title: A Prospective and Retrospective Cohort Study to Refine and Expand the Knowledge on Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

summary:
Primary Objective:

To describe the clinical features and their severity at the time of diagnosis and their evolution over time in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD
To describe Clinician-Reported Outcomes (ClinROs) and Patient-Reported Outcomes (PROs) at enrollment and their evolution over time; disease severity at the time of diagnosis and its evolution over time

Secondary Objectives:

To describe abnormal values in laboratory parameters and all values of specific clinical and imaging assessments at the time of diagnosis and their evolution over time
To study the use and applicability towards validation of a newly developed ASMD disease severity scoring system
To study the use and applicability towards validation of a newly developed ASMD PRO tool
To describe ASMD-related disease burden among patients with ASMD, caregivers, and healthcare resource utilization
To describe the association between patient demographics (eg, age, gender, race, Ashkenazi ancestry) and genotype with selected clinical features in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD

primary_purpose: Other

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://vivli.org

lead_sponsor: Sanofi

location_country:
Germany
Italy
Portugal
Argentina
Romania
Brazil
Belgium
United States
Spain
Turkey
Chile
Czechia
France

location_facility:
Investigational Site Number :7920001
Investigational Site Number :7920003
Investigational Site Number :8400002
Investigational Site Number :380001
Investigational Site Number :6200002
Investigational Site Number :2030001
Investigational Site Number :152002
Investigational Site Number :7240001
Investigational Site Number :0560001
Investigational Site Number :8400003
Investigational Site Number :8400001
Investigational Site Number :0760006
Investigational Site Number :0320002
Investigational Site Number :0320001
Investigational Site Number :2500002
Investigational Site Number :6420001
Investigational Site Number :2500001
Investigational Site Number :380002
Investigational Site Number :0760001
Investigational Site Number :7920005
Investigational Site Number :7240005
Investigational Site Number :2760005
Investigational Site Number :2760002
Investigational Site Number :6200001
Investigational Site Number :152001
Investigational Site Number :2500003
Investigational Site Number :7240004
Investigational Site Number :0760002

intervention_type: Procedure


type: EudraCT Number

type: Registry Identifier

title: A First-in-Human, Open-label, Dose-escalation Trial With Expansion Cohorts to Evaluate Safety and Anti-tumor Activity of GEN1042 in Subjects With Malignant Solid Tumors

summary: To evaluate the safety and anti-tumor activity of GEN1042 in patients with metastatic or locally advanced solid tumors.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Interventional

design_allocation: Non-Randomized

primary_purpose: Treatment

enrollment_type: Anticipated

ReferenceType: derived

ReferenceCitation: Muik A, Adams 3rd HC, Gieseke F, Altintas I, Schoedel KB, Blum JM, Sanger B, Burm SM, Stanganello E, Verzijl D, Spires VM, Vascotto F, Toker A, Quinkhardt J, Fereshteh M, Diken M, Satijn DPE, Kreiter S, Ahmadi T, Breij ECW, Tureci O, Sasser K, Sahin U, Jure-Kunkel M. DuoBody-CD40x4-1BB induces dendritic-cell maturation and enhances T-cell activation through conditional CD40 and 4-1BB agonist activity. J Immunother Cancer. 2022 Jun;10(6):e004322. doi: 10.1136/jitc-2021-004322. Erratum In: J Immunother Cancer. 2022 Sep;10(9):

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by-nc

license_normalized: cc-by-nc

version: publishedVersion

evidence: oa repository (via pmcid lookup)

host_type: repository

license_normalized: no license

repository_normalized: PubMed Central

repositories_concat: PubMed Central

unpaywall_oa_status: gold

publisher_dissemination: British Medical Journal

ipd_sharing: No

lead_sponsor: Genmab

sponsor_collaborators:
Genmab
BioNTech SE

location_country:
Germany
Italy
Israel
United Kingdom
Denmark
United States
Korea, Republic of
Spain
Taiwan
Moldova, Republic of
Georgia
France

location_facility: Azienda Ospedaliera S.Croce e Carle Cuneo

location_facility: Severance Hospital, Yonsei University Health System

location_facility: University of Maryland Greenebaum Comprehensive Cancer Center

location_facility: Novant Health Cancer Institute - Forsyth (Medical Oncology)

location_facility: Klinikum der Stadt Ludwigshafen am Rhein gGmbH

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: Chang Gung Memorial Hospital Linkou Branch

location_facility: Azienda Ospedaliera Spedali Civili di Brescia

location_facility: Hospital General Universitario Gregorio Maran

location_facility: Nationales Centrum fr Tumorerkrankungen NCT

location_facility: Centre hospitalier Universitaire de Bordeaux

location_facility: ARENSIA Research Clinic at the Research Institute of Clinical Medicine

location_facility: Chang Gung Memorial Hospital (CGMH) - Kaohsiung Branch

location_facility: Pusan National University Yangsan Hospital

location_facility: Hospital Universitario Insular de Gran Canaria

location_facility: Central Washington Health Services Association

location_facility: ARENSIA Research Clinic at the Oncology Institute

location_facility: Complejo Hospitalario Universitario de Santiago (CHUS)

location_facility: HM CIOCC Hospital Universitario HM Sanchinarro

location_facility: Kaohsiung Medical University Memorial Hospital

location_facility: Moores Cancer Center at the UC San Diego Health

location_facility: Kaiser Permanente (KP) Oncology/Hematology

location_facility: Washington University School of Medicine

location_facility: University Hospital of Southern Denmark, Vejle Hospital

location_facility: Rigshospitalet (Copenhagen University Hospital)

location_facility: START Madrid - Hospital Universitario Fundacion Jimenez Diaz

location_facility: Hospital Duran i Reynals - ICO L Hospitalet

location_facility: Taipei Veterans General Hospital, VGHTPE

location_facility: Department of Dermatology, University of Mainz

location_facility: Hospital Clinico Universitario de Valencia

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Ongoing


acronym: WAVE

lead_sponsor: AstraZeneca

summary: The aims of the study are to monitor the long-term safety of durvalumab, to provide continued treatment or retreatment with durvalumab to eligible patients, and to collect overall survival (OS) information.

sponsor_collaborators:
AstraZeneca
Iqvia Pty Ltd
Parexel
Medidata Solutions
CISCRP

status: Active, not recruiting

title: An Open-Label, Multi-Center, Global Study to Evaluate Long Term Safety and Efficacy in Patients Who Are Receiving or Who Previously Received Durvalumab in Other Protocols (WAVE)

source: euctr

enrollment_type: Actual

ipd_sharing_description: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All requests will be evaluated as per the AZ disclosure commitment.

location_country:
Germany
Poland
Argentina
Australia
Brazil
Japan
Thailand
Vietnam
Turkey
Chile
Czechia
Canada
Serbia
France
Hungary
Switzerland
United States
Ukraine
India
Romania
Belgium
Malaysia
United Kingdom
Netherlands
Korea, Republic of
Spain
Greece
Taiwan
Bulgaria
Russian Federation
Israel

ipd_sharing: Yes


title: A Phase 2 Single Arm Open-Label Clinical Trial of ADP-A2M4 SPEAR™ T Cells in Subjects With Advanced Synovial Sarcoma or Myxoid/Round Cell Liposarcoma

summary: This is a study to investigate the efficacy and safety of ADP-A2M4 in HLA-A*02 eligible and MAGE-A4 positive subjects with metastatic or inoperable (advanced) Synovial Sarcoma (Cohort 1, 2 and 3 ) or MRCLS (Cohort 1) .

ReferenceCitation: Sanderson JP, Crowley DJ, Wiedermann GE, Quinn LL, Crossland KL, Tunbridge HM, Cornforth TV, Barnes CS, Ahmed T, Howe K, Saini M, Abbott RJ, Anderson VE, Tavano B, Maroto M, Gerry AB. Preclinical evaluation of an affinity-enhanced MAGE-A4-specific T-cell receptor for adoptive T-cell therapy. Oncoimmunology. 2019 Nov 24;9(1):1682381. doi: 10.1080/2162402X.2019.1682381. eCollection 2020.

evidence: open (via free pdf)

evidence: oa repository (via OAI-PMH doi match)

repository_institution: PubMed Central - Europe PMC

publisher_dissemination: Informa

lead_sponsor: Adaptimmune

location_country:
United Kingdom
United States
Spain
Canada
France

location_facility: Northwestern University Robert H. Lurie Comprehensive Cancer Center

location_facility: Medical College of WI Froedtert Hospital

location_facility: Start Madrid-FJD, Fundación Jimѐnez Díaz

intervention_type: Genetic


lead_sponsor: Zambon SpA

summary: The objective of the trial is to assess the long-term safety and efficacy of L-CsA plus Standard of Care (SoC) in the treatment of BOS in single (SLT) and double lung transplant (DLT) recipients.

location_facility: University of Florida Dept of Pulmonary Medicine

location_facility: Royal Papworth Hospital NHS Foundation Trust

location_facility: Unidad de Trasplante Pulmonar del Hospital La Fe

location_facility: Hospital Universitario Puerta de Hierro - Unidad de Trasplante Pulmonar

location_facility: University of Texas Southwestern Medical Center

status: Enrolling by invitation

title: A Phase III, Extension Clinical Trial to Demonstrate Efficacy and Safety of Liposomal Cyclosprine A Via the PARI Investigational eFlow® Device and SoC in Treating Bronchiolitis Obliterans in Patients Post Single or Double Lung Transplant

location_country:
Germany
Israel
United Kingdom
Denmark
Austria
Belgium
United States
Spain
France

status_simplified: Unknown


title: Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial

summary: This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Savarirayan R, De Bergua JM, Arundel P, McDevitt H, Cormier-Daire V, Saraff V, Skae M, Delgado B, Leiva-Gea A, Santos-Simarro F, Salles JP, Nicolino M, Rossi M, Kannu P, Bober MB, Phillips J 3rd, Saal H, Harmatz P, Burren C, Gotway G, Cho T, Muslimova E, Weng R, Rogoff D, Hoover-Fong J, Irving M. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. Ther Adv Musculoskelet Dis. 2022 Mar 21;14:1759720X221084848. doi: 10.1177/1759720X221084848. eCollection 2022.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Hospices Civils de Lyon

repository_normalized: HAL

repository_institution: University of Bristol - Bristol Research

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - UJM - Université Jean Monnet

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

repository_institution: California Digital Library - eScholarship

publisher_dissemination: SAGE Publications

lead_sponsor: QED Therapeutics, Inc.

location_country:
Germany
Italy
Norway
Argentina
Singapore
Australia
United Kingdom
United States
Spain
Canada
France

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: Manchester University Children's Hospital

location_facility: Children's Hospital - London Health Sciences Center

location_facility: Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan

location_facility: Nemours Alfred I. Dupont Hospital for Children

location_facility: University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

location_facility: Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS

location_facility: Otto-von-Guericke-University Magdeburg Medical Fakulty


title: A Phase 1/2 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Allogeneic CRISPR-Cas9-Engineered T Cells (CTX110) in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON)

summary: This is an open-label, multicenter, Phase 1/2 study evaluating the safety and efficacy of CTX110 in subjects with relapsed or refractory B-cell malignancies.

lead_sponsor: CRISPR Therapeutics AG

location_country:
Germany
Australia
United States
Spain
Canada
France

location_facility: Weill Cornell Medical College / New York Presbyterian Hospital

location_facility: Harold C. Simmons Comprehensive Cancer Center, UT Southwestern Medical Center

location_facility: Markey Cancer Center, University of Kentucky

location_facility: Virginia Commonwealth University Massey Cancer Center

location_facility: Emory University Winship Cancer Institute

intervention_type: Biological


lead_sponsor: Boehringer Ingelheim

summary:
The primary objective is to provide dose-ranging data for 4 dose regimens of BI 655130 compared to placebo on the primary endpoint of percentage change from baseline in PPP ASI at Week 16. The target dose(s) will be estimated from the model by incorporating information on the minimum clinically relevant effect and accounting for safety.

