title: A Multicenter Study for the Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody

summary: International, multicenter, long-term, follow-up study that will enroll HLH patients who have received NI-0501 in a previous clinical trial, in the context of the clinical development program for NI-0501

ReferenceCitation: Locatelli F, Jordan MB, Allen C, Cesaro S, Rizzari C, Rao A, Degar B, Garrington TP, Sevilla J, Putti MC, Fagioli F, Ahlmann M, Dapena Diaz JL, Henry M, De Benedetti F, Grom A, Lapeyre G, Jacqmin P, Ballabio M, de Min C. Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis. N Engl J Med. 2020 May 7;382(19):1811-1822. doi: 10.1056/NEJMoa1911326.

name: From the Department of Pediatrics, Sapienza, University of Rome (F.L.), and the Department of Pediatric Hematology–Oncology (F.L.) and Division of Rheumatology (F.D.B.), IRCCS Bambino Gesù Children’s Hospital, Rome, Pediatric Hematology–Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona (S.C.), the Pediatric Hematology–Oncology Unit, Department of Pediatrics, University of Milano–Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza (C.R.), the Clinic...

family: Locatelli

full_name: Franco Locatelli

given: Franco

family: Jordan

full_name: Michael B. Jordan

given: Michael B.

family: Allen

full_name: Carl Allen

given: Carl

family: Cesaro

full_name: Simone Cesaro

given: Simone

family: Rizzari

full_name: Carmelo Rizzari

given: Carmelo

family: Rao

full_name: Anupama Rao

given: Anupama

family: Degar

full_name: Barbara Degar

given: Barbara

family: Garrington

full_name: Timothy P. Garrington

given: Timothy P.

family: Sevilla

full_name: Julian Sevilla

given: Julian

family: Putti

full_name: Maria-Caterina Putti

given: Maria-Caterina

family: Fagioli

full_name: Franca Fagioli

given: Franca

family: Ahlmann

full_name: Martina Ahlmann

given: Martina

family: Dapena Diaz

full_name: Jose-Luis Dapena Diaz

given: Jose-Luis

family: Henry

full_name: Michael Henry

given: Michael

family: De Benedetti

full_name: Fabrizio De Benedetti

given: Fabrizio

family: Grom

full_name: Alexei Grom

given: Alexei

family: Lapeyre

full_name: Genevieve Lapeyre

given: Genevieve

family: Jacqmin

full_name: Philippe Jacqmin

given: Philippe

family: Ballabio

full_name: Maria Ballabio

given: Maria

family: de Min

full_name: Cristina de Min

given: Cristina

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

location_country:
Italy
France
Spain
United States
United Kingdom

location_facility: Spectrum Health Helen Devos Children's Hospital

location_facility: Ospedale Donna Bambino - U.O.C. Oncoematologia Pediatrica

location_facility: University of North Carolina at Chapel Hill

location_facility: Cincinnati Children's Hospital - Division of Immunobiology

location_facility: Great Ormond Street Hospital - Department of Haematology

location_facility: Fondazione MBBM c/o Ospedale San Gerardo Clinica Pediatrica

location_facility: Hospital Universitario Niño Jesús Servicio de Hemato-Oncología Pediátrica

location_facility: Ospedale Pediatrico Bambino Gesù - UO Reumatologia

location_facility: Hospital Universitario Vall d'Hebron Servei de Hematologia i Oncologia


intervention_type: Drug

status: Completed

study_start_date_type: Actual

study_type: Interventional

lead_sponsor: Mirum Pharmaceuticals, Inc.

enrollment_type: Actual

primary_purpose: Treatment

location_country:
United States
United Kingdom
France
Poland

acronym: INDIGO

location_facility: The Children's Memorial Health Institute

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Children's Hospital of Pittsburgh of UPMC

ipd_sharing: No

study_completion_date_type: Actual

type: EudraCT Number

source: euctr

title: Open Label Study of the Efficacy and Long Term Safety of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis

summary: This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus associated with PFIC.

lead_sponsor_type: industriel

status_simplified: Completed


design_allocation: Randomized

intervention_type: Other

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

lead_sponsor: Shire

location_country:
United States
Mexico
France
Australia
Spain
Canada
United Kingdom

acronym: AIM-IT

location_facility: Children's Hospital and Research Center at Oakland

location_facility: Hospital Infantil Universitario Niño Jesus

location_facility: Women's and Children's Hospital, 72 King William Road

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

ipd_sharing: Yes

title: A Controlled, Randomized, Two-arm, Open-label, Assessor-blinded, Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

summary: Study HGT-HIT-094 is a multicenter study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter syndrome and cognitive impairment who have previously received and tolerated a minimum of 4 months of therapy with Elaprase.


title: A Prospective, Multicenter, Single-Arm Trial With the Lutonix Drug Coated Balloon for Treatment of Long Lesions in Femoropopliteal Arteries

summary: To demonstrate efficacy and safety of the Lutonix® Drug Coated Balloon for treatment of long TASC II Class C and D lesions (≥ 14 cm) lesions in the SFA

study_type: Observational

time_perspective: Prospective

lead_sponsor: C. R. Bard

location_country: Austria Switzerland France Belgium Germany

location_facility: Luzerner Kantonsspital, Division of Angiology

location_facility: Krankenhaus Barmherzige Brüder Regensburg

intervention_type: Device


title: The Robotic Vascular and Endovascular Registry (ROVER)

acronym: ROVER

summary:
The purpose of the registry is to gather both retrospective and prospective case data on the use of the commercially available Magellan Robotic System and Magellan Robotic Catheters in accordance with the approved intended use. For prospective cases, follow-up patient data will be collected at 14 days (± 5 days) post procedure to assess treatment success, primary patency of intended targeted vessel region, and adverse events.

The data will be analyzed for medical education, societal presentation, and/or publication by the investigators.

Over the next 2+ years, physicians who meet the selection criteria will be invited to participate in the registry.

status: Terminated

lead_sponsor: Hansen Medical

location_country:
Germany
United States
France

location_facility:
Good Samaritan Hospital
Keck Medical Center of USC
Herz- und Gefäßzentrum Bad Bevensen
Miami Valley Hospital
Miami Cardiac & Vascular Institute
The Methodist Hospital
University Hospital of Strasbourg

status_simplified: Unknown


type: Registry Identifier

type: Other Identifier

type: U.S. NIH Grant/Contract

title: A Randomized Two-Arm Phase II Study of Trametinib Alone and in Combination With GSK2141795 in Patients With Advanced Uveal Melanoma

summary: This randomized phase II trial studies how well trametinib with or without Akt inhibitor GSK2141795 (GSK2141795) works in treating patients with uveal melanoma that has spread to other parts of the body (metastatic). Trametinib and GSK2141795 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether trametinib is more effective with or without GSK2141795 in treating patients with metastatic uveal melanoma.

lead_sponsor: National Cancer Institute (NCI)

location_country:
United States
United Kingdom
France

location_facility: Vanderbilt University/Ingram Cancer Center

location_facility: Emory University Hospital/Winship Cancer Institute

location_facility: Columbia University/Herbert Irving Cancer Center


title: An Open-Label, Single Arm, Phase 1 Study of the Pharmacokinetics and Safety of Carfilzomib in Subjects With Advanced Malignancies and Varying Degrees of Hepatic Impairment

summary: The purpose of this study is to compare the safety and efficacy of carfilzomib, including measuring the amount of the study drug in the blood at certain times following dosing. This study is being done in people with varying degrees of liver function to see if they respond differently to the study drug.

design_allocation: Non-Randomized

primary_purpose: Other

ReferenceType: derived

ReferenceCitation: Brown J, Plummer R, Bauer TM, Anthony S, Sarantopoulos J, De Vos F, White M, Schupp M, Ou Y, Vaishampayan U. Pharmacokinetics of carfilzomib in patients with advanced malignancies and varying degrees of hepatic impairment: an open-label, single-arm, phase 1 study. Exp Hematol Oncol. 2017 Oct 3;6:27. doi: 10.1186/s40164-017-0086-1. eCollection 2017.

family: Brown

full_name: Jennifer Brown

given: Jennifer

sequence: first

family: Plummer

full_name: Ruth Plummer

given: Ruth

sequence: additional

family: Bauer

full_name: Todd M. Bauer

given: Todd M.

