week in Patients With Alport Syndrome

acronym: HERA

summary:
Primary Objectives:

To assess the efficacy of lademirsen (SAR339375) in reducing the decline in renal function
To assess the safety and tolerability of lademirsen (SAR339375) in subjects with Alport syndrome

Secondary Objectives:

To assess plasma pharmacokinetic (PK) parameters of the parent compound and its metabolites
To assess the potential formation of anti-drug antibodies (ADAs) following administration of lademirsen (SAR339375)
To assess the pharmacodynamic effect of lademirsen (SAR339375) on miR-21 and on changes in renal injury and function biomarkers.

ReferenceCitation: Kashtan CE, Gross O. Clinical practice recommendations for the diagnosis and management of Alport syndrome in children, adolescents, and young adults-an update for 2020. Pediatr Nephrol. 2021 Mar;36(3):711-719. doi: 10.1007/s00467-020-04819-6. Epub 2020 Nov 6. Review. Erratum in: Pediatr Nephrol. 2021 Jan 12;:.

family: Kashtan

full_name: Clifford E. Kashtan

given: Clifford E.

family: Gross

full_name: Oliver Gross

given: Oliver

journal_name: Pediatric Nephrology

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

title: Clinical practice recommendations for the diagnosis and management of Alport syndrome in children, adolescents, and young adults–an update for 2020

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com/

lead_sponsor: Genzyme, a Sanofi Company

location_country:
United States
Spain
Germany
France
Australia
China
Canada
United Kingdom

location_facility: Columbia University Medical Center_Investigational Site Number :8400004

location_facility: The Cleveland Clinic Foundation_Investigational Site Number :8400001

location_facility: University of Utah_Investigational Site Number :8400005

location_facility: University of Minnesota Childrens' Hospital_Investigational Site Number :8400003

location_facility: Universitätsmedizin Göttingen, Klinik für Nephrologie und Rheumatologie_Investigational Site Number :2760002

location_facility: Uniklinik Köln, Innere Medizin II - Nephrologie, Rheumatologie, Diabetologie und Allgemeine Innere Medizin_Investigational Site Number :2760001


title: Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

summary:
it is a randomised, double blind, parallel group, placebo controlled study. A total of 179 male ambulant subjects will be randomised 2:1 (givinostat:placebo).

Subjects will be stratified for their concomitant use of steroids in 4 strata:

Deflazacort daily regimen
Deflazacort intermittent regimen
Other steroids daily regimen
Other steroids intermittent regimen. The study duration is planned for 19 months.

lead_sponsor: Italfarmaco

location_country:
Italy
United States
Serbia
Netherlands
Germany
France
Israel
Belgium
Spain
Canada
United Kingdom

location_facility: Connecticut Children's Medical Center - Division Neurology

location_facility: Washington University School of Medicine in St Louis - Department of Neurology

location_facility: Holland Bloorview Kids Rehabilitation Hospital

location_facility: University of Minnesota - Department of Neurology

location_facility: Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN)

location_facility: Hospital Sant Joan de Déu - Neuromuscular Pathology Unit

location_facility: Rady Children's Hospital center - UCSD Department of Neuroscience

location_facility: A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari

location_facility: Hopital Armand Trousseau I-Motion, Plateforme d'essais cliniques pédiatriques

location_facility: Child Health Research Institute - Department of Pediatrics

location_facility: Klinik un Policlinik fur Kinder und Jugendmedizin - Universitatsklinikum Hamburg Eppendorf

location_facility: Universitatsklinikum Essen - Kinder und Jugendmedizin Neuropadiatrie

location_facility: Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta

location_facility: The John Walton Muscular Dystrophy Research Centre - Freeman Hospital - Newcastle University - Institute of Genetic Medicine

location_facility: Klinikum der Uniersitat Munchen - Campus Innenstadt

location_facility: Hospital Universitari i Politécnic de la Fe - Servicio Neurologia

location_facility: Centro Clinico NeMO Fondazione Serena ONLUS Area SUD

location_facility: Kinsmen Research Centre - Alberta Children's Hospital - Alberta Health Services

location_facility: Virginia Commonwealth University Childrens Hospital of Richmond at Virginia Commonwealth University

location_facility: University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology

location_facility: CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest

location_facility: Ospedale Pediatrico Bambin Gesù, Malattie Neuromuscolari e Neurodegenerative

location_facility: The University of British Columbia, Children's and Womens Health Centre of BC Branch

location_facility: Neuromuscular Research Center UC Davis Department of Physical Medicine and Rehabilitation

location_facility: UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD

location_facility: IRCCS Istituto G.Gaslini, U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica

location_facility: Hospital Materno-Infantil - Passeig de la Vall d'Hebron

location_facility: The Children's Hospital of Philadelphia Colket Translational Research Building

location_facility: Institute of Neurology - Schneider Children's Medical Center of Israel Kaplan, 14

location_facility: The Robert Jones and Agnes Hunt Orthopaedic Hospital - NHS Foundation Trust

location_facility: Clinic of Neurology and Psychiatry for Children and Youth - Neurology Department Dr. Subotic 6a,


title: An Open-label, Multicenter Phase 1 Trial to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Splicing Modulator H3B-8800 for Subjects With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia

summary: This is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antitumor activity of H3B-8800 in subset of participants with Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML), or Chronic Myelomonocytic Leukemia (CMML). The study consists of two parts, a dose escalation part (Part 1) exploring a multiple once daily (QD) schedules and an expansion part (Part 2) exploring a twice daily (BID) schedule.

lead_sponsor: H3 Biomedicine Inc.

location_country:
Germany
United States
France
Spain

location_facility: Hospital Universitario HM Sanchinarro - CIOCC

location_facility: H Lee Moffitt Cancer Center and Research Institute

location_facility: University of North Carolina Chapel Hill

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: Hospital Universitario Fundacion Jimenez Diaz