Supportive dose-ranging assessments will also be done on pre-specified secondary endpoints.

location_facility: Markusovszky University Teaching Hospital

location_facility: Shirasaki Dermatology and Neurology Clinic

location_facility: Universitätsklinikum Schleswig-Holstein, Campus Kiel

location_facility: University Hospital Kyoto Prefectural University of Medicine

location_facility: CRU Hungary Ltd, Private Practice, Miskolc

location_facility: The Psoriasis Treatment Center of Central New Jersey

location_facility: The Guenther Dermatology Research Centre

location_facility: Veszprem County Csolnoky Ferenc Hospital

location_facility: Washington University School of Medicine

location_facility: Seoul National University Bundang Hospital

location_facility: Tokyo Dental College Ichikawa General Hospital

location_facility: Dermatovenereological Dispensary #10, St. Petersburg

location_facility: SimcoDerm Medical and Surgical Dermatology Centre

location_facility: Total Skin and Beauty Dermatology Center, PC

location_facility: Shiga University of Medical Science Hospital

location_facility: York Dermatology Clinic and Research Centre

status: Completed

title: Multi-center, Double-blind, Randomised, Placebo-controlled, Phase IIb Dose-finding Study to Evaluate Safety and Efficacy of Different Subcutaneous Doses of BI 655130 in Patients With Moderate to Severe Palmoplantar Pustulosis (PPP)

ipd_sharing_description:
After the study is completed and the primary manuscript is accepted for publishing, researchers can use this following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement". Also, Researchers can use the following link https://www.mystudywindow.com/msw/datasharing to find information in order to request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.

The data shared are the raw clinical study data sets.

design_allocation: Randomized

study_completion_date_type: Actual

location_country:
Germany
Poland
Hungary
United Kingdom
Australia
Japan
Netherlands
Belgium
United States
Korea, Republic of
Taiwan
Czechia
Canada
Russian Federation
France

status_simplified: Completed


title: APOLLO-B: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

summary: The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.

lead_sponsor: Alnylam Pharmaceuticals

location_country:
New Zealand
Argentina
Australia
Brazil
Japan
Chile
Czechia
France
Portugal
Hong Kong
Denmark
United States
Belgium
Italy
United Kingdom
Netherlands
Mexico
Korea, Republic of
Taiwan
Bulgaria
Sweden


acronym: A treatment study in patients with WHIM Syndrome.

lead_sponsor: X4 Pharmaceuticals

summary: This study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period. The primary objective of the Randomized Placebo-Controlled Period is to demonstrate the efficacy of mavorixafor in participants with WHIM syndrome as assessed by increasing levels of circulating neutrophils compared with placebo, and relative to a clinically meaningful threshold. The primary objective of the Open-Label Period is to evaluate the safety and tolerability of mavorixafor in participants with WHIM syndrome. Participants are allowed to continue treatment in the Open-Label Period, if regionally applicable, until mavorixafor becomes commercially available, or until the study is terminated by the Sponsor.

location_facility: Seoul National University Hospital, Children's Hospital

location_facility: Emma Children's Hospital Academic Medical Center (AMC)

location_facility: Università degli Studi di Brescia, Scienze Cliniche e Sperimentali

location_facility: Academician I.P. Pavlov First Saint Petersburg State Medical University

location_facility: University of Debrecen, Affiliated Department of Infectology

location_facility: Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology

location_facility: University of California San Diego Health/Rady Children's Hospital

location_facility: CHU de Lyon, Institut d'Hematologie et d'Oncologie Pediatrique

location_facility: CHU Paris Est, Hôpital d'Enfants Armand-Trousseau

location_facility: University of Texas Southwestern Medical Center

location_facility: Medical University of Vienna - Medizinische Universität Wien

title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension

type: Other Identifier

location_country:
Italy
Hungary
Israel
Denmark
Australia
Netherlands
Austria
United States
Korea, Republic of
Spain
Turkey
Russian Federation
France


title: A Phase 3 Multicenter, Randomized, Double-blind, Placebo-controlled, Clinical Study to Assess the Efficacy and Safety of Linzagolix in Subjects With Moderate to Severe Endometriosis-associated Pain.

summary: The primary objective of this study is to demonstrate the efficacy and safety of linzagolix administered orally once daily for 3 months at a dose of 75 mg alone or of 200 mg in combination with add-back hormone replacement therapy (ABT: estradiol (E2) 1 mg / norethisterone acetate (NETA) 0.5 mg) versus placebo, in the management of moderate to severe endometriosis-associated pain (EAP).

lead_sponsor: Kissei Pharmaceutical Co., Ltd.

location_country:
Poland
Hungary
Romania
Austria
United States
Spain
Czechia
Bulgaria
Ukraine
France

location_facility: UWCR - Lyndhurst Clinical Researc/ ID# 605

location_facility: Hospital General Universitario Reina Sofia/ ID # 304

location_facility: Next Innovative Clinical Research/ ID # 617

location_facility: AKH Vienna University of Medicine Vienna / ID # 101

location_facility: Clinical Medical Research sp. Z o. O/ ID # 406

location_facility: Ternopil' Communal City Hospital #2/ ID # 511

location_facility: Kyiv City Clinical Hospital #9/ ID # 501

location_facility: Centrum Ginekologii Endokrynologii i Medycyny Rozrodu "Artemida"/ ID # 409

location_facility: CHRU Strasbourg - Hopital Hautepierre/ ID # 205

location_facility: Hospital Regional Universitario de Málaga/ ID # 303

location_facility: Centrum Medyczne Chodźki Lublin/ ID # 403

location_facility: Multiprofile Hospital for Active Treatment St. Sofia/ ID # 137

location_facility: Multiprofile Hospital for Active Treatment Sliven/ ID # 131

location_facility: AKH Vienna University of Medicine Vienna/ ID # 103

location_facility: Lubelskie Centrum Diagnostyczne/ ID # 402

location_facility: Prywatna Klinika Ginekologiczno - Położnicza/ ID # 404

location_facility: Niepubliczny Zaklad Opieki Zrowotnej GynCentrum/ ID # 407

location_facility: City clinical maternity hospital №1/ ID # 502

location_facility: Universal Axon - Homestead, LLC/ ID # 620

location_facility: Obstetrics & Gynecology Associates, Inc./ ID# 606

location_facility: The Center for Women's Health & Wellnes/ ID # 603

location_facility: University Multiprofile Hospital for Active Treatment "Sofiamed"/ ID # 133

location_facility: Group Practice for Specialized Medical Assistance in GINART/ ID # 132

location_facility: Kyiv City Maternity Hospital #6/ ID # 504

location_facility: Szent Anna Szuleszeti-Nogyogyaszati/ ID # 251

location_facility: Kotarski Jan Specjalistyczny Gabinet Ginekologiczno Polozniczy/ ID # 405

location_facility: Affinity Clinical Research Institute/ ID # 622

location_facility: Wojewodzki Szpital im. Sw. Ojca Pio w Przemyslu/ ID # 410

location_facility: Gynekologicko - Porodnicka Ambulance/ ID # 162

location_facility: Zaporizhzhya Regional Clinical Hospital/ ID # 505

location_facility: Specjalistyczny Gabinet Lekarski Krzysztof Dynowski / ID # 401

location_facility: Institute of Pediatrics Obstetrics and Gynecology/ ID # 508

location_facility: Gynekologie MU Dr.Lubomir Mikulasek / ID # 160

location_facility: Ivano-Frankivsk regional perinatal center, Family planning center/ ID # 506

location_facility: Spitalul Clinic "Nicolae Malaxa"/ ID # 352

location_facility: Chattanooga Medical Research LLC/ ID# 602

location_facility: Indywidualna Specjalistyczna Praktyka Lekarska Krzysztof Wilk/ ID # 411

location_facility: Medical center of LLC "Medical Center "Verum"/ ID # 503

location_facility: MUDr Jan Drahonovsky, Ustav pro peci o matku a dite/ ID #163

location_facility: Sp Cl de Obstetrica si Ginecologie "Prof. Dr. Panait Sirbu"/ ID # 354

location_facility: Invocare Medical Research & Healthcare Center/ ID # 607

location_facility: Kyiv City Center of Reproductive and Perinatal Medicine/ ID # 509

location_facility: Vinnytsia City Clinical Hospital "Center of Mother and the Child "/ ID # 510

location_facility: Multi-Specialty Research Associates, Inc./ ID # 625

location_facility: Women's & Family Care, LLC dba GTC Research/ ID # 608


title: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

summary: This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will be conducted for the selection of a high dose (100 mg/kg versus 200 mg/kg) and its comparison with the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.

lead_sponsor: Sarepta Therapeutics, Inc.

location_country:
Germany
Ireland
Poland
Taiwan
New Zealand
Turkey
Czechia
Serbia
France
Slovenia
Colombia
Hungary
Denmark
Switzerland
United States
India
Romania
Italy
Norway
United Kingdom
Netherlands
Mexico
Korea, Republic of
Spain
Greece
Jordan

location_facility: Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: New Zealand Clinical Research - Auckland

location_facility: The Specialty Hospital (TSH)/Advanced Clinical Center

location_facility: Rigshospitalet Copenhagen University Hospital

location_facility: Pusan National University Yangsan Hospital

location_facility: Jaicare Hospital (A Unit of Sarvee Integra Pvt Ltd.)

location_facility: Oslo Universitetssykehus HF Rikshospitalet

location_facility: Pharmaceutical Research Center/Jordan University of Science and Technology

location_facility: Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"

location_facility: Instituto de Investigaciones Clinicas para la Salud A.C

location_facility: Clinic for Neurology and Psychiatry for Children and Youth

location_facility: National Clinical Hospital for Children Neurorehabilitation "Dr. Nicolae Robănescu"

location_facility: Children's Department and Department for Children's Habilitation at Stavanger University Hospital

location_facility: Children's Health Ireland (CHI) at Temple Street

location_facility: UCL Institute of Child Health Great Ormond Street

location_facility: Kyungpook National University Chilgok Hospital

location_facility: Instituto Neurologico de Colombia (INDEC)

location_facility: Deenanath Mangeshkar Hospital & Research Centre

location_facility: Radboud University Nijmegen Medical Centre


title: Prospective, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Investigate the Efficacy and Safety of NT 201 in the Treatment of Lower Limb Spasticity Caused by Stroke or Traumatic Brain Injury in Adult Subjects, Followed by an Open Label Extension With or Without Combined Upper Limb Treatment

acronym: PATTERN

summary:
The purpose of this study is to determine whether a single treatment with administration of 400 Units NT 201 (botulinum toxin) is superior to placebo (no medicine) for the treatment of lower limb spasticity caused by stroke or traumatic brain injury (Main Period). Participants will be assigned to the treatment groups by chance and neither the participants nor the research staff who interact with them will know the allocation.