family: Anthony

full_name: Stephen Anthony

given: Stephen

family: Sarantopoulos

full_name: John Sarantopoulos

given: John

family: De Vos

full_name: Filip De Vos

given: Filip

family: White

full_name: Mike White

given: Mike

family: Schupp

full_name: Marco Schupp

given: Marco

family: Ou

full_name: Ying Ou

given: Ying

family: Vaishampayan

full_name: Ulka Vaishampayan

given: Ulka

bso_classification: Medical research

currency_apc_doaj: GBP

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Experimental Hematology & Oncology

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

repository_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

evidence: oa repository (via pmcid lookup)

license_normalized: no license

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

lead_sponsor: Amgen

location_country:
United States
United Kingdom
France
Netherlands

location_facility: Sir Bobby Robson Cancer Trials Research Centre

location_facility: Northern Ireland Cancer Trials Centre - Queen's University Belfast TriaIs Centre


title: Biogen Idec Multiple Sclerosis Pregnancy Exposure Registry

summary: The primary objective of the study is to prospectively evaluate pregnancy outcomes in women with multiple sclerosis who were exposed to a Registry-specified Biogen Multiple Sclerosis product during the eligibility window for that product. The Registry-specified Biogen MS products being studied are dimethyl fumarate, and Pegylated human interferon beta-1a. The secondary objective of the study is to prospectively evaluate pregnancy outcomes in women with MS who were unexposed to disease-modifying therapies (DMTs).

status: Recruiting

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ReferenceCitation: Hellwig K, Rog D, McGuigan C, Houtchens MK, Bruen DR, Mokliatchouk O, Branco F, Peng X, Everage NJ. Interim Analysis of Pregnancy Outcomes After Exposure to Dimethyl Fumarate in a Prospective International Registry. Neurol Neuroimmunol Neuroinflamm. 2021 Nov 23;9(1). pii: e1114. doi: 10.1212/NXI.0000000000001114. Print 2022 Jan.

family: Hellwig

full_name: Kerstin Hellwig

given: Kerstin

family: Rog

full_name: David Rog

given: David

family: McGuigan

full_name: Christopher McGuigan

given: Christopher

family: Houtchens

full_name: Maria K. Houtchens

given: Maria K.

family: Bruen

full_name: Denise R. Bruen

given: Denise R.

family: Mokliatchouk

full_name: Oksana Mokliatchouk

given: Oksana

family: Branco

full_name: Filipe Branco

given: Filipe

family: Peng

full_name: Xiaomei Peng

given: Xiaomei

family: Everage

full_name: Nicholas J. Everage

given: Nicholas J.

currency_apc_doaj: USD

journal_name: Neurology - Neuroimmunology Neuroinflammation

oa_host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

publisher: Ovid Technologies (Wolters Kluwer Health)

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

lead_sponsor: Biogen

location_country:
Italy
United States
Germany
France
Poland
Ireland
Australia
Spain
Canada
United Kingdom

status_simplified: Ongoing


ReferenceCitation: Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4.

family: Eichler

full_name: Florian Eichler

given: Florian

family: Duncan

full_name: Christine Duncan

given: Christine

family: Musolino

full_name: Patricia L. Musolino

given: Patricia L.

family: Orchard

full_name: Paul J. Orchard

given: Paul J.

family: De Oliveira

full_name: Satiro De Oliveira

given: Satiro

family: Thrasher

full_name: Adrian J. Thrasher

given: Adrian J.

family: Armant

full_name: Myriam Armant

given: Myriam

family: Dansereau

full_name: Colleen Dansereau

given: Colleen

family: Lund

full_name: Troy C. Lund

given: Troy C.

family: Miller

full_name: Weston P. Miller

given: Weston P.

family: Raymond

full_name: Gerald V. Raymond

given: Gerald V.

family: Sankar

full_name: Raman Sankar

given: Raman

family: Shah

full_name: Ami J. Shah

given: Ami J.

family: Sevin

full_name: Caroline Sevin

given: Caroline

family: Gaspar

full_name: H. Bobby Gaspar

given: H. Bobby

family: Gissen

full_name: Paul Gissen

given: Paul

family: Amartino

full_name: Hernan Amartino

given: Hernan

family: Bratkovic

full_name: Drago Bratkovic

given: Drago

family: Smith

full_name: Nicholas J.C. Smith

given: Nicholas J.C.

family: Paker

full_name: Asif M. Paker

given: Asif M.

family: Shamir

full_name: Esther Shamir

given: Esther

family: O’Meara

full_name: Tara O’Meara

given: Tara

family: Davidson

full_name: David Davidson

family: Aubourg

full_name: Patrick Aubourg

given: Patrick

family: Williams

full_name: David A. Williams

given: David A.

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

repository_institution: University College London - UCL Discovery

repository_institution: California Digital Library - eScholarship

ipd_sharing_description: Bluebird bio is committed to transparency and appropriately de-identified patient-level datasets and supporting documents may be shared following attainment of applicable marketing approvals associated with this study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.

location_country:
United States
France
Argentina
Australia
Germany
United Kingdom

location_facility: Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center

location_facility: Boston Children's Hospital/Massachusetts General Hospital

location_facility: Great Ormond Street Hospital for Children NHS Foundation Trust

title: A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

summary: This trial will assess the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the participant following myeloablative conditioning.


title: Practice of Ventilation in Critically Ill Patients Without Acute Respiratory Distress Syndrome - An International Observational Study

acronym: PRoVENT

summary:
The purpose of this international, multicenter study is to determine ventilation practice and outcomes of intubated and ventilated intensive care unit (ICU) patients. Ventilation characteristics and outcomes will be compared between patients without the acute respiratory distress syndrome (ARDS), patients at risk for ARDS, and patients with mild, moderate or severe ARDS.

Participating centres worldwide will include adult patients undergoing mechanical ventilation in the ICU during a 7-day period. Patients data will be collected during the first 7 days in the ICU, or until ICU discharge. Follow up is until day 90. Primary endpoint is the tidal volume size used during mechanical ventilation. Secondary endpoints are development of ARDS in patients without ARDS at the onset of mechanical ventilation, worsening of ARDS in patients with ARDS at the onset of mechanical ventilation, pulmonary infection, other pulmonary complications, need for tracheostomy, extra-pulmonary complications, duration of ventilation, length of ICU and hospital stay, and ICU, hospital and 90-day mortality.

ReferenceType: background

ReferenceCitation: Hemmes SN, Serpa Neto A, Schultz MJ. Intraoperative ventilatory strategies to prevent postoperative pulmonary complications: a meta-analysis. Curr Opin Anaesthesiol. 2013 Apr;26(2):126-33. doi: 10.1097/ACO.0b013e32835e1242.

ReferenceCitation: Serpa Neto A, Cardoso SO, Manetta JA, Pereira VG, Espósito DC, Pasqualucci Mde O, Damasceno MC, Schultz MJ. Association between use of lung-protective ventilation with lower tidal volumes and clinical outcomes among patients without acute respiratory distress syndrome: a meta-analysis. JAMA. 2012 Oct 24;308(16):1651-9. doi: 10.1001/jama.2012.13730.

ReferenceCitation: Determann RM, Royakkers A, Wolthuis EK, Vlaar AP, Choi G, Paulus F, Hofstra JJ, de Graaff MJ, Korevaar JC, Schultz MJ. Ventilation with lower tidal volumes as compared with conventional tidal volumes for patients without acute lung injury: a preventive randomized controlled trial. Crit Care. 2010;14(1):R1. doi: 10.1186/cc8230. Epub 2010 Jan 7.

ReferenceCitation: Severgnini P, Selmo G, Lanza C, Chiesa A, Frigerio A, Bacuzzi A, Dionigi G, Novario R, Gregoretti C, de Abreu MG, Schultz MJ, Jaber S, Futier E, Chiaranda M, Pelosi P. Protective mechanical ventilation during general anesthesia for open abdominal surgery improves postoperative pulmonary function. Anesthesiology. 2013 Jun;118(6):1307-21. doi: 10.1097/ALN.0b013e31829102de.