The following 4 to 5 treatment cycles will investigate the safety and tolerability of treatment with NT 201 (botulinum toxin) when administered in doses between 400 and 800 Units (Open Label Extension Period). All participants will receive the treatment and the dose will depend on whether only lower limb spasticity or combined upper and lower limb spasticity are treated.

lead_sponsor: Merz Pharmaceuticals GmbH

location_country:
Germany
Italy
Poland
Slovakia
Hungary
Norway
United Kingdom
Australia
Switzerland
Belgium
United States
Spain
Czechia
Ukraine
Russian Federation
France

location_facility: Hospital Universitario La Paz, Merz Investigational Site #0340007

location_facility: Universitätsklinikum Hamburg-Eppendorf, Merz Investigational site #0490079

location_facility: Filia 7 Centrum Kompleksowej Rehabilitacji Sp. z o.o., Merz Investigational Site #0480100

location_facility: Sunnaas Rehabilitation Hospital, Merz Investigational Site #0470002

location_facility: Federal State Autonomous Institution "National Medical Research Centre "Treatment and Rehabilitation" of the Ministry of Health of the Russian Federation, Neurology department, Merz Investigational Site # 0070305

location_facility: UCL Bruxelles, Physical Medicine & Rehabilitation Department, Merz Investigational Site #0320013

location_facility: Hospital Universitari Mútua de Terrassa, Merz Investigational Site #0340003

location_facility: Neuryt Diagnostyka I Terapia Neurologiczna, Merz investigational site #0480106

location_facility: Mazowiecki Szpital Brodnowski, Merz investigational site #0480064

location_facility: Fakultni nemocnice Kralovske Vinohrady, Neurology Department, Merz Investigational Site #4200050

location_facility: Heinrich Heine University Duesseldorf, Department of Neurology; Merz Investigational Site #0490071

location_facility: The Walton Centre NHS Foundation Trust; Neuroscience Research Centre; Merz Investigational site #0440004

location_facility: National Hospital for Neurology and Neurosurgery - UCLH; Merz Investigational Site #0440033

location_facility: Pardubice Regional Hospital; Merz investigational site #4200025

location_facility: Neurologie Bewegt; Merz Investigational site #0490378

location_facility: UZ Leuven Pellenberg campus; Merz investigational site #0320014

location_facility: Cambridge University Hospitals NHS Foundation Trust; Merz Investigational Site 0440023

location_facility: St George Hospital, Department of Rehabilitation Medicine, Merz Investigational Site #0610004

location_facility: Ospedale Alfredo Fiorini, UOSD Neurologia Universitaria; Merz Investigational site #0390022

location_facility: Specjalistyczna Praktyka Lekarska Dr. n. med Stanisław Ochudło, Merz Investigational Site #0480077

location_facility: Federal State Budgetary Institution "National Medical Research Centre of Psychiatry and Neurology n.a. V.M. Bekhterev" of the Ministry of Healthcare of the Russian Federation, Merz Investigational Site #0070009

location_facility: CBBM, Institut für Neurogenetik, Haus 66; Merz Investigational Site #0490332

location_facility: New England institute for clinical research; Merz Investigational Site #0010441

location_facility: University of Szeged, Department of Neurology, Merz investigational site #0360008

location_facility: NeuroKlinika Gabinet Lekarski Prof Andrzej Bogucki, Merz Investigational Site #0480101

location_facility: Indywidualna Praktyka Lekarska dr. hab. Med. Anna Szczepanska-Szerej, Merz Investigational Site #0480096

location_facility: Royal Melbourne Hospital, Department of Rehabilitation Medicine; Merz Investigational Site #0610002

location_facility: Haukeland University Hospital, Dept of Neurology; Merz investigational site #0470001

location_facility: University of Texas Health Science Center McGovern, Medical School Department of PM&R; merz investigational site #0010183

location_facility: University Hospital Bratislav, 2nd Dept. of Neurology, Merz Investigational Site # 4210005

location_facility: Neurologicka klinika, Fakultna nemocnica Trnava; Merz investigational site #4210009

location_facility: Specjalistyczne Gabinety Sp z o.o., Merz Investigational Site #0480059

location_facility: UT Southwestern Medical Center, Department of Physical Medicine & Rehabilitation, Merz investigational site #0010082

location_facility: IRCCS Ospedale Policlinico S. Martino, U.O.Complessa Riabilitazione e Rieducazione Funzionale; Merz investigational site #0390020

location_facility: AOUI -Ospedale Borgo Roma, U.O.C Neuroriabilitazione, Merz Investigational Site #0390014

location_facility: St.Damjan Hospital, Kisvarda; Merz investigational site #0360019

location_facility: DKD HELIOS Medical Center, Fachbereich Neurologie, Merz Investigational Site #0490081

location_facility: UCL Mont Godinne, Physical Medicine & Rehabilitation Department; Merz Investigational Site #0320008

location_facility: HFR Fribourg - Hôpital Cantonal, Department of Rehabilitation, Merz Investigational site #0410014

location_facility: Federal State Budgetary Educational Institution of Higher Education "Krasnoyarsk State Medical University n.a. Professor V.F. Voino-Yasenetskiy of Ministry Healthcare of Russian Federation", Merz Investigational Site #0070306

location_facility: Icahn School of Medicine at Mount Sinai, Neurology Department, Merz Investigational Site #0010191

location_facility: Ospedale Maggiore della Carita, Dipartimento di Medicina fisica e Riabilitativa, Merz Investigational Site #0390017

location_facility: Rivne City Hospital 2, Department of Clinical Neurology and Neurorehabilitation; Merz investigational site #3800016

location_facility: University of Pittsburgh Medical Center, Physical Medicine & Rehabiliation, Merz Investigational Site #0010211

location_facility: State Autonomous Institution of Healthcare of Moscow City "Moscow Scientific and Practical Centre of Medical Rehabilitation, Regenerative and Sports Medicine of Moscow City Healthcare Department", Merz Investigational Site #0070011

location_facility: Azienda Ospedaliera Universitaria Ospedali Riuniti Di Foggia; Merz Investigational Site #0390018

location_facility: Raymond Poincaré University Hospital, Service de MPR Pôle Handicap-Rééducation, Merz Investigational site #00330018

location_facility: NPE "Regional Clinical Centre of Neurosurgery and Neurology" Transcarpathian Regional Council; Merz investigational site #3800014

location_facility: Institut Universitaire de Readaptation Clemenceau, Physical Medicine / Rehabilitation Medecine, Merz Investigational site # 0330063

location_facility: Royal Devon and Exeter Foundation Trust Hospital, Department of Neurology, Merz Investigational Site #0440047

location_facility: Uniklinik RWTH Aachen, Neurologie, Merz Investigational Site #0490191

location_facility: Ospedale Valduce, Villa Beretta Rehabilitation Center, Merz investigational site #0390005

location_facility: Instytut Psychiatrii i Neurologii, II Klinika Neurologiczna, Merz Investigational Site #0480023

location_facility: Medical College of Wisconsin,Department of PM&R, Merz Investigational Site #0010237

location_facility: Howard A. Rusk Rehabilitation Center; Merz investigational site #0010283

location_facility: Projekt Samodzielni Sp z o.o., Merz Investigational Site #0480099

location_facility: Rangueil Hospital-University Hospital Center (CHU) of Toulouse, Médecine physique et de réadaptation, Merz investigational site #0330025

location_facility: Neurologische Klinik und Poliklinik des Universitätsklinikums Würzburg, Merz Investigational Site #0490302

location_facility: Centre Hospitalier Universitaire Vaudois - Neurorehabilitation and Physical Medicine, Merz Investigational Site #0410013

location_facility: University Hospital of Bern, Center of Parkinson's diseases and Movement disorders (ZfPB), Merz investigational site #0410015

location_facility: Neurology and Physiotherapy Skopalikova, Merz Investigational Site #4200049

location_facility: CHU Brugmann Neurology; Merz investigational site #0320015

location_facility: University Hospital Ostrava; Neurology Departement, Merz investigational site #4200024

location_facility: Weill Cornell Medical Center, Department of Rehabilitation Medicine; Merz Investigational Site #0010440

location_facility: Rehabilitation Hospital, Kessler Institute for Rehabilitation, Merz Investigational Site #0010199


acronym: PROMISE

lead_sponsor: Guerbet

summary: The trial aims at evaluating the efficacy and the safety of gadopiclenol for body Magnetic resonance Imaging (MRI)

location_facility: Istituto Nazionale Tumori IRCCS "Fondazione G. Pascale"

location_facility: Klinik für Diagnostische und Interventionelle Radiologie

location_facility: Acibadem City Clinic Tokuda Hospital Sofia

location_facility: Szpital Uniwersytecki Nr 1 im. dr Antoniego Jurasza w Bydgoszczy

location_facility: Jagiellońskie Centrum Innowacji Sp. z o. o.

location_facility: Axis Heilsa S de R.L. de C.V - Althian Clinical Research Management Center

location_facility: Hospital Universitario y Politécnico La Fe

location_facility: Jasz-Nagykun-Szolnok Megyei Hetenyi Geza Korhaz-Rendelointezet

location_facility: UConn Health - University of Connecticut

location_facility: The University of Chicago Medical Center

title: Efficacy and Safety of Gadopiclenol for Body Magnetic Resonance Imaging (MRI)

primary_purpose: Diagnostic

location_country:
Germany
Italy
Poland
Hungary
Mexico
United States
Korea, Republic of
Spain
Bulgaria
Ukraine
France


title: A Randomized, Double-Blind, Parallel, Phase 1 Study to Compare the Pharmacokinetics of BMSCHO1-Nivolumab Process D to Nivolumab Process C After Complete Resection of Stage IIIa/b/c/d or Stage IV Melanoma

summary: The purpose of this study is to compare the drug levels, immunogenicity and safety of Nivolumab Process D to Nivolumab Process C after complete resection of stage IIIa/b/c/d or stage IV melanoma.

lead_sponsor: Bristol-Myers Squibb

location_country:
Ireland
Italy
Poland
New Zealand
Argentina
Romania
Australia
Brazil
Mexico
United States
Spain
Chile
Canada
France

location_facility:
Local Institution - 0017
Local Institution - 0030
Local Institution - 0031
Local Institution - 0029
Local Institution - 0018
Local Institution - 0003
Local Institution - 0011
Local Institution - 0009
Local Institution - 0041
Local Institution - 0035
Local Institution - 0015
Local Institution - 0027
Local Institution - 0040
Local Institution - 0032
Local Institution - 0039
Local Institution - 0024
Local Institution - 0038
Local Institution - 0028
Local Institution - 0005
Local Institution - 0014
Local Institution - 0034
Local Institution - 0043
Local Institution - 0037
Local Institution - 0016
Local Institution - 0023
Local Institution - 0001
Local Institution - 0022
Local Institution - 0008
Local Institution - 0033
Local Institution - 0013
Local Institution - 0045
Local Institution - 0004
Local Institution - 0026
Local Institution - 0010
Local Institution - 0006
Local Institution - 0046
Local Institution - 0036


title: A Randomized, Double Blind, Parallel-group, Placebo Controlled Study to Evaluate the Efficacy and Safety of IBP-9414 in Premature Infants 500-1500g Birth Weight in the Prevention of Necrotizing Enterocolitis - The Connection Study

summary: IBP-9414 will be evaluated in preterm infants with a birth weight of 500-1500g, compared to placebo with regards to efficacy and safety in the prevention of necrotizing enterocolitis.