ReferenceCitation: Serpa Neto A, Barbas CSV, Artigas A, et al. Rationale and Study Design of Provent-An International Multicenter Observational Study on Practice of Ventilation in Critically Ill Patients without ARDS. J Clin Trials 2013;3;[in press]

ReferenceCitation: Simonis FD, Barbas CSV, Artigas-Raventós A, Canet J, Determann RM, Anstey J, Hedenstierna G, Hemmes SNT, Hermans G, Hiesmayr M, Hollmann MW, Jaber S, Martin-Loeches I, Mills GH, Pearse RM, Putensen C, Schmid W, Severgnini P, Smith R, Treschan TA, Tschernko EM, Vidal Melo MF, Wrigge H, de Abreu MG, Pelosi P, Schultz MJ, Neto AS; PRoVENT investigators; PROVE Network investigators. Potentially modifiable respiratory variables contributing to outcome in ICU patients without ARDS: a secondary analysis of PRoVENT. Ann Intensive Care. 2018 Mar 21;8(1):39. doi: 10.1186/s13613-018-0385-7.

family: Simonis

full_name: Fabienne D. Simonis

given: Fabienne D.

family: Barbas

full_name: Carmen S. V. Barbas

given: Carmen S. V.

family: Artigas-Raventós

full_name: Antonio Artigas-Raventós

given: Antonio

family: Canet

full_name: Jaume Canet

given: Jaume

family: Determann

full_name: Rogier M. Determann

given: Rogier M.

family: Anstey

full_name: James Anstey

given: James

family: Hedenstierna

full_name: Goran Hedenstierna

given: Goran

family: Hemmes

full_name: Sabrine N. T. Hemmes

given: Sabrine N. T.

family: Hermans

full_name: Greet Hermans

given: Greet

family: Hiesmayr

full_name: Michael Hiesmayr

given: Michael

family: Hollmann

full_name: Markus W. Hollmann

given: Markus W.

family: Jaber

full_name: Samir Jaber

given: Samir

family: Martin-Loeches

full_name: Ignacio Martin-Loeches

given: Ignacio

family: Mills

full_name: Gary H. Mills

given: Gary H.

family: Pearse

full_name: Rupert M. Pearse

given: Rupert M.

family: Putensen

full_name: Christian Putensen

given: Christian

family: Schmid

full_name: Werner Schmid

given: Werner

family: Severgnini

full_name: Paolo Severgnini

given: Paolo

full_name: Roger Smith

given: Roger

family: Treschan

full_name: Tanja A. Treschan

given: Tanja A.

family: Tschernko

full_name: Edda M. Tschernko

given: Edda M.

family: Vidal Melo

full_name: Marcos F. Vidal Melo

given: Marcos F.

repository_institution: University of Melbourne - Minerva Access

repository_institution: Harvard University - Digital Access to Scholarship at Harvard (DASH)

repository_institution: KTH Royal Institute of Technology - KTH Publication Database DiVA

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

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Paris Descartes

ReferenceCitation: Neto AS, Barbas CSV, Simonis FD, Artigas-Raventós A, Canet J, Determann RM, Anstey J, Hedenstierna G, Hemmes SNT, Hermans G, Hiesmayr M, Hollmann MW, Jaber S, Martin-Loeches I, Mills GH, Pearse RM, Putensen C, Schmid W, Severgnini P, Smith R, Treschan TA, Tschernko EM, Melo MFV, Wrigge H, de Abreu MG, Pelosi P, Schultz MJ; PRoVENT; PROVE Network investigators. Epidemiological characteristics, practice of ventilation, and clinical outcome in patients at risk of acute respiratory distress syndrome in intensive care units from 16 countries (PRoVENT): an international, multicentre, prospective study. Lancet Respir Med. 2016 Nov;4(11):882-893. doi: 10.1016/S2213-2600(16)30305-8. Epub 2016 Oct 4.

location_country: Italy Austria Chile Brazil Netherlands France Belgium Spain Germany

location_facility: Hospital Clínico de la Pontificia Universidad Católica de Chile

location_facility: Hospital Universitari Germans Trias I Pujol


title: A Randomised, Double-blind, Placebo-controlled, Phase I/II Trial of RNActive®-Derived Cancer Vaccine (CV9104) in Asymptomatic or Minimally Symptomatic Patients With Metastatic Castrate-refractory Prostate Cancer

summary: The purpose of this study is to determine whether the new RNActive®-derived prostate cancer vaccine CV9104 prolongs survival in patients with asymptomatic or minimally symptomatic metastatic prostate cancer that is castrate resistant.

lead_sponsor: CureVac AG

location_country:
Sweden
Switzerland
United Kingdom
France
Poland
Spain
Germany
Czech Republic

location_facility: Centralny Szpital Kliniczny MSWiA, Klinika Onkologii I Hematologii

location_facility: Szpital Uniwersytecki, Katedra i Klinika Urologii i Onkologii Urologicznej

location_facility: Urologische Klinik und Poliklinik der Technischen Universität München Klinikum Rechts der Isar

location_facility: Clínica Universitaria de Navarra Departamento de Oncología

location_facility: Fakultní nemocnice Olomouc, Urologická klinika

location_facility: Nottingham City Hospital Department of Oncology

location_facility: Chirurgische Universitätsklinik Freiburg Klinik für Urologie

location_facility: Urologische Klinik Dr. Castingius München

location_facility: Skånes Universitetssjukhus Malmö Urologmottagningen

location_facility: Kantonsspital St. Gallen Department Innere Medizin Hämatologie Medizinische Onkologie

location_facility: Vivantes Klinikum Am Urban Klinik für Urologie

location_facility: Complejo Hospitalario Universitario Santiago Departamento de Oncología

location_facility: Krajská zdravotní, a.s. - Nemocnice Chomutov, o.z.Onkologické oddělení

location_facility: Hospital de Madrid Norte Sanchinarro Centro Integral Oncológico Clara Campal (CIOCC)

location_facility: Hospital Clínico Virgen de la Victoria Campus Universitario de Teatinos s/n

location_facility: Karolinska Universitetssjukhuset Solna Urologiska kliniken

location_facility: CHUV Centre Pluridisciplinaire d'Oncologie

location_facility: NZOZ Magodent, Centrum Medyczne Ostrobramska, Oncologii Klinicznej i Chemíoterapii

location_facility: Kantonsspital Graubünden Department Innere Medizin Hämatologie und Onkologie

location_facility: Szpital Sw. Elżbiety - Mokotowskie Centrum Medyczne

location_facility: Nationales Zentrum für Tumorerkrankungen Medizinische Onkologie

location_facility: Universitätsklinikum Münster Klinik und Poliklinik für Urologie

location_facility: Thomayerova nemocnice, Urologické oddělení

location_facility: Ortenau Klinikum Urologie und Kinderurologie

location_facility: Profesorskie Centrum Medyczne OPTIMUM Wrocław

location_facility: Multiscan, a.s, Oddělení klinické a radiační onkologie

location_facility: Instytut M. Curie-Skłodowskiej Centrum Onkologii

location_facility: Universitätsklinikum Aachen Klinik für Urologie

location_facility: Instituto Valenciano de Oncología Unidad de Investigación Clínica

location_facility: Universitätsspital Basel Medizinische Onkologie

location_facility: Krajská zdravotní, a.s. - Masarykova nemocnice Ústí nad Labem

location_facility: Universitätsklinikum Dresden Klinik und Poliklinik für Urologie

intervention_type: Biological


title: Clinical Study of the aura6000™ Targeted Hypoglossal Implantable Neurostimulation (THN) Sleep Therapy System

summary: The objective of the study is to confirm the safety and efficacy in patients utilizing the aura6000 System for the treatment of Obstructive Sleep Apnea (OSA).

ReferenceCitation: Zaidi FN, Meadows P, Jacobowitz O, Davidson TM. Tongue anatomy and physiology, the scientific basis for a novel targeted neurostimulation system designed for the treatment of obstructive sleep apnea. Neuromodulation. 2013 Jul-Aug;16(4):376-86; discussion 386. doi: 10.1111/j.1525-1403.2012.00514.x. Epub 2012 Aug 31. Review.