primary_purpose: Prevention

lead_sponsor: Infant Bacterial Therapeutics

location_country:
France
Poland
Hungary
United Kingdom
Romania
United States
Spain
Bulgaria
Serbia
Israel

location_facility: Banner University Medical Center / University of Arizona

location_facility: Szent György University Teaching Hospital of County Fejér

location_facility: Northwell Health Cohen Children'S Medical Center of Ny

location_facility: Spitalul Clinic de Urgenta pentru Copii "Louis Turcanu" Timisoara

location_facility: Uniwersyteckie Centrum Kliniczne, Warszawskiego Uniwersytetu Medycznego

location_facility: Helen DeVos Children's Hospital Spectrum Health Research

location_facility: University of Miami/Holtz Children'S Hospital

location_facility: Spitalul Clinic de Obstetrica-Ginecologie "Cuza-Voda" Iasi

location_facility: Szszb County Hospital and Jósa András University Teaching Hospital

location_facility: Medical University of South Carolina (MUSC)

location_facility: Harvard Medical School - Beth Israel Deaconess Medical Center (Bidmc)

location_facility: Centre Hospitalier Universitaire de Caen Normandie

location_facility: Connecticut Children'S Medical Center - Uconn - School of Medicine

location_facility: Children's Clinic of the Clinical Center of Kragujevac

location_facility: SIU School of Medicine - HSHS St. John's Children's Hospital

location_facility: University of Texas Medical Branch - Ocr

location_facility: Specialized Hospital in Active Treatment of Pediatric Diseases

location_facility: University Multiprofile Hospital for Active Treatment "Dr. Georgi Stranski" - Clinic of Neonatology

location_facility: University of Tennessee Health Science Center

location_facility: Sheridan Healthcare of Texas, P.A./Medical City Plano

location_facility: Associates in Newborn Medicine - Saint Paul

location_facility: Hospital Universitario Hm Puerta Del Sur

location_facility: Spitalul Clinic Municipal de Urgenta Timisoara

location_facility: St David'S Health Care - St. David'S Medical Center/North Austin Medical Center

location_facility: Borsod- Abaúj-Zemplén County Central Hospital and University Teaching Hospital

location_facility: Veszprém County Csolnoky Ferenc Hospital

location_facility: Spitalul Clinic de Obstetrica si Ginecologie "Filantropia"

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 2 Pomorski Uniwersytet Medyczny (PUM) w Szczecinie /Pomeranian Medical University

location_facility: Nottingham University Hospitals, Neonatology: City Campus

location_facility: Maria Fareri Children's Hospital - Westchester Medical Center

location_facility: Spitalul Clinic Judetean de Urgenta Cluj-Napoca

location_facility: University Hospital of Obstetrics and Gynecology Maichin Dom - Neonatology Clinic

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 1 Pomorskiego Uniwersytetu Medycznego im. prof. Tadeusza Sokołowskiego

location_facility: Children'S Hospital of the University of Illinois

location_facility: University of Califorina, Los Angeles (UCLA)

location_facility: University of Arkansas For Medical Services

location_facility: Texas Health Presbyterian Hospital - Plano

location_facility: Acibadem City Clinic Tokuda Hospital (Tokushukai Medical Corporation - Tokuda Hospital Sofia) (THS)

location_facility: University of Szeged, Albert Szent-Györgyi Health Centre

location_facility: University of Mississippi Medical Center

location_facility: Sheridan Clinical Research / Plantation General Hospital

location_facility: Nottingham University Hospitals NHS Trust


lead_sponsor: PDC*line Pharma SAS

summary: PDC-LUNG-101 trial is an open-label, dose-escalation, phase I/II study to assess the safety, the tolerability, the immunogenicity and the preliminary clinical activity of the therapeutic cancer vaccine, PDC*lung01, associated or not with anti-PD-1 treatment in patients with non-small-cell lung cancer.

location_facility: Centre Léon Bérard, Centre de lutte contre le cancer

location_facility: Antoni Van Leeuwenhoek (Nederlands Kanker Instituut)

location_facility: Jeroen Bosch Ziekenhuis - 's hertogenbosch

title: An Open-label, Dose-escalation, Phase I/II Study to Assess the Safety, the Tolerability, the Immunogenicity and the Preliminary Clinical Activity of the Therapeutic Cancer Vaccine, PDC*lung01, Associated or Not With Anti-PD-1 Treatment in Patients With Non-small-cell Lung Cancer (NSCLC)

location_country: Germany Poland Netherlands Belgium France


title: GBM AGILE: Global Adaptive Trial Master Protocol: An International, Seamless Phase II/III Response Adaptive Randomization Platform Trial Designed To Evaluate Multiple Regimens In Newly Diagnosed and Recurrent GBM

acronym: GBM AGILE

summary: Glioblastoma (GBM) adaptive, global, innovative learning environment (GBM AGILE) is an international, seamless Phase II/III response adaptive randomization platform trial designed to evaluate multiple therapies in newly diagnosed (ND) and recurrent GBM.

ipd_sharing: Undecided

ipd_sharing_description: Global Coalition for Adaptive Research (GCAR) is in the planning stages at this time.

lead_sponsor: Global Coalition for Adaptive Research

sponsor_collaborators:
Global Coalition for Adaptive Research
Bayer
Kazia Therapeutics Limited
Kintara Therapeutics, Inc.
Biohaven Pharmaceuticals, Inc.
Vigeo Therapeutics, Inc.
Polaris Group

location_country:
Germany
Australia
Switzerland
United States
Canada
France

location_facility: Northern Sydney Cancer Centre/Royal North Shore Hospital

location_facility: Comprehensive Cancer Center of Wake Forest

location_facility: Washington University School of Medicine - Siteman Cancer Center

location_facility: University Hospitals Cleveland Medical Center

location_facility: Cedars Sinai - Samuel Oschin Comprehensive Cancer Institute

location_facility: Froedtert Hospital/Medical College of Wisconsin

location_facility: Centre Hospitalier Universitaire Vaudois Lausanne

location_facility: University of Pittsburgh Medical Center - Hillman Cancer Center

location_facility: Montreal Neurological Institute and Hospital, McGill University

location_facility: University of Utah - Huntsman Cancer Institute

location_facility: Centre Hospitalier Lyon Sud / Hôpital Neurologique P. Wertheimer

location_facility: Yale Cancer Center / Smilow Cancer Hospital

location_facility: LSU Health Sciences Center - New Orleans

location_facility: Perlmutter Cancer Center, NYU Langone Health

location_facility: University of Texas - MD Anderson Cancer Center

location_facility: Medical University of South Carolina - Hollings Cancer Center

location_facility: Winship Cancer Institute of Emory University

location_facility: University of Texas Southwestern Medical Center

location_facility: Dr. Senckenbergisches Institut für Neuroonkologie

location_facility: University of Pennsylvania - Perelman Center for Advanced Medicine


acronym: Open-label Active-control Pediatric Safety, Efficacy, PK Study of MK-7655A

lead_sponsor: Merck Sharp & Dohme LLC

summary: The primary purpose of this study is to evaluate the safety and tolerability of imipenem/cilastatin/relebactam (IMI/REL) in participants from birth to less than 18 years of age with confirmed or suspected gram-negative bacterial infection. Participants are expected to require hospitalization through completion of intravenous (IV) study intervention, and have at least one of the following primary infection types: hospital-acquired bacterial pneumonia (HABP) or ventilator-associated bacterial pneumonia (VABP); complicated intra-abdominal infection (cIAI); or complicated urinary tract infection (cUTI). Participants will be randomized in a 3:1 ratio to receive IMI/REL or active control. This study will also evaluate the efficacy of IMI/REL by assessing all-cause mortality at Day 28 post-randomization, as well as clinical and microbiological response to treatment. It will also evaluate the pharmacokinetics of IMI/REL.

location_facility: Instytut Centrum Zdrowia Matki Polki ( Site 0223)

location_facility: Wojewodzki Szpital Zespolony im. Rydgiera ( Site 0220)

location_facility: Empilweni Services and Research Unit ( Site 1557)

location_facility: Haukeland Universitetssjukehus ( Site 0500)

location_facility: SPZOZ im. Dzieci Warszawy w Dziekanowie Lesnym ( Site 0226)

location_facility: Pediatric Hematology Oncology and Immunology Centre n.a. D.Rogachev. ( Site 0233)

location_facility: State Budgetary Healthcare Institution of Novosibirsk Region City Childrens Clinical Emergency Hospi

location_facility: Cukurova University Medical Faculty ( Site 0200)

location_facility: Ivano-Frankivsk Regional Children Clinical Hospital ( Site 0131)

location_facility: Hospital Infantil Universitario Nino Jesus ( Site 0114)

location_facility: University of the Philippines-Philippine General Hospital ( Site 0318)

location_facility: Fundacion Hospital Infantil Universitario de San Jose ( Site 0268)

location_facility: Centenario Hospital Miguel Hidalgo-Pediatrics Department ( Site 0290)

location_facility: Ankara Universitesi Tip Fakultesi. ( Site 0202)

location_facility: SI Dnipropetrovsk Regional Children Clinical Hospital DOR ( Site 0121)

location_facility: Children's Hospital of Richmond at VCU ( Site 0359)

location_facility: St.Petersburg State Pediatric Medical University ( Site 0236)

location_facility: Chris Hani Baragwanath Academic Hospital ( Site 0156)

location_facility: Hospital Universitario La Paz ( Site 0113)

location_facility: Communal non-commercial enterprise "Kryvorizka city clinical hospital 16" of Kryvyy Rig city council

location_facility: Philippine Children s Medical Center ( Site 0317)

location_facility: UMHAT Dr. Georgi Stranski EAD ( Site 0174)

location_facility: Rady Children's Hospital-San Diego ( Site 0347)

location_facility: Children's City Clinical Hospital #1 ( Site 0237)

location_facility: Tufts Medical Center-Floating Hospital for Children ( Site 0350)

location_facility: Hospital del Nino y Adolescente Morelense ( Site 0286)

location_facility: University of Athens - Aghia Sophia Childrens Hospital ( Site 0243)

location_facility: Fundacion Hospital San Vicente de Paul ( Site 0269)

location_facility: Hippokration General Hospital of Thessaloniki ( Site 0244)

location_facility: Hadassah Ein Karem Hebrew University Medical Center ( Site 0188)

location_facility: Regional Childrens Clinical Hospital ( Site 0400)

location_facility: Municipal Enterprise Children's City Clinical Hospital in Poltava City Council ( Site 0122)

location_facility: Hospital Universitario Virgen del Rocio ( Site 0115)

location_facility: Pan and Aglaia Kyriakou Children s Hospital ( Site 0247)