ReferenceCitation: Mwenge GB, Rombaux P, Dury M, Lengelé B, Rodenstein D. Targeted hypoglossal neurostimulation for obstructive sleep apnoea: a 1-year pilot study. Eur Respir J. 2013 Feb;41(2):360-7. doi: 10.1183/09031936.00042412. Epub 2012 May 17.

lead_sponsor: ImThera Medical, Inc.

location_country:
Italy
United States
France
Belgium
Germany

location_facility:
G.B. Morgagni- L. Pierantoni Hospital
Orange Regional Medical Center
Sharp Rees Stealy Medical Center
Porter Adventist Hospital
Pitié-Salpêtrière Hospital
Advanced Sleep Research
St. Joseph-Stift
Clinique Univ. Saint-Luc
Chicago ENT


title: Randomized Multi-Center Study Comparing No Drainage to Preoperative Biliary Drainage Using Metal Stents in Patients With Resectable Pancreatic or Periampullary Cancer

summary: The purpose of this study is to demonstrate that preoperative biliary drainage using self-expanding metal stents (SEMS) does not negatively impact overall surgical outcomes in patients undergoing pancreaticoduodenectomy for treatment of pancreatic or periampullary cancer.

status: Active, not recruiting

lead_sponsor: Boston Scientific Corporation

location_country:
Italy
India
Hong Kong
France
Japan
Belgium
Australia
China
United States

location_facility: Xijing Hospital of Digestive Diseases Fourth Military Medical University

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

location_facility: Fondazione Policlinico Universitario Agostino Gemelli


title: A Phase 2, Multicenter, Open-Label, Randomized Study to Investigate the Safety and Efficacy of GS-7977 and Ribavirin Administered for 48 Weeks in Patients Infected With Chronic HCV With Cirrhosis and Portal Hypertension With or Without Liver Decompensation

summary: This study will evaluate the antiviral efficacy of combination therapy with sofosbuvir (SOF) plus ribavirin (RBV) for 48 weeks in adults with compensated and decompensated chronic hepatitis C virus (HCV) infection. Approximately 50 adults will be randomized (1:1) to receive study drug for 48 weeks or take part in an untreated observational arm for the first 24 weeks followed by study drug for another 48 weeks.

lead_sponsor: Gilead Sciences

location_country:
France
Australia
New Zealand
Spain
United States


title: Noninvasive and Continuous Hemoglobin Monitoring for Surgical Blood Management

acronym: NACHO

summary:
This is a multi-center cluster-randomized trial with the following Specific Aims:

To evaluate if continuous noninvasive hemoglobin monitoring will reduce the RBC transfusions in patients undergoing surgeries associated with a significant risk of bleeding.
To evaluate if patients monitored with continuous noninvasive hemoglobin experience less frequent complications and shorter hospital stay compared with patients who are not being monitored with continuous noninvasive hemoglobin.

Accordingly, the study hypotheses are defined as follows:

The primary null hypothesis is that continuous noninvasive hemoglobin monitoring will not reduce the RBC transfusions in patients undergoing surgeries associated with a significant risk of bleeding.
The secondary hypothesis is that in patients monitored with continuous noninvasive hemoglobin, there will be earlier warning of critical drops in hemoglobin, and thus, there will be less frequent complications compared with patients who are not being monitored with continuous noninvasive hemoglobin.

status: Unknown status

primary_purpose: Diagnostic

lead_sponsor: Society for the Advancement of Blood Management, Inc

location_country:
Italy
United States
France
Japan

location_facility: Sapporo Medical University School of Medicine

location_facility: The Ohio State University Wexner Medical Center


title: A Multicenter, Open Label, Phase 1B Study of Escalating Doses of RO5045337 Administered Orally, With Cytarabine Administered A) Subcutaneously, or B) Intravenously, in Patients With Acute Myelogenous Leukemia (AML)

summary: This multi-center, open-label, Phase 1b study will evaluate the safety, pharmacokinetics and efficacy of RO5045337 in combination with cytarabine in patients with acute myelogenous leukemia. In Arm A, cohorts of previously untreated patients deemed unsuitable for standard induction therapy will receive escalating oral doses of RO5045377 and cytarabine 20 mg/m2 subcutaneously daily for Days 1 to 10 of each 28-day cycle. In Arm B, cohorts of patients who have relapsed or are refractory after at least one cytarabine/anthracycline containing regimen will receive escalating oral doses of RO5045377 on Days 1 to 5 and cytarabine 1 gm/m2 intravenously on Days 1 to 6 of each 28-day cycle. Patients will receive up to 4 cycles of therapy, patients in Arm A who achieve hematologic response may continue additional cycles until disease progression.

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
France
United States
United Kingdom
Canada


title: Xarelto® for Long-term and Initial Anticoagulation in Venous Thromboembolism (VTE)

acronym: XALIA

summary:
Following the findings of the clinical trials in drug development, this global non-interventional cohort field study will investigate rivaroxaban under clinical practice conditions in comparison with current standard of care for patients with acute deep vein thrombosis (DVT).

The main goal is to analyze long-term safety in the use of rivaroxaban in the treatment of acute DVT in routine clinical practice.

ReferenceCitation: Ageno W, Mantovani LG, Haas S, Kreutz R, Monje D, Schneider J, Bugge JP, Gebel M, Turpie AGG. Patient Management Strategies and Long-Term Outcomes in Isolated Distal Deep-Vein Thrombosis versus Proximal Deep-Vein Thrombosis: Findings from XALIA. TH Open. 2019 Mar 26;3(1):e85-e93. doi: 10.1055/s-0039-1683968. eCollection 2019 Jan.

name: Department of Medicine and Surgery, University of Insubria, Varese, Italy

family: Ageno

full_name: Walter Ageno

given: Walter

name: CESP-Center for Public Health Research, University of Milano-Bicocca, Monza, Italy

family: Mantovani

full_name: Lorenzo Mantovani

given: Lorenzo

name: Formerly Institute for Experimental Oncology and Therapy Research, Technical University of Munich, Munich, Germany

family: Haas

full_name: Sylvia Haas

given: Sylvia

name: Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Institute of Clinical Pharmacology and Toxicology, Berlin, Germany

family: Kreutz

full_name: Reinhold Kreutz

given: Reinhold

family: Monje

full_name: Danja Monje

given: Danja

family: Schneider

full_name: Jonas Schneider

given: Jonas

family: Bugge

full_name: Jörg-Peter Bugge

given: Jörg-Peter

family: Gebel

full_name: Martin Gebel

given: Martin

name: Department of Medicine, Hamilton Health Sciences, Hamilton, Ontario, Canada

family: Turpie

full_name: Alexander Turpie

given: Alexander

journal_name: TH Open

oa_colors: green other

repository_institution: Technical University Munich - mediaTUM – the media and publications repository of the Technical University Munich

evidence: oa repository (semantic scholar lookup)

version: submittedVersion

publisher: Georg Thieme Verlag KG

publisher_dissemination: Georg Thieme

publisher_group: Georg Thieme

publisher_normalized: Georg Thieme

ReferenceCitation: Ageno W, Mantovani LG, Haas S, Kreutz R, Monje D, Schneider J, van Eickels M, Gebel M, Zell E, Turpie AG. Safety and effectiveness of oral rivaroxaban versus standard anticoagulation for the treatment of symptomatic deep-vein thrombosis (XALIA): an international, prospective, non-interventional study. Lancet Haematol. 2016 Jan;3(1):e12-21. doi: 10.1016/S2352-3026(15)00257-4. Epub 2015 Dec 8.

full_name: Lorenzo G Mantovani

given: Lorenzo G

location_country:
Greece
Belgium
Denmark
Czech Republic
Ukraine
France
Norway
Moldova, Republic of
Canada
Slovenia
Sweden
Switzerland
Netherlands
Hungary
Portugal
Germany
United Kingdom
Italy
Austria
Israel
Spain


title: Xarelto® on Prevention of Stroke and Non-central Nervous System Systemic Embolism in Patients With Non-valvular Atrial Fibrillation

acronym: XANTUS

summary: This is an international observational study in patients with non-valvular atrial fibrillation who are prescribed rivaroxaban under routine treatment conditions to prevent stroke or non-central nervous system systemic embolism.

ReferenceCitation: Kirchhof P, Haas S, Amarenco P, Hess S, Lambelet M, van Eickels M, Turpie AGG, Camm AJ; XANTUS Investigators*. Impact of Modifiable Bleeding Risk Factors on Major Bleeding in Patients With Atrial Fibrillation Anticoagulated With Rivaroxaban. J Am Heart Assoc. 2020 Mar 3;9(5):e009530. doi: 10.1161/JAHA.118.009530. Epub 2020 Feb 21.

name: Institute of Cardiovascular Sciences UHB and Sandwell & West Birmingham Hospitals NHS Trusts University of Birmingham United Kingdom

name: University Heart and Vascular Center Hamburg Hamburg Germany

family: Kirchhof

full_name: Paulus Kirchhof

given: Paulus

name: Formerly Technical University of Munich Munich Germany

name: Department of Neurology and Stroke Centre Paris‐Diderot‐Sorbonne University Paris France

family: Amarenco

full_name: Pierre Amarenco

given: Pierre

name: Chrestos Concept GmbH & Co KG Essen Germany

family: Hess

full_name: Susanne Hess

given: Susanne

family: Lambelet

full_name: Marc Lambelet

given: Marc

family: van Eickels

full_name: Martin van Eickels

name: Department of Medicine McMaster University Hamilton Ontario Canada

full_name: Alexander G. G. Turpie

given: Alexander G. G.

name: Cardiovascular and Cell Sciences Research Institute and Cardiology Clinical Academic Group St George's, University of London London United Kingdom

family: Camm

full_name: A. John Camm

given: A. John

journal_name: Journal of the American Heart Association

repository_institution: University of Birmingham - University of Birmingham Research Portal

repository_normalized: Pure (Denmark)

repository_institution: St George's University of London - St George's Online Research Archive

version: acceptedVersion

repositories:
PubMed Central
Pure (Denmark)
openaccess.sgul.ac.uk

ReferenceCitation: Amarenco P, Haas S, Hess S, Kirchhof P, Lambelet M, Bach M, Turpie AGG, Camm AJ. Outcomes associated with non-recommended dosing of rivaroxaban: results from the XANTUS study. Eur Heart J Cardiovasc Pharmacother. 2019 Apr 1;5(2):70-79. doi: 10.1093/ehjcvp/pvy041.

name: Department of Neurology and Stroke Center, Paris-Diderot-Sorbonne University, 16 rue Henri Huchard, Paris, France

name: Formerly Technical University Munich, Arcisstraße 21, Munich, Germany

name: Global Medical Affairs, Bayer AG, Müllerstraße 178, Berlin, Germany

name: Institute of Cardiovascular Sciences, University of Birmingham and Sandwell & West Birmingham Hospitals NHS Trust, Edgbaston, Birmingham, UK

name: Chrestos Concept GmbH & Co KG, Girardetstraße 1-5, Essen, Germany

name: Department of Medicine, McMaster University, 1200 Main Street West, Hamilton, Ontario, Canada

name: Cardiovascular and Cell Sciences Research Institute, St George’s, Cranmer Terrace, University of London, London, UK

name: Department of Genetics and Imaging, Faculty of Medicine, Imperial College London, London, UK

journal_name: European Heart Journal - Cardiovascular Pharmacotherapy

ReferenceCitation: Kirchhof P, Radaideh G, Kim YH, Lanas F, Haas S, Amarenco P, Turpie AGG, Bach M, Lambelet M, Hess S, Camm AJ; Global XANTUS program Investigators. Global Prospective Safety Analysis of Rivaroxaban. J Am Coll Cardiol. 2018 Jul 10;72(2):141-153. doi: 10.1016/j.jacc.2018.04.058.

journal_name: Journal of the American College of Cardiology

ReferenceCitation: Camm AJ, Turpie AGG, Hess S, Amarenco P, Lambelet M, Haas S, van Eickels M, Kirchhof P; XANTUS Investigators. Outcomes after catheter ablation and cardioversion in patients with non-valvular atrial fibrillation: results from the prospective, observational XANTUS study. Europace. 2018 Jun 1;20(6):e87-e95. doi: 10.1093/europace/eux127.

name: Cardiovascular and Cell Sciences Research Institute, St George’s, University of London and Imperial College, Cranmer Terrace, London SW17 0RE, UK

name: Department of Medicine, McMaster University, Hamilton, Ontario, Canada

name: Department of Neurology and Stroke Center, Paris-Diderot-Sorbonne University, Paris, France

name: Chrestos Concept GmbH & Co KG, Essen, Germany

name: Institute of Cardiovascular Sciences, University of Birmingham, Sandwell and West Birmingham Hospitals and UHB NHS Trusts, Birmingham, UK

name: Department of Cardiovascular Medicine, University of Münster, Münster, Germany

ReferenceCitation: Camm AJ, Amarenco P, Haas S, Hess S, Kirchhof P, Kuhls S, van Eickels M, Turpie AG; XANTUS Investigators. XANTUS: a real-world, prospective, observational study of patients treated with rivaroxaban for stroke prevention in atrial fibrillation. Eur Heart J. 2016 Apr 7;37(14):1145-53. doi: 10.1093/eurheartj/ehv466. Epub 2015 Sep 1.

ReferenceCitation: Camm AJ, Amarenco P, Haas S, Hess S, Kirchhof P, van Eickels M, Turpie AG. XANTUS: rationale and design of a noninterventional study of rivaroxaban for the prevention of stroke in patients with atrial fibrillation. Vasc Health Risk Manag. 2014 Jul 17;10:425-34. doi: 10.2147/VHRM.S63298. eCollection 2014.

location_country:
Belgium
Russian Federation
Denmark
Czech Republic
Ukraine
France
Poland
Slovakia
Norway
Moldova, Republic of
Canada
Slovenia
Sweden
Netherlands
Hungary
Ireland
Portugal
Germany
United Kingdom
Austria
Israel


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:
Italy
United States
France
Belgium
Australia
Spain
Canada
United Kingdom

location_facility: University of Utah / Huntsman Cancer Institute Huntsman

location_facility: Massachusetts General Hospital Mass General 2

title: A Phase Ib/II Open-label, Multi-center Study of the Combination of MEK162 Plus AMG 479 (Ganitumab) in Adult Patients With Selected Advanced Solid Tumors

summary:
This is a multi-center, open-label, phase Ib/II study. First, the aim of the phase Ib part is to estimate the MTD(s) and/or to identify the recommended phase II dose(s) (RP2D) for the combination of MEK162 and AMG 479 (ganitumab), followed by the phase II part to assess the clinical efficacy and to further assess the safety of the combination in selected patient populations. The dose escalation part of the study will be guided by a Bayesian Logistic Regression Model (BLRM). At least 18 patients are expected to be enrolled in the dose escalation part.

Following MTD/ RP2D declaration, patients will be enrolled in three phase II arms to assess efficacy of the combination as well as to better understand the safety, tolerability, PK, antibody concentrations and PD of the combination at MTD/RP2D. Phase II arm 1 will consist of approximately 25 patients with KRAS-mutant colorectal adenocarcinoma. Phase II arm 2 will consist of approximately 20 patients with metastatic pancreatic adenocarcinoma. Phase II arm 3 will consist of approximately 28 patients with mutant BRAFV600 melanoma.

Patients will be treated until progression of disease, unacceptable toxicity develops, or withdrawal of informed consent, whichever occurs first. All patients will be followed up - at minimum patients must complete the safety follow-up assessments 30 days after the last dose of the study treatment.


title: An Exploratory Non-interventional Study of Ex-vivo Spiking Followed by Thromboelastography as a Method for Predicting the Efficacy of Recombinant Activated Human FVII (rFVIIa, NovoSeven®) in Adult Congenital Haemophilia A or B Patients With Inhibitors

summary: This trial is conducted in Europe and the United States of America (USA). The aim of this trial is to evaluate the basal and spiked TEG® (Thromboelastography) or ROTEM® (Thromboelastometry) profiles of frequently bleeding haemophilia subjects with inhibitors in a non-bleeding state.

lead_sponsor: Novo Nordisk A/S

location_country:
Denmark
United States
United Kingdom
France


title: Diabetes Attitudes Wishes and Needs (DAWN) 2 Study: A Multinational, Multi-stakeholder Survey Study of Psychosocial Issues in Diabetes and Patient-centred Diabetes Care

summary:
The study is conducted in Africa, Asia, Europe, Japan, North America and South America.

The multinational surveys will explore the experiences and unmet needs of people with diabetes, as well as those of family members of people with diabetes, and of healthcare professionals treating people with diabetes.