location_facility: Debreceni Egyetem Klinikai Kozpont ( Site 0100)

location_facility: Szabolcs-Szatmar-Bereg Megyei Kórházak és Egyetemi Otatókórház-Gyermekosztály ( Site 0105)

location_facility: Schneider Children's Medical Center ( Site 0187)

location_facility: Miller Children's & Women's Hospital ( Site 0349)

location_facility: Hospital General de Tijuana ( Site 0284)

location_facility: Instituto Nacional de Pediatria ( Site 0291)

location_facility: Sociedad de Cirugía de Bogotá - Hospital de San Jose ( Site 0265)

location_facility: MHAT City Clinic Sv. Georgi EOOD ( Site 0167)

location_facility: Smolensk Regional Clinical Hospital ( Site 0231)

location_facility: Children s City Clinical Hospital 5 n.a. N.F. Filatov ( Site 0235)

location_facility: Eskisehir Osmangazi University Medical ( Site 0201)

location_facility: SBU Sariyer Hamidiye Etfal Egitim ve Arastirma Hastanesi ( Site 0198)

location_facility: Kharkiv City Children Hospital 16 ( Site 0130)

location_facility: West Virginia University Ruby Memorial Hospital ( Site 0344)

location_facility: Ege Universitesi Tıp Fakultesi Hastanesi ( Site 0199)

location_facility: Hopitaux Pediatriques CHU Lenval ( Site 0143)

location_facility: Morozovskaya Children City Clinical Hospital ( Site 0241)

location_facility: Banner University Medical Center ( Site 0356)

title: A Phase 2/3 Open-label, Randomized, Active-controlled Clinical Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of MK-7655A in Pediatric Participants From Birth to Less Than 18 Years of Age With Confirmed or Suspected Gram-negative Bacterial Infection

location_country:
Poland
Colombia
Russian Federation
Hungary
Israel
Norway
Mexico
United States
Spain
Greece
South Africa
Chile
Turkey
Ukraine
Bulgaria
Estonia
Philippines
France


acronym: Centerstone

lead_sponsor: Hoffmann-La Roche

summary: Otherwise healthy index patients (IP) are randomized to either baloxavir marboxil or placebo if their influenza symptoms onset was within 48 hours of screening. Their households are enrolled within 24 hours of randomization if at least 1 household contacts (HHC) have not received influenza vaccine within 6 months of screening and if all HHC screen negative for influenza infection. The main endpoints are assessed based on multiple respiratory swabs, obtained from both IP and HHC up to 9 (+/-1) days post IP randomization, and through the assessment of symptoms.

ReferenceCitation: Komeda T, Takazono T, Hosogaya N, Ogura E, Fujiwara M, Miyauchi H, Ajisawa Y, Iwata S, Watanabe H, Honda K, Kitanishi Y, Hara K, Mukae H. Comparison of Household Transmission of Influenza Virus From Index Patients Treated With Baloxavir Marboxil or Neuraminidase Inhibitors: A Health Insurance Claims Database Study. Clin Infect Dis. 2021 Jun 1;72(11):e859-e867. doi: 10.1093/cid/ciaa1622.

oa_host_type: publisher

evidence: open (via crossref license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repositories_concat: closed

unpaywall_oa_status: hybrid

publisher_dissemination: Oxford University Press

location_facility: Huashan Hospital Affiliated to Fudan University

location_facility: MEDICAL CENTER HERA EOOD; Office of Pulmonology and Phtisiatrics

location_facility: Sawada Internal Medicine / Respiratory Clinic

location_facility: The Village Practice; Thornton Medical Centre

location_facility: Hatakeyama Internal Medicine and Gastroenterology Clinic

location_facility: Centro de Investigacion Clínica GRAMEL S.C

location_facility: Premier Pulmonary Critical Care and Sleep Medicine

location_facility: MHAT Sveta Paraskeva; Office of Cardiology Diseases

location_facility: Maccabi health services - Moked Hashalom; Maccabi health services - Moked Ramat Hasharon

location_facility: ICIMED Instituto de Investigación en Ciencias Médicas

location_facility: Gushiken-Cardiology and Internal medicine

location_facility: Queen Mary Hospital; Uni Dept of Medicine, Division of Gastroenterology

location_facility: Medical Centre "Asklepii", OOD; Office of Pulmonology and Phtisiatrics

location_facility: Urgent Care Clinical Trials at Complete Urgent Care

location_facility: Hometown Urgent Care and Research - Dayton

location_facility: The First Affiliated Hospital of Guangzhou Medical University

location_facility: National Healthcare Group Polyclinics- Yishun

location_facility: Hometown Urgent Care and Occupational Health - Springdale

location_facility: Olive Branch Family Medical Center; Family Medicine

location_facility: Children's hospital of Nanjing medical university

location_facility: Sir J J Group of Hospitals; Department of Pulmonary Medicine

location_facility: Limpopo Clinical Research Initiative; Tamboti Medical Centre

location_facility: Centro de Cancer Pontificie Universidad Catolica de Chile

location_facility: CHU Nantes - Hôtel Dieu; Urgences (Unité d'Investigation Clinique)

location_facility: Medizinski Zentrar-1-Sevlievo EOOD; Office of Pulmonology and Phtisiatrics

location_facility: Jehangir Clinical development Centre Pvt Ltd.

location_facility: Urgent Care Clinical Trials at Urgent Care Eleven

location_facility: Capital Medical University Beijing Hospital of Traditional Chinese Medicine

location_facility: KO-MED Centra Kliniczne Sp. z o.o.; Osrodek Badan Klinicznych

location_facility: Sterling Hospital; Clinical Research Department

location_facility: Toda Internal Medicine & Neurology Clinic

location_facility: Szigony utcai háziorvosi rendel? -Dr. Simon Judit

location_facility: Maccabi health services - Moked Hashalom

location_facility: Clalit Health Services- Pediatric Ambulatory Clinic; Pediatric Ambulatory Clinic

location_facility: Igarashi Internal Medicine Surgery Clinic

location_facility: Hacettepe University Medical Faculty; Infectious Diseases

location_facility: MHAT Stamen Iliev AD; Deparment of Infectious Diseases

location_facility: Metropolitan Clinical Research Institute

location_facility: Tongji Hospital Tongji Medical College Huazhong University of Science and Technology

location_facility: Hometown Urgent Care and Occupational Health - Clintonville

location_facility: MHAT Sliven - Military Medial Academy; Department of Infectious Diseases

location_facility: JSS Hospital; Department of clinical pharmacy

location_facility: Suzuki internal & circulatory Medical Clinic

location_facility: Siaal Research Center for Family Medicine and Primary Care; Faculty of Health Sciences

location_facility: JLN Medical College and Hospital; TB and Chest Department

location_facility: Ajanta Research centre; Research Department

location_facility: Atakent Acibadem Private Hosptial Halkali Merkez Mh.,

location_facility: Sotiria General Hospital of Athens; 3rd Department of Internal Medicine

location_facility: Koc University Medical Faculty; Department of Gynecology & Obstetrics

location_facility: SHAT for Pneumo-Phtysiatric Diseases ?Dr. Dimitar Gramatikov? EOOD; Department of Pneumology

location_facility: Medical Center Nevrocentrum; Office of Pulmonology and Phtisiatrics

location_facility: Beijing Children's Hospital, Capital Medical University

location_facility: Gyermekháziorvosi rendel?- Dr. Újhelyi János

location_facility: Attikon University General Hospital; 4th Academic Department of Internal Medicine

location_facility: Guangzhou Women and Children's Medical Center

location_facility: Medical Corporation Association KANWAKAI Musashikoganei Clinic

location_facility: Centro Integrado de Oncologia de Curitiba

location_facility: Criticare Hospital & Research Institute; Research department

location_facility: First Affiliated Hospital of Soochow University

location_facility: Diskapi Egitim Ve Arastirma Hastanesi; Infection

location_facility: Childern's Hospital of Chongqing Medical University

location_facility: Medical Center Zdrave 1; Office of Pulmonology and Phtisiatrics

location_facility: M.V Hospital and Research Centre; Research Hall

location_facility: Medical Corporation Saitadayoshikai Saikatsu Clinic

location_facility: Centro de Salud Las Aguilas; Medicina General

location_facility: Washington University School of Medicine

location_facility: Investigaciones en Patologias Respiratorias

location_facility: CH VICTOR DUPOUY; Reanimation Polyvalente

location_facility: Ankara University Faculty of Medicine Cebeci Hospital

location_facility: Santa Casa de Misericordia; de Belo Horizonte

location_facility: CEMER Centro Medico de Enfermedades Respiratorias

location_facility: III. Háziorvosi Körzet- Dr. Csomós Katalin

location_facility: Nemours Children's Hospital; Nemour's Research Institute

location_facility: Siaal Research Center for Family Medicine and Primary Care; Clalit Tet Clinic

location_facility: Medical corporation Shirayurikai Swing Nozaki Clinic

location_facility: Dr As'ad Ebrahim Bhorat; Soweto Clinical Trial Centre

location_facility: Claudia T Martorell MD LLC dba The Research Institute

location_facility: Into Research; Life Groenkloof Hospital Medical Centre

location_facility: Karadeniz Technical Uni School of Medicine; Infection Diseases & Clinical Mikrobiology Dept

location_facility: Centro de Investigacion Medico Biologico y Terapia Avanzada, S.C.

location_facility: Dokuz Eylul University Medical Faculty; Infection

location_facility: Medical Center Hera Sofia; Office of Pulmonology and Phtisiatrics

location_facility: SHAT for Pneumo-Phtysiatric Diseases ?Dr. Dimitar Gramatikov?; Dept. of Pulmonology and Phtisiatrics

location_facility: Jedda Enterprises dba Galen Clinical Research

location_facility: Medical corporation Seijinkai Takei Clinic

location_facility: Lifepoint Multispeciality Hospital; Clinical research department.

location_facility: Setshaba Research Centre; Clinical Research

location_facility: Kasturba Medical College and Hospital; Department of Medicine

location_facility: Helios Clinical Research, Inc (former Ventavia Research Group)

location_facility: Centro de Estudios Clinicos de Queretaro (CECLIQ)

location_facility: Laiko General Hospital - Uni of Athens; 1St Dept. of Internal Medicine

location_facility: Tokyo Shinagawa Hospital Medical Corporation Association Tokyokyojuno-kai

location_facility: The Third People's Hospital of Hainan Province

location_facility: Taizhou People's Hospital; Infectious Diseases

location_facility: Siaal Research Center for Family Medicine and Primary Care; Clalit Neve Zeev

location_facility: General Hospital of Ningxia Medical University

location_facility: Mazumdar Shaw Medical Centre- A unit of Narayana Hrudayalaya

location_facility: The First Affiliated Hospital of Wenzhou Medical College

location_facility: CHU de Grenoble - Hôpital André Michallon; Pôle Urgences

title: A Phase IIIB, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Clinical Efficacy Study of Baloxavir Marboxil for the Reduction of Direct Transmission of Influenza From Otherwise Healthy Patients to Household Contacts

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:
Poland
New Zealand
Argentina
Japan
Brazil
Costa Rica
Turkey
Chile
France
Hungary
Hong Kong
United States
India
China
Puerto Rico
United Kingdom
Singapore
Mexico
Spain
Greece
South Africa
Bulgaria
Israel


acronym: CANOPY-N

lead_sponsor: Novartis Pharmaceuticals

summary: The purpose of this study was to evaluate the major pathological response (MPR) rate of canakinumab given as a neoadjuvant treatment, either as single agent or in combination with pembrolizumab, in addition to evaluate the MPR of pembrolizumab as a single agent and the dynamic of the tumor microenvironment changes on treatment.

ReferenceCitation: Garrido P, Pujol JL, Kim ES, Lee JM, Tsuboi M, Gomez-Rueda A, Benito A, Moreno N, Gorospe L, Dong T, Blin C, Rodrik-Outmezguine V, Passos VQ, Mok TS. Canakinumab with and without pembrolizumab in patients with resectable non-small-cell lung cancer: CANOPY-N study design. Future Oncol. 2021 Apr;17(12):1459-1472. doi: 10.2217/fon-2020-1098. Epub 2021 Mar 2.

oa_colors: green hybrid

evidence: open (via page says license)

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

version: submittedVersion

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

repositories_concat: HAL

publisher_dissemination: Future Science Ltd

location_facility: Methodist Hospital / Methodist Cancer Center

location_facility: University of Kansas Medical Center Neurology Dept.

status: Terminated

title: A Randomized, Open-label, Phase II Study of Canakinumab or Pembrolizumab as Monotherapy or in Combination as Neoadjuvant Therapy in Subjects With Resectable Non-small Cell Lung Cancer (CANOPY-N)

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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com.

location_country:
Germany
Japan
Netherlands
Belgium
United States
Spain
Greece
Turkey
Taiwan
Canada
Russian Federation
France


title: A Phase Ia/Ib, Open Label, Dose-escalation Study of the Combination of BI 907828 With BI 754091 (Ezabenlimab) and BI 754111 and the Combination of BI 907828 With BI 754091 (Ezabenlimab) Followed by Expansion Cohorts, in Patients With Advanced Solid Tumors

summary:
This study has 2 parts. The first part is open to adults with different types of advanced cancer (solid tumors). The second part is open to people with specific types of soft tissue sarcoma, advanced lung cancer, and cancer in the stomach, bladder or bile ducts.

The participants get a combination of 2 medicines called BI 907828 and ezabenlimab (also called BI 754091). BI 907828 is a so-called MDM2 inhibitor that is being developed to treat cancer. Ezabenlimab is an antibody that may help the immune system fight cancer (immune checkpoint inhibitor). When the study started, some participants got a third medicine called BI 754111 in addition. Treatment with BI 754111 was stopped because data from another study showed no additional effect of BI 754111.

The purpose of the first part of the study is to find out the highest dose of BI 907828 that the participants can tolerate in combination with ezabenlimab. Once the best dose of BI 907828 for the combination with ezabenlimab is found, it will be used in the second part of the study.

The purpose of the second part is to see whether the combination of BI 907828 with ezabenlimab is able to make tumors shrink.

The participants are in the study as long as they benefit from and can tolerate treatment. During this time, they get infusions of ezabenlimab, and take tablets with BI 907828 every 3 weeks. The doctors check how many participants have health problems during the study. The doctors also monitor the size of the tumour.

ipd_sharing_description:
After the study is completed and the primary manuscript is accepted for publishing, researchers can use this following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement". Also, Researchers can use the following link https://www.mystudywindow.com/msw/datasharing.

com/ to find information in order to request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website. The data shared are the raw clinical study data sets.

location_country:
Germany
Hungary
Singapore
United Kingdom
Australia
Japan
Netherlands
Belgium
United States
Spain
France

location_facility: Washington University School of Medicine

location_facility: Central Hospital of Northern Pest - Military Hospital

location_facility: The University of Texas MD Anderson Cancer Center


title: Evaluating the Long-Term Outcomes and Durability of Effect Following Treatment With Cladribine Tablets for MS: An Exploratory Phase IV Ambispective Study of Patients Who Previously Participated in the CLARITY/CLARITY-EXT and ORACLE MS Clinical Trials (CLASSIC-MS)

summary: The objective of this study was to collect data both retrospectively and prospectively in order to evaluate the long-term outcomes, durability of effect, and real-world treatment patterns following treatment with Cladribine Tablets or placebo in participants with multiple sclerosis (MS) who were previously participated in the parent studies (ORACLE MS and CLARITY/CLARITY-EXT).

ReferenceType: result

ReferenceCitation: Giovannoni G, Boyko A, Correale J, Edan G, Freedman MS, Montalban X, Rammohan K, Stefoski D, Yamout B, Leist T, Aydemir A, Borsi L, Verdun di Cantogno E. Long-term follow-up of patients with relapsing multiple sclerosis from the CLARITY/CLARITY Extension cohort of CLASSIC-MS: An ambispective study. Mult Scler. 2023 May;29(6):719-730. doi: 10.1177/13524585231161494. Epub 2023 Apr 3.

license: cc-by

license_normalized: cc-by

repository_institution: Universitat Autònoma de Barcelona - Dipòsit Digital de Documents de la UAB

repositories_concat: PubMed Central;ddd.uab.cat

ipd_sharing_description: Per company policy, following approval of a new product or a new indication for an approved product in both the EU and the US, EMD Serono will share study protocols, anonymized patient level and study level data and redacted clinical study reports from clinical trials in patients with qualified scientific and medical researchers, upon request, as necessary for conducting legitimate research. Further information on how to request data can be found on our website https://www.emdgroup.com/en/research/our-approach-to-research-and-development/healthcare/clinical-trials/commitment-responsible-data-sharing.html

lead_sponsor: EMD Serono Research & Development Institute, Inc.

location_country:
Finland
Germany
Poland
Australia
Croatia
Czechia
Canada
Serbia
France
Lithuania
Portugal
Switzerland
United States
Ukraine
Romania
Tunisia
Lebanon
Belgium
Estonia
Italy
Norway
United Kingdom
Austria
Korea, Republic of
Spain
Bulgaria
Georgia
Russian Federation
Sweden

location_facility: Regional Multiple Sclerosis Centre b/o CC ECM "Neftyanik" - Neurology

location_facility: Saint-Petersburg SU on b.o. City Multifield Hospital #2 - Intensive Pulmonology and Thoracal Surgery

location_facility: Hospital Universitario Nuestra Señora de la Candelaria - Servicio de Neurologia

location_facility: Ospedale San Raffaele - U.O. di Neurologia

location_facility: SBIH "Samara Regional Clinical Hospital n.a. V.D. Seredavin

location_facility: SEIHPE "Rostov State Medical University of MoH of RF"

location_facility: Multiprofile Hospital for Active Treatment - Stara Zagora

location_facility: Barmherzige Brueder Konventspital Linz - Abteilung fuer Neurologie

location_facility: Spitalul Clinic Judetean de Urgenta Targu Mures - Sectia Clinica Neurologie I

location_facility: Vinnitsa State Medical University - Neurology dept

location_facility: RSHI"State Novosibirsk Regional Clinical Hospital"

location_facility: Hopital Roger Salengro - CHU Lille - service de neurologie D

location_facility: Ospedale Clinicizzato SS. Annunziata - Centro Regionale Sclerosi Multipla

location_facility: Military Medical Academy - MHAT - Pleven

location_facility: Fakultni nemocnice Olomouc - Neurologicka klinika

location_facility: Azienda Ospedaliera San Camillo Forlanini

location_facility: Fakultni nemocnice v Motole - Interní klinika 2. LF UK a FN Motol

location_facility: Privatni ordinace - neurologie - Nestatni zdravotnicke zarizeni

location_facility: Fondazione Istituto Neurologico Casimiro Mondino - Unità Complessa Malattie Cerebrovascolari/Stroke U

location_facility: SBIH of Moscow "City Clinical Hospital # 24" - Branch 1

location_facility: SI Institute of Neurology, Psychiatry and Narcology of NAMSU - Dept of Neuroinfections and Multiple Sclerosis

location_facility: Azienda Ospedaliera Universitaria Policlinico Tor Vergata - Dip. Neuroscienze-Centro per la Sclerosi Multipla

location_facility: Vseobecna fakultni nemocnice v Praze - Dept of Neurologicka klinika 1.LF UK a VFN v Praze

location_facility: Azienda Ospedaliera Universitaria "Federico II" - Gastroenterologia Pediatrica

location_facility: Fakultni nemocnice Ostrava - Dept of Neurology

location_facility: NHI "Central Clinical Hospital #2 of JSC "Russian Railways" n.a. N.A. Semashko

location_facility: SBHI of Yaroslavl Region "Clinical Hospital # 8" - Cardiology

location_facility: Prof. Dr. med. Zbigniew Stelmasiak Specjalistyczny Gabinet Neurologiczny

location_facility: A.O.U. Policlinico V. Emanuele - Presidio Gaspare Rodolico - Clinica Neurologica I

location_facility: Szpital im. Mikołaja Kopernika - Neurology

location_facility: Royal Hallamshire Hospital - Dept of Neurology

location_facility: Centro Hospitalar de Lisboa Central, E.P.E. - Hospital de Santo António dos Capuchos - Serviço de Neurologia

location_facility: SAIH "Kemerovo Regional Clinical Hospital" - PARENT

location_facility: CHU Rennes - Hopital Pontchaillou - Neurologie - Clinique Neurologique

location_facility: Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari - Neurofisiopatologia

location_facility: Szpital Kliniczny im.Heliodora Swiecickiego Uniwersytetu Medycznego im.K. Marcinkowskiego w Poznaniu - Dept of Neurology

location_facility: Uniwersyteckie Centrum Kliniczne - Dept of Neurology

location_facility: American University of Beirut Medical Center

location_facility: Hôpital Habib Bourguiba - Service de Neurologie

location_facility: SBIH of Moscow region " Moscow Regional Scientific and Research Clinical Institute n.a. M.F. Vladimi

location_facility: Azienda Ospadaliero Universitaria San Martino - PARENT

location_facility: Rowan University School of Osteopathic Medicine - Department of Medicine

location_facility: Pavlov First Saint Petersburg State Medical University - PARENT

location_facility: Azienda Socio Sanitaria Territoriale della Valle Olona (presidio di Gallarate)

location_facility: Department of Neurology, Haukeland University

location_facility: (CHUV), Centre Hospitalier Universitaire Vaudois - Departement des Neurosciences Cliniques

location_facility: Clinical Center Nis - Clinic of Neurology

location_facility: Universitaetsmedizin Rostock - Klinik und Poliklinik fuer Neurologie

location_facility: MHAT - "National Heart Hospital" EAD - Multiple Clinics

location_facility: Empire Neurology, PC - Empire Neurology PC

location_facility: Krajska zdravotni, a.s. - Nemocnice Teplice, o.z. - Neurologicke oddeleni

location_facility: University of Maryland, Baltimore - Maryland Center for MS

location_facility: RSBIH "Smolensk Regional Clinical Hospital"

location_facility: SBIH "Leningrad Regional Clinical Hospital"

location_facility: Nottingham University Hospital - Division of Clinical Neurology

location_facility: CHU de Nîmes - Hôpital Carémeau - Service de Neurologie

location_facility: Heinrich-Heine-Universitaet Duesseldorf - Klinik fuer Nephrologie

location_facility: Hospital of Lithuanian University of Health Sciences Kaunas Clinics - Neurology Clinic

location_facility: Spitalul Clinic Judetean de Urgenta "Pius Branzeu" Timisoara - Clinica de Neurologie II

location_facility: Ospedale Sant'Andrea di Roma - MS Center

location_facility: SBEI HPE "Saratov State Medical University n.a. V. I. Razumovskiy" of the MoH of the RF

location_facility: Azienda Ospedaliero_Universitaria S. Luigi Gonzaga - Centro di Riferimento Regionale Sclerosi Multipla

intervention_type: Other


title: Phase I/II, Open Label, Multicenter Study of Rapcabtagene Autoleucel in Adult Patients With CLL/SLL, 3L+ DLBCL, ALL and 1L HR LBCL

summary: This is a phase I/II study to evaluate the feasibility, safety and preliminary antitumor efficacy of rapcabtagene autoleucel (also known as YTB323). Rapcabtagene autoleucel will be investigated in combination with ibrutinib in chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) and as single agent in diffuse large B-cell lymphoma (3L+ DLBCL), adult acute lymphoblastic leukemia (ALL) and 1st Line High Risk Large B-Cell Lymphoma (1L HR LBCL).