ReferenceType: result

ReferenceCitation: Peyrot M, Burns KK, Davies M, Forbes A, Hermanns N, Holt R, Kalra S, Nicolucci A, Pouwer F, Wens J, Willaing I, Skovlund SE. Diabetes Attitudes Wishes and Needs 2 (DAWN2): a multinational, multi-stakeholder study of psychosocial issues in diabetes and person-centred diabetes care. Diabetes Res Clin Pract. 2013 Feb;99(2):174-84. doi: 10.1016/j.diabres.2012.11.016. Epub 2012 Dec 27.

family: Peyrot

full_name: Mark Peyrot

given: Mark

family: Burns

full_name: Katharina Kovacs Burns

given: Katharina Kovacs

family: Davies

full_name: Melanie Davies

given: Melanie

family: Forbes

full_name: Angus Forbes

given: Angus

family: Hermanns

full_name: Norbert Hermanns

given: Norbert

family: Holt

full_name: Richard Holt

given: Richard

family: Kalra

full_name: Sanjay Kalra

given: Sanjay

family: Nicolucci

full_name: Antonio Nicolucci

given: Antonio

family: Pouwer

full_name: Frans Pouwer

given: Frans

family: Wens

full_name: Johan Wens

given: Johan

family: Willaing

full_name: Ingrid Willaing

given: Ingrid

family: Skovlund

full_name: Søren E. Skovlund

given: Søren E.

journal_name: Diabetes Research and Clinical Practice

oa_colors: green hybrid

evidence: open (via crossref license)

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repository_normalized: irua

repository_institution: King's College London - Research Portal

repository_institution: Tilburg University - Research portal

repositories:
Pure (Denmark)
irua
tilburguniversity.edu

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

title: Diabetes Attitudes Wishes and Needs 2 (DAWN2): A multinational, multi-stakeholder study of psychosocial issues in diabetes and person-centred diabetes care

ReferenceCitation: Nicolucci A, Kovacs Burns K, Holt RI, Comaschi M, Hermanns N, Ishii H, Kokoszka A, Pouwer F, Skovlund SE, Stuckey H, Tarkun I, Vallis M, Wens J, Peyrot M; DAWN2 Study Group. Diabetes Attitudes, Wishes and Needs second study (DAWN2™): cross-national benchmarking of diabetes-related psychosocial outcomes for people with diabetes. Diabet Med. 2013 Jul;30(7):767-77. doi: 10.1111/dme.12245. Erratum in: Diabet Med. 2013 Oct;30(10):1266.

name: Consorzio Mario Negri Sud; Santa Maria Imbaro; Italy

full_name: A. Nicolucci

given: A.

name: Edmonton Clinic Health Academy; University of Alberta; Edmonton; AB; Canada

name: Istituto Clinico Ligure di Alta Specialit; GVM Care and Research; Rapallo; Italy

name: Research Institute Diabetes; Diabetes Zentrum Mergentheim; Bad Mergentheim; Germany

name: Medical University of Warsaw; Warsaw; Poland

name: Tilburg University; Tilburg; the Netherlands

name: Penn State College of Medicine; Hershey; PA; USA

name: Dalhousie University; Halifax; NS; Canada

name: Loyola University Maryland; Baltimore; MD; USA

title: Diabetes Attitudes, Wishes and Needs second study (DAWN2™): Cross-national benchmarking of diabetes-related psychosocial outcomes for people with diabetes

ReferenceCitation: Kovacs Burns K, Nicolucci A, Holt RI, Willaing I, Hermanns N, Kalra S, Wens J, Pouwer F, Skovlund SE, Peyrot M; DAWN2 Study Group. Diabetes Attitudes, Wishes and Needs second study (DAWN2™): cross-national benchmarking indicators for family members living with people with diabetes. Diabet Med. 2013 Jul;30(7):778-88. doi: 10.1111/dme.12239.

name: Consorzio Mario Negri Sud; Santa Maria Imbaro; Chieti; Italy

name: Human Development and Health; Faculty of Medicine; University of Southampton; Southampton; UK

name: Steno Diabetes Center; Gentofte; Denmark

name: Bharti Hospital and BRIDE; Karnal; India

title: Diabetes Attitudes, Wishes and Needs second study (DAWN2™): Cross-national benchmarking indicators for family members living with people with diabetes

ReferenceCitation: Holt RI, Nicolucci A, Kovacs Burns K, Escalante M, Forbes A, Hermanns N, Kalra S, Massi-Benedetti M, Mayorov A, Menéndez-Torre E, Munro N, Skovlund SE, Tarkun I, Wens J, Peyrot M; DAWN2 Study Group. Diabetes Attitudes, Wishes and Needs second study (DAWN2™): cross-national comparisons on barriers and resources for optimal care--healthcare professional perspective. Diabet Med. 2013 Jul;30(7):789-98. doi: 10.1111/dme.12242.

name: Consorzio Mario Negri Sud; Santa Maria Imbaro; Chienti; Italy

name: Edmonton Clinic Health Academy; University of Alberta, Edmonton; AB; Canada

name: University of Guadalajara; Instituto Mexicano del Seguro Social; Guadalajara; Jalisco; Mexico

name: UBF for International Health Research; HIRS; Perugia; Italy

name: National Research Centre for Endocrinology; Moscow; Russia

name: Hospital Central de Asturias; Oviedo; Spain

title: Diabetes Attitudes, Wishes and Needs second study (DAWN2™): Cross-national comparisons on barriers and resources for optimal care-healthcare professional perspective

ReferenceCitation: Funnell M. Beyond the data: moving towards a new DAWN in diabetes. Diabet Med. 2013 Jul;30(7):765-6. doi: 10.1111/dme.12244.

name: Department of Medical Education; University of Michigan Medical School; Ann Arbor; MI; USA

ReferenceCitation: Peyrot M, Egede LE, Campos C, Cannon AJ, Funnell MM, Hsu WC, Ruggiero L, Siminerio LM, Stuckey HL. Ethnic differences in psychological outcomes among people with diabetes: USA results from the second Diabetes Attitudes, Wishes, and Needs (DAWN2) study. Curr Med Res Opin. 2014 Nov;30(11):2241-54. doi: 10.1185/03007995.2014.947023. Epub 2014 Aug 18.

name:
Loyola University Maryland
Baltimore, MDUSA

name:
Medical University of South Carolina
Charleston, SCUSA

name:
The Institute for Public Health and Education Research
New Braunfels, TXUSA

name: Sleep & Wellness Medical Associates Hamilton, NJUSA

name:
University of Michigan Medical School
Ann Arbor, MIUSA

name:
Joslin Health Solutions International, Joslin Diabetes Center, Harvard Medical School
Boston, MAUSA

name:
Institute for Health Research and Policy, University of Illinois at Chicago
Chicago, ILUSA

name:
University of Pittsburgh Diabetes Institute
Pittsburgh, PAUSA

name:
Penn State University College of Medicine
Hershey, PAUSA

ReferenceCitation: Peyrot M, Egede LE, Funnell MM, Hsu WC, Ruggiero L, Siminerio LM, Stuckey HL. US ethnic group differences in self-management in the 2nd diabetes attitudes, wishes and needs (DAWN2) study. J Diabetes Complications. 2018 Jun;32(6):586-592. doi: 10.1016/j.jdiacomp.2018.03.002. Epub 2018 Mar 8.

journal_name: Journal of Diabetes and its Complications

ReferenceCitation: Nefs G, Pouwer F. The role of hypoglycemia in the burden of living with diabetes among adults with diabetes and family members: results from the DAWN2 study in The Netherlands. BMC Public Health. 2018 Jan 18;18(1):156. doi: 10.1186/s12889-018-5064-y.

repositories_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

ReferenceCitation: Barnard KD, Holt RI, Dyson PA, Cummings MH, Kanumilli N, O'Neill S, Hall G. Could the Discrepancy in Perceived Emotional Care Received and Provided Be a Barrier to Active Diabetes Self-management? Insights From the Second Diabetes Attitudes, Wishes and Needs (DAWN2) Study. Diabetes Care. 2016 Feb;39(2):e20-1. doi: 10.2337/dc15-0674. Epub 2015 Dec 30.