location_country:
Germany
Italy
Australia
Japan
Austria
United States
Spain
France

location_facility: University of Chicago Medical Center Hematology and Oncology

location_facility: Sarah Cannon Research Institute Methodist Hospital

location_facility: H Lee Moffitt Cancer Center and Research Institute .

location_facility: Northwestern University Northwestern Memorial Hospital Trans

location_facility: University of Pennsylvania Clinical Perelman Center for Adv Med

location_facility: Sarah Cannon Research Institute Drug Ship - 4

location_facility: University Of California Los Angeles UCLA Hematology Oncology

location_facility: University of Kansas Cancer Center SC - CTL019C2201

location_facility: Medical College of Wisconsin Main Centre


acronym: REFALS-ES

lead_sponsor: Orion Corporation, Orion Pharma

summary:
This study provides an opportunity for subjects in the REFALS (3119002; NCT03505021) study to continue treatment with oral levosimendan. The study will also provide more information about long-term safety and effectiveness of oral levosimendan in patients with ALS.

This is an open-label study, so that all eligible subjects that complete the double-blind REFALS study (48-weeks of treatment) will have the opportunity to receive oral levosimendan treatment. The primary objective, in addition to continuing treatment for subjects enrolled in the REFALS study, is to evaluate long-term safety of oral levosimendan in ALS patients. Another important objective is to explore long-term effectiveness of oral levosimendan in the treatment of patients with ALS.

This study is open only to patients taking part in the REFALS study.

ReferenceCitation: Cudkowicz M, Genge A, Maragakis N, Petri S, van den Berg L, Aho VV, Sarapohja T, Kuoppamaki M, Garratt C, Al-Chalabi A; REFALS investigators. Safety and efficacy of oral levosimendan in people with amyotrophic lateral sclerosis (the REFALS study): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet Neurol. 2021 Oct;20(10):821-831. doi: 10.1016/S1474-4422(21)00242-8.

oa_host_type: closed

publisher_dissemination: Elsevier

location_facility: Karolinska University Horpital Huddinge Neurology Clinic

location_facility: Helsinki University Central Hospital, Neurology Outpatients Clinic

location_facility: University of California Irvine Medical Center

location_facility: Norrlanda University Hospital Neuro-huvud-hals-centrum Vasterbotten

location_facility: Universitatsklinikum Munster, Institut fur Schalfmedizin und Neuromuskalaire Erkrankungen

location_facility: Centre Hospitalier Affilie Universitaire de Quebec

location_facility: University of Florida McKnight Brain Institute

location_facility: Centre Hospitalier Regional de la Vitadelle

location_facility: Perron Institute for Neurological and Translational Science

location_facility: Hospital Universitario Reina Sofia Servicio Neurologia

location_facility: University of Michigan, Michigan Medicine University Hospital

location_facility: Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino

location_facility: Karlstad Central Hospital Neurology and Rehabilitation

location_facility: Azienda Ospedaliero Universitaria Pisana Ospedale Santa Chiara

location_facility: The George Washington Medical Faculty Associates

location_facility: Neurosciences Institute - Neurology Charlotte

location_facility: Barts Health NHS Trust Royal London hospital

location_facility: Northwestern University Feinberg School of Medicine

location_facility: Universitatsklinikum Jena, Klinik fur Neurologie

location_facility: Azienda Ospedaliera Universitaria-maggiore della Carita di Novara

location_facility: Universitatsmedizin Rostock, Klinik und Poliklinik fuer Neurologie

location_facility: Montreal Neurological Institute and Hospital

location_facility: The Ohio State University Wexner Medical center

location_facility: Universitatsklinikum Ulm, Poliklinik fur Neurologie

location_facility: University of Alberta, Division of Neurology

location_facility: Medizinische Universitat wein Universitatsklinik ffur Neurologie

location_facility: Holy Cross Hospital Neuroscience Institute

location_facility: Washington University School of Medicine

location_facility: University of Utah Health-Imaging & Neurosciences Center in research Park

location_facility: Beaumont Hospital, Clinical Research Centre

location_facility: Centre Hospitalier Universitaire de Limoges Service de Neurologie

location_facility: Hopital Pasteur Centre de reference des Malades Neuromusculaires et SLA

location_facility: Policlinico Umberto I di Roma Clinica Neurologica

location_facility: Alberta Health Services-Neuromuscular Clinic

location_facility: The Walton Centre NHs Foundation Trust, Neurology and Neurosurgery

location_facility: Hospital Universitario y Politecnico de La Fe

location_facility: Charite Universitatmedizin Berlin- Campus Virchow-Klinikum

location_facility: Neuromuscular research Centre and Neuromuscular Clinic of Arizona

location_facility: Wake Forest University Baptist Medical Center

location_facility: ICS Maugeri Spa S UO Riabilitazione Nurologica

location_facility: Hopital Gui de Chauliac Service de Neurologie

title: Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS: Open-Label Extension for Patients Completing Study 3119002

location_country:
Finland
Germany
Ireland
Italy
United Kingdom
Australia
Sweden
Netherlands
Austria
Belgium
United States
Spain
Canada
France


acronym: DYSTANCE 51

lead_sponsor: Wave Life Sciences Ltd.

summary: This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping intervention (DYSTANCE 51)

location_facility: U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud

location_facility: Fondazione Policlinico Universitario A Gemelli

location_facility: London Health Sciences Centre - Hospital

location_facility: Drottning Silvias Barn Och Ungdomssjukhus

title: A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)

location_country:
Italy
United Kingdom
Sweden
Belgium
United States
Czechia
Canada
France


acronym: NTHI MCAT-009

lead_sponsor: GlaxoSmithKline

summary:
This study will provide information regarding the sequential administration of two vaccines adjuvanted with AS01.

The aim of this study is to understand immunogenicity and safety of NTHi-Mcat vaccine when administered sequentially after Shingrix vaccine and to compare to the immunogenicity of NTHi-Mcat vaccine administered alone. This study will also provide information regarding whether a specific time period is required between the administration of these two different vaccines containing the same adjuvant- AS01 components.

The population of this study will include healthy smokers and ex-smokers of 50 to 80 years of age which will be used as a proxy for the COPD population.

title: Immunogenicity and Safety Study of GSK's Investigational Vaccine (GSK3277511A) When Administered in Healthy Smokers and Ex-smokers Following Receipt of Shingrix Vaccine

ipd_sharing_description: IPD for this study will be made available via the Clinical Study Data Request site.

location_country: Finland Italy Spain Estonia France


title: A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

summary: The purpose of this study is to obtain information pertaining to the occurrence of antibodies to investigational SPK-3006 capsid and GAA, GAA activity and GAA antigen levels in the usual care setting of Late-Onset Pompe Disease (LOPD) participants on an enzyme replacement regimen. Additionally, a careful evaluation of laboratory and functional testing in patients with LOPD may provide information to better understand the disease features and better drive the design of a future interventional investigational gene therapy trial. An understanding of the underlying status of liver and muscle health in individuals with LOPD may also inform best surveillance during the conduct of gene therapy trials.

primary_purpose: Screening

lead_sponsor: Spark Therapeutics

location_country:
Germany
Italy
United Kingdom
Netherlands
United States
France

location_facility: Fondazione Mondino Istituto Neurologico Nazionale a Carattere Scientifico

location_facility: Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino

location_facility: Assistance Publique Hôpitaux de Marseille

location_facility: Universita degli Studi di Milano - Clinica Oculistica I

location_facility: Azienda Ospedaliero Universitaria Pisana

location_facility: Università degli Studi di Napoli Federico II

intervention_type: Diagnostic Test


lead_sponsor: Novo Nordisk A/S

summary:
The study compares 2 medicines used for the treatment of children who are born small and who stayed small: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe).

Participants will either get somapacitan or Norditropin® - which treatment is decided by chance. Both participants and the study doctor will know which treatment the participants get. The study will last for 4 years. Participants will take either an injection once every week or once every day. Participants will have 9 clinic visits and will be in the study for 1 year. The follow-up period is at least 30 days.

title: A Dose-finding Trial Evaluating the Effect and Safety of Once-weekly Treatment of Somapacitan Compared to Daily Norditropin® in Children With Short Stature Born Small for Gestational Age With no Catch-up Growth by 2 Years of Age or Older

ipd_sharing_description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

location_country:
Ireland
Poland
Algeria
Japan
Thailand
Serbia
France
Hungary
United States
Latvia
Ukraine
India
Estonia
Italy
United Kingdom
Austria
Spain
Russian Federation
Israel


title: A Phase 1 First-in-Human Dose Escalation and Expansion Study for the Evaluation of Safety, Pharmacokinetics, Pharmacodynamics and Anti-tumor Activity of SAR441000 Administered Intratumorally as Monotherapy and in Combination With Cemiplimab in Patients With Advanced Solid Tumors

summary:
Primary Objectives:

Dose Escalation: To determine maximum tolerated dose (MTD) or maximum administered dose (MAD) and overall safety and tolerability profile of SAR441000 when administered intratumorally as monotherapy and in combination with cemiplimab in patients who have no alternative standard treatment options.
Dose Expansion (Combination): To determine the objective response rate of SAR441000 administered intratumorally in combination with cemiplimab in patients with melanoma, cutaneous squamous cell carcinoma or head and neck squamous cell carcinoma.