ReferenceCitation: Peyrot M, Egede LE, Funnell MM, Hsu WC, Ruggiero L, Siminerio LM, Stuckey HL. Ethnic differences in family member diabetes involvement and psychological outcomes: results from the second Diabetes Attitudes, Wishes and Needs (DAWN2) study in the USA. Curr Med Res Opin. 2015;31(7):1297-307. doi: 10.1185/03007995.2015.1043251. Epub 2015 May 20.

name: aLoyola University Maryland, Baltimore, MD, USA

name: bMedical University of South Carolina, Charleston, SC, USA

name: cUniversity of Michigan Medical School, Ann Arbor, MI, USA

name: dJoslin Diabetes Center, Harvard Medical School, Boston, MA, USA

name: eInstitute for Health Research and Policy, School of Public Health, University of Illinois at Chicago, Chicago, IL, USA

name: fUniversity of Pittsburgh Diabetes Institute, Pittsburgh, PA, USA

name: gPenn State University College of Medicine, Hershey, PA, USA

location_country:
Italy
India
Mexico
Netherlands
Germany
Spain
France
Japan
Poland
Russian Federation
Algeria
China
Turkey
Denmark
United States
United Kingdom
Canada


title: Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry

summary:
This observational registry is conducted in Europe, Asia, Africa and the United States of America (USA).

The purpose of the registry is to evaluate the efficacy and safety of activated recombinant human factor VII (rFVIIa) during bleeding episodes and for the prevention of bleeding during invasive procedures/surgery in patients with Glanzmann's thrombasthenia (GT) with past or present refractoriness to platelet transfusions. Attention will be directed towards complications related to thrombo-embolic events and concomitant medications especially antifibrinolytics.

ReferenceCitation: Zotz RB, Poon MC, Di Minno G, D'Oiron R; Glanzmann Thrombasthenia Registry Investigators . The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes. TH Open. 2019 Sep 12;3(3):e286-e294. doi: 10.1055/s-0039-1696657. eCollection 2019 Jul.

name: Institute for Laboratory Medicine, Blood Coagulation and Transfusion Medicine (LBT), Düsseldorf, Germany

name: Department of Hemostasis, Hemotherapy and Transfusion Medicine, Heinrich Heine University Medical Centre, Düsseldorf, Germany

family: Zotz

full_name: Rainer B. Zotz

given: Rainer B.

name: Departments of Medicine, Pediatrics and Oncology, University of Calgary, Calgary, Alberta, Canada

name: Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Foothills Medical Centre, Calgary, Alberta, Canada

family: Poon

full_name: Man-Chiu Poon

given: Man-Chiu

name: Department of Clinical Medicine and Surgery, Regional Reference Center for Coagulation Disorders, Federico II University, Naples, Italy

family: Di Minno

full_name: Giovanni Di Minno

given: Giovanni

name: Center for Hemophilia and Rare Congenital Bleeding Disorders, University Hospitals Paris-Sud, AP-HP, Bicêtre Hospital, Le Kremlin-Bicêtre, France

family: D'Oiron

full_name: Roseline D'Oiron

given: Roseline

name: for the Glanzmann Thrombasthenia Registry Investigators

ReferenceCitation: Di Minno G, Zotz RB, d'Oiron R, Bindslev N, Di Minno MN, Poon MC; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes of non-surgical bleeding episodes in patients with Glanzmann thrombasthenia. Haematologica. 2015 Aug;100(8):1031-7. doi: 10.3324/haematol.2014.121475. Epub 2015 May 22.

full_name: G. Di Minno

given: G.

full_name: R. B. Zotz

given: R. B.

family: d'Oiron

full_name: R. d'Oiron

given: R.

family: Bindslev

full_name: N. Bindslev

given: N.

full_name: M. N. D. Di Minno

given: M. N. D.

full_name: M.-C. Poon

currency_apc_doaj: EUR

journal_name: Haematologica

license: publisher-specific license

license_normalized: publisher-specific

publisher: Ferrata Storti Foundation (Haematologica)

title: The international prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes in non-surgical bleeding episodes in Glanzmann thrombasthenia patients

ReferenceCitation: Poon MC, d'Oiron R, Zotz RB, Bindslev N, Di Minno MN, Di Minno G; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment and outcomes in surgical intervention. Haematologica. 2015 Aug;100(8):1038-44. doi: 10.3324/haematol.2014.121384. Epub 2015 May 22.

title: The international prospective Glanzmann Thrombasthenia Registry: treatment and outcomes in surgical intervention

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
Germany
Pakistan
France
Bulgaria
Hungary
Belgium
Algeria
Spain
United States
United Kingdom


title: A Clinical Evaluation of the Abbott Vascular ESPRIT BVS (Bioresorbable Vascular Scaffold) System for the Treatment of Subjects With Symptomatic Claudication From Occlusive Vascular Disease of the Superficial Femoral (SFA) or Common or External Iliac Arteries.

summary: The purpose of the ESPRIT I Clinical Investigation is to evaluate the safety and performance of the ESPRIT BVS in subjects with symptomatic claudication from occlusive vascular disease of the superficial femoral (SFA) or common or external iliac arteries.

ReferenceCitation: Lammer J, Bosiers M, Deloose K, Schmidt A, Zeller T, Wolf F, Lansink W, Sauguet A, Vermassen F, Lauwers G, Scheinert D, Popma JJ, McGreevy R, Rapoza R, Schwartz LB, Jaff MR. Bioresorbable Everolimus-Eluting Vascular Scaffold for Patients With Peripheral Artery Disease (ESPRIT I): 2-Year Clinical and Imaging Results. JACC Cardiovasc Interv. 2016 Jun 13;9(11):1178-87. doi: 10.1016/j.jcin.2016.02.051.

family: Lammer

full_name: Johannes Lammer

given: Johannes

family: Bosiers

full_name: Marc Bosiers

given: Marc

family: Deloose

full_name: Koen Deloose

given: Koen

family: Schmidt

full_name: Andrej Schmidt

given: Andrej

family: Zeller

full_name: Thomas Zeller

given: Thomas

family: Wolf

full_name: Florian Wolf

given: Florian

family: Lansink

full_name: Wouter Lansink

given: Wouter

family: Sauguet

full_name: Antoine Sauguet

given: Antoine

family: Vermassen

full_name: Frank Vermassen

given: Frank

family: Lauwers

full_name: Geert Lauwers

given: Geert

family: Scheinert

full_name: Dierk Scheinert

given: Dierk

family: Popma

full_name: Jeffrey J. Popma

given: Jeffrey J.

family: McGreevy

full_name: Robert McGreevy

given: Robert

family: Rapoza

full_name: Richard Rapoza

given: Richard

family: Schwartz

full_name: Lewis B. Schwartz

given: Lewis B.

family: Jaff

full_name: Michael R. Jaff

given: Michael R.

journal_name: JACC: Cardiovascular Interventions

license: elsevier-specific: oa user license

license_normalized: elsevier-specific

lead_sponsor: Abbott Medical Devices

location_country: Austria Germany Belgium France

location_facility:
Herzzentrum Bad Krozingen
Abbott Vascular International BVBA
Sint-Blasius Hospital
Gent University Hospital
Medical University of Vienna
Oost Limburg Ziekenhuis
Clinique Pasteur
Park-Krankenhaus Leipzig


title: Allogeneic Stem Cell Transplantation in Children and Adolescents With Acute Lymphoblastic Leukaemia

acronym: ALL-SCT-BFMi

summary:
With this protocol the ALL-SCT BFM international study group wants

to evaluate whether hematopoietic stem cell transplantation (HSCT) from matched family or unrelated donors (MD) is equivalent to the HSCT from matched sibling donors (MSD).
to evaluate the efficacy of hematopoietic stem cell transplantation (HSCT)from mismatched family or unrelated donors (MMD) as compared to HSCT from matched sibling donors or matched donors.
to determine whether therapy has been carried out according to the main HSCT protocol recommendations. The standardisation of the treatment options during HSCT from different donor types aims at the achievement of an optimal comparison of survival after HSCT with survival after chemotherapy only.
to prospectively evaluate and compare the incidence of acute and chronic Graft-versus-Host-Disease (GvHD) after HSCT from matched sibling donor (MSD), from matched donor (MD) and from mismatched donor (MMD).

ReferenceCitation: Peters C, Schrauder A, Schrappe M, von Stackelberg A, Stary J, Yaniv I, Gadner H, Klingebiel T; BFM Study Group, the IBFM-Study Group and the Paediatric Disease Working Party of the EBMT. Allogeneic haematopoietic stem cell transplantation in children with acute lymphoblastic leukaemia: the BFM/IBFM/EBMT concepts. Bone Marrow Transplant. 2005 Mar;35 Suppl 1:S9-11.