Secondary Objectives:

To characterize the pharmacokinetic (PK) profile of SAR441000 administered as monotherapy and in combination with cemiplimab.
To assess the immunogenicity of SAR441000.
To characterize the safety of SAR441000 when administered intratumorally in combination with cemiplimab.
To determine the disease control rate (DCR), duration of response (DoR) and progression free survival (PFS) of SAR441000.
To determine the recommended dose of SAR441000 for the expansion phase.

lead_sponsor: Sanofi

sponsor_collaborators:
Sanofi
BioNTech RNA Pharmaceuticals GmbH

location_country:
Germany
Netherlands
Belgium
United States
Spain
France

location_facility: Dana Farber Cancer Institute Site Number : 8400003

location_facility: ~University of Texas - MD Anderson Cancer Center Site Number : 8400002

location_facility: The University of Kansas Clinical Research Center Site Number : 8400001

location_facility: Cleveland Clinic Foundation Site Number : 8400007


acronym: MyCIDPchoice

lead_sponsor: UCB Biopharma S.P.R.L.

summary: The purpose of the study is to evaluate clinical efficacy of rozanolixizumab as a treatment for subjects with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).

title: A Multicenter, Randomized, Subject-Blind, Investigator-Blind, Placebo-Controlled, Parallel-Group Study Evaluating the Efficacy, Safety, and Tolerability of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

ipd_sharing_description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

location_country:
Germany
United Kingdom
Denmark
Netherlands
Belgium
United States
Spain
France


title: A PHASE 2B, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, MULTICENTER, DOSE-RANGING STUDY TO EVALUATE THE EFFICACY AND SAFETY PROFILE OF PF-06700841 IN PARTICIPANTS WITH ACTIVE SYSTEMIC LUPUS ERYTHEMATOSUS (SLE)

summary: Assessment of PF-06700841 in participants with moderate to severe active, generalized Systemic Lupus Erythematosus (SLE) that have inadequate response to standard of care.

ReferenceCitation: Hannon CW, McCourt C, Lima HC, Chen S, Bennett C. Interventions for cutaneous disease in systemic lupus erythematosus. Cochrane Database Syst Rev. 2021 Mar 9;3(3):CD007478. doi: 10.1002/14651858.CD007478.pub2.

oa_host_type: repository

unpaywall_oa_status: green

publisher_dissemination: Wiley

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

lead_sponsor: Pfizer

location_country:
Germany
Poland
Argentina
Australia
Japan
Czechia
Canada
Serbia
France
Colombia
Portugal
Hungary
Hong Kong
United States
Ukraine
Romania
Belgium
China
Italy
United Kingdom
Mexico
Korea, Republic of
Spain
Greece
Taiwan
Bulgaria

location_facility: Tianjin Medical University General Hospital

location_facility: Complexo Hospitalario Universitario A Coruna

location_facility: Laiko General Hospital, University of Athens

location_facility: Arthritis and Osteoporosis Medical Center

location_facility: Ruijin Hospital- Shanghai Jiaotong University School of Medicine

location_facility: CNPE "Ternopil University Hospital" of Ternopil regional council, Department of Rheumatology

location_facility: Ziekenhuisnetwerk Antwerpen Jan Palfijn (ZNA Jan Palfijn)

location_facility: Hôpital Pitié Salpêtrière, Centre des Maladies Auto-immunes

location_facility: Feinstein Institute for Medical Research

location_facility: The Catholic Univ. of Korea Seoul St. Mary's Hospital

location_facility: West Broward Rheumatology Associates, Inc

location_facility: Centro Peninsular de Investigación Clínica S.C.P.

location_facility: Centro de Investigación en Reumatología y Especialidades Médicas SAS - CIREEM SAS

location_facility: Centro de Investigaciones Medicas Tucuman

location_facility: Doncaster Royal Infirmary, Doncaster and Bassetlaw Teaching Hospitals NHS Foundation Trust

location_facility: Zakarpattia Regional Clinical Hospital n.a. A. Novak

location_facility: Atlanta Research Center for Rheumatology

location_facility: Communal non-profitable enterprise "Lviv City Clinical Hospital #4"

location_facility: The First Affiliated Hospital of Soochow University

location_facility: Zespol Poradni Specjalistycznych Reumed, Onyksowa Filia nr 2

location_facility: CITER Centro de Investigación y Tratamiento de las Enfermedades Reumáticas S.A. de C.V.

location_facility: SC Centrul Medical de Diagnostic si Tratament Ambulator Neomed SRL

location_facility: National Hospital Organization Kyushu Medical Center

location_facility: UAB Hospital-Clinical Research Unit (CRU)

location_facility: Irving Institute for Clinical and Transitional Research

location_facility: Investigación Biomédica para el Desarrollo de Fármacos S.A. de C.V

location_facility: Centro Hospitalar Universitario do Porto, E.P.E

location_facility: Investigational Drug Services University of Kansas Hospital

location_facility: Vojnomedicinska akademija, Klinika za reumatologiju

location_facility: Bluecare Salud S.A.S Sede Centro Médico Integral Chicó MedPlus CRI

location_facility: Klinicki Centar Srbije, Klinika za alergologiju i imunologiju

location_facility: Guy's and St Thomas' NHS Foundation Trust Guy's Hospital

location_facility: East Bay Rheumatology Medical Group, Inc.

location_facility: Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán

location_facility: Rheumatic Disease Clinical Research Center, LLC

location_facility: NYU Langone Ambulatory Care Brooklyn Heights

location_facility: Arthritis & Rheumatology Center of Oklahoma PLLC

location_facility: Centrum Medyczne MEDENS S.C. Grupowa Praktyka Lekarska

location_facility: CNPE "Odesa Regional Clinical Hospital" of Odesa Regional Council

location_facility: CHU de Bordeaux, Groupe Hospitalier Sud, Hôpital Haut Lévèque

location_facility: UAB Department of Medicine Clinical Research Enterprise

location_facility: National Hospital Organization Chiba-East Hospital

location_facility: Inland Rheumatology Clinical Trials, Inc.

location_facility: Morales Vargas Centro de Investigación S.C.

location_facility: Multifield Medical Center of Odessa National Medical University

location_facility: National Hospital Organization Asahikawa Medical Center

location_facility: Narodowy Instytutu Geriatrii, Reumatologii i Rehabilitacji

location_facility: Centre intégré de santé et de services sociaux du Bas-Saint-Laurent

location_facility: Kyungpook National University Hospital (KNUH)

location_facility: CNPE "Vinnytsya regional Clinical Hospital named after N.I.Pirogov Vinnytsia Regional Council"

location_facility: Inland Rheumatology and Osteoporosis Medical Group

location_facility: Centre de Recherche Musculo-Squelettique

location_facility: University of Michigan Brighton Center for Specialty Care

location_facility: Institut Niska Banja, Klinika za Reumatologiju

location_facility: Universitätsklinikum Schleswig Holstein - Campus Lübeck

location_facility: Centro Integral de Reumatología del Caribe S.A.S - CIRCARIBE S.A.S

location_facility: Hospital Professor Doutor Fernando da Fonseca, E.P.E

location_facility: Chang Gung Memorial Hospital Linkou Branch

location_facility: Mazowieckie Centrum Reumatologii i Osteoporozy M. Przygodzka Spolka jawna

location_facility: Spitalul Clinic Judetean de Urgenta Cluj-Napoca

location_facility: University General Hospital of Heraklion

location_facility: State Institution National Scientific Center "M.D. Strazhesko Institute of Cardiology"

location_facility: The Second Xiangya Hospital of Central South University

location_facility: Unidade Local de Saude do Alto Minho, E.P.E.

location_facility: The Leeds Teaching Hospitals NHS Trust, Chapel Allerton Hospital

location_facility: Investigación y Biomedicina de Chihuahua, Sociedad Civil

location_facility: CINTRE Centro de Investigación y Tratamiento Reumatológico S.C.


acronym: PRISM

lead_sponsor: miRagen Therapeutics, Inc.

summary:
The main objective of this clinical trial is to study the efficacy and safety of cobomarsen (also known as MRG-106) for the treatment of cutaneous T-cell lymphoma (CTCL), mycosis fungoides (MF) subtype in subjects who have confirmed disease progression following treatment with vorinostat in the SOLAR clinical study (MRG106-11-201). Cobomarsen is designed to inhibit the activity of a molecule called miR-155 that may be important to the growth and survival of MF cancer cells.

The effects of treatment will be measured based on changes in skin lesion severity, disease-associated symptoms, and quality of life, as well as the length of time that the subject's disease remains stable or improved, without evidence of disease progression. The safety and tolerability of cobomarsen will be assessed based on the frequency and severity of observed side effects.

location_facility: University of Washington/Seattle Cancer Care Alliance

location_facility: Washington University School of Medicine

location_facility: The Ohio State University and Wexner Medical Center

title: PRISM: An Open-label, Multi-Center Extension Study to Investigate the Efficacy and Safety of Cobomarsen (MRG-106) Following Systemic Treatment in Subjects With Cutaneous T-Cell Lymphoma (CTCL), Mycosis Fungoides (MF) Subtype, Who Have Completed the SOLAR Study

location_country:
United States
Belgium
France


summary: The study to evaluate M7824 monotherapy in participants with advanced or metastatic biliary tract cancer (BTC) who failed or were intolerant to first-line (1L) chemotherapy.

ReferenceCitation: Yoo C, Oh DY, Choi HJ, Kudo M, Ueno M, Kondo S, Chen LT, Osada M, Helwig C, Dussault I, Ikeda M. Phase I study of bintrafusp alfa, a bifunctional fusion protein targeting TGF-beta and PD-L1, in patients with pretreated biliary tract cancer. J Immunother Cancer. 2020 May;8(1):e000564. doi: 10.1136/jitc-2020-000564.

repository_institution: Yonsei University Medical Library - YUHSpace

repositories_concat: PubMed Central;ir.ymlib.yonsei.ac.kr

sponsor_collaborators: EMD Serono Research & Development Institute, Inc.

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Kindai University Hospital - Dept of Gastroenterology

location_facility: National Cancer Center Hospital - Dept of Hepatobiliary and Pancreatic Oncology

location_facility: Affiliated Tumor Hospital of Harbin Medical University

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori Milano

location_facility: Groupe Hospitalier Sud - Hôpital Haut Lévêque - Service Hepato Gastroentérologie Oncologie Digest

location_facility: Sidney Kimmel Comprehensive Cancer Center at John Hopkins

location_facility: H. Lee Moffitt Cancer Center and Research Institute, Inc

location_facility: Hospital Universitari Vall d'Hebron - Dept of Oncology

location_facility: Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi - Unita' Operativa di Pediatria

location_facility: Seoul National University Bundang Hospital

location_facility: Kyorin University Hospital - Dept of Oncology

location_facility: Mayo Clinic in Florida - Department of Neurology

location_facility: Policlinico Universitario Agostino Gemelli - UOC Oncologia Medica

location_facility: St James's University Hospital - Dept of Oncology

location_facility: Hospital Universitario HM Madrid Sanchinarro - Servicio de Oncologia

title: A Phase II, Multicenter, Open-label Study to Investigate the Clinical Efficacy of M7824 Monotherapy in Participants With Locally Advanced or Metastatic Biliary Tract Cancer Who Fail or Are Intolerant to First-line Platinum-Based Chemotherapy

ipd_sharing_description: We are committed to enhancing public health through responsible sharing of clinical trial data. Following approval of a new product or a new indication for an approved product in both the US and European Union, the study sponsor and/or its affiliated companies will share study protocols, anonymized patient data and study level data, and redacted clinical study reports with qualified scientific and medical researchers, upon request, as necessary for conducting legitimate research. Further information on how to request data can be found on our website bit.ly/IPD21

location_country:
Italy
United Kingdom
Japan
United States
China
Korea, Republic of
Spain
Taiwan
France