ReferenceCitation: Pulsipher MA, Peters C, Pui CH. High-risk pediatric acute lymphoblastic leukemia: to transplant or not to transplant? Biol Blood Marrow Transplant. 2011 Jan;17(1 Suppl):S137-48. doi: 10.1016/j.bbmt.2010.10.005. Review.

family: Pulsipher

full_name: Michael A. Pulsipher

given: Michael A.

family: Peters

full_name: Christina Peters

given: Christina

family: Pui

full_name: Ching-Hon Pui

given: Ching-Hon

journal_name: Biology of Blood and Marrow Transplantation

title: High-Risk Pediatric Acute Lymphoblastic Leukemia: To Transplant or Not to Transplant?

ReferenceCitation: Peters C, Schrappe M, von Stackelberg A, Schrauder A, Bader P, Ebell W, Lang P, Sykora KW, Schrum J, Kremens B, Ehlert K, Albert MH, Meisel R, Matthes-Martin S, Gungor T, Holter W, Strahm B, Gruhn B, Schulz A, Woessmann W, Poetschger U, Zimmermann M, Klingebiel T. Stem-cell transplantation in children with acute lymphoblastic leukemia: A prospective international multicenter trial comparing sibling donors with matched unrelated donors-The ALL-SCT-BFM-2003 trial. J Clin Oncol. 2015 Apr 10;33(11):1265-74. doi: 10.1200/JCO.2014.58.9747. Epub 2015 Mar 9.

name: Christina Peters, Susanne Matthes-Martin, and Ulrike Poetschger, St Anna Children's Hospital, Vienna, Austria; Martin Schrappe, University Medical Center Schleswig-Holstein and Christian-Albrechts-University Kiel; André Schrauder, Kinderarztpraxis am Aalborgring, Kiel; Arend von Stackelberg and Wolfram Ebell, Charité–Children's Hospital Berlin, Berlin; Peter Bader and Thomas Klingebiel, Johann Wolfgang Goethe University, Frankfurt; Peter Lang, University Hospital Tübingen, Tübingen; Karl-Walter Sykora...

family: Schrappe

full_name: Martin Schrappe

given: Martin

family: von Stackelberg

full_name: Arend von Stackelberg

given: Arend

family: Schrauder

full_name: André Schrauder

given: André

family: Bader

full_name: Peter Bader

given: Peter

family: Ebell

full_name: Wolfram Ebell

given: Wolfram

family: Lang

full_name: Peter Lang

family: Sykora

full_name: Karl-Walter Sykora

given: Karl-Walter

family: Schrum

full_name: Johanna Schrum

given: Johanna

family: Kremens

full_name: Bernhard Kremens

given: Bernhard

family: Ehlert

full_name: Karoline Ehlert

given: Karoline

family: Albert

full_name: Michael H. Albert

given: Michael H.

family: Meisel

full_name: Roland Meisel

given: Roland

publisher: American Society of Clinical Oncology (ASCO)

title: Stem-Cell Transplantation in Children With Acute Lymphoblastic Leukemia: A Prospective International Multicenter Trial Comparing Sibling Donors With Matched Unrelated Donors—The ALL-SCT-BFM-2003 Trial

ReferenceCitation: Tasian SK, Peters C. Targeted therapy or transplantation for paediatric ABL-class Ph-like acute lymphocytic leukaemia? Lancet Haematol. 2020 Dec;7(12):e858-e859. doi: 10.1016/S2352-3026(20)30369-0.

ReferenceCitation: Balduzzi A, Dalle JH, Wachowiak J, Yaniv I, Yesilipek A, Sedlacek P, Bierings M, Ifversen M, Sufliarska S, Kalwak K, Lankester A, Toporski J, Di Maio L, Glogova E, Poetschger U, Peters C. Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study. Biol Blood Marrow Transplant. 2019 Nov;25(11):2197-2210. doi: 10.1016/j.bbmt.2019.07.011. Epub 2019 Jul 15.

location_country:
Italy
Austria
Sweden
Netherlands
France
Poland
Israel
Slovakia
Turkey
Denmark
Czech Republic

location_facility: Department of Pediatric Hematology-Oncology and Pediatric Stem Cell Transplantation, Akdeniz University School of Medicine

location_facility: Department of Pediatric Bone Marrow Transplantation Unit, University Childrens´ Hospital

location_facility: Department of Pediatrics, Gülhane Military Medical Academy

location_facility: Department of Pediatric Hematology, Oncology and BMT, Istanbul School of Medicine

location_facility: Universitätsklinik für Kinder- und Jugendheilkunde, Klin. Abt. f. Hämato-Onkologie

location_facility: Department of Paediatric Haematology and Oncology, Wilhelmina Children's Hospital

location_facility: Children's University Hospital - Hematology - Oncology

location_facility: Radboud University - Nijmegen Medical Centre

location_facility: Department of Paediatric Haematology and Oncology HSCT-Unit

location_facility: Wroclaw Medical University, Dept. of Children Hematology and Oncology

location_facility: University Hospital, Collegium Medicum UMK, Pediatric Hematology and Oncology

location_facility: Dept. of Paediatrics - BMT Unit, School of Medicine, University of Ankara

location_facility: Department of Transplantation, University Children's Hospital

location_facility: Department of Pediadric Oncology, Hematology and Transplantology, University of Medical Sciences

location_facility: Schneider Children's Medical Center of Israel

location_facility: Clinica Pediatrica dell'Universita di Milano Bicocca, Hospitale San Gerardo

location_facility: Pediatric Immuno-Hematology Unit Robert Debré Hospital

location_facility: Department of Pediatric Oncology, Lund University Hospital


title: A Post Marketing, Multi-Center, Trial to Measure Human Anti-Human Antibodies (HAHA) to Soliris (Eculizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria

summary: How does long term treatment with Soliris affect HAHA in PNH patients?

lead_sponsor: Alexion Pharmaceuticals

location_country:
Italy
Switzerland
Netherlands
Germany
France
Ireland
Belgium
Australia
United States
United Kingdom

location_facility: Institut fur Klinische Transfusionmedizin und Immungenetick

location_facility: Azienda Ospediliera Universitatia Careggi


title: C1 Inhibitor Treatment Registry to Assess the Safety and Immunological Profile of Ruconest in the Treatment of HAE Attacks

summary: This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.

lead_sponsor: Pharming Technologies B.V.

location_country:
Italy
Sweden
North Macedonia
Croatia
Czechia
Bulgaria
France
Poland
Norway
Hungary
Slovakia
Germany
Slovenia

location_facility: Semmelweis University, 3rd Department of Internal Medicine


title: A Prospective, Randomized, Multicenter Study to Demonstrate the Superiority of the Barricaid® to Discectomy for Primary Lumbar Disc Herniation: Extended Follow-Up of the Barricaid® Annular Closure Device (ACD) Randomized Control Trial (RCT) Postmarket Cohort for Lumbar Disc Herniation and Interaction With Other Risk Factors

summary: A randomized study to demonstrate the superiority of the Barricaid® when used in conjunction with limited discectomy, compared to limited discectomy alone, with regard to preventing reherniation and the recurrence of pain or dysfunction.

ReferenceCitation: Thomé C, Klassen PD, Bouma GJ, Kuršumović A, Fandino J, Barth M, Arts M, van den Brink W, Bostelmann R, Hegewald A, Heidecke V, Vajkoczy P, Fröhlich S, Wolfs J, Assaker R, Van de Kelft E, Köhler HP, Jadik S, Eustacchio S, Hes R, Martens F; Annular Closure RCT Study Group. Annular closure in lumbar microdiscectomy for prevention of reherniation: a randomized clinical trial. Spine J. 2018 Dec;18(12):2278-2287. doi: 10.1016/j.spinee.2018.05.003. Epub 2018 May 3.

family: Thomé

full_name: Claudius Thomé

given: Claudius

family: Klassen

full_name: Peter Douglas Klassen

given: Peter Douglas

family: Bouma

full_name: Gerrit Joan Bouma

given: Gerrit Joan

family: Kuršumović

full_name: Adisa Kuršumović

given: Adisa

family: Fandino

full_name: Javier Fandino

given: Javier

family: Barth

full_name: Martin Barth

family: Arts

full_name: Mark Arts

given: Mark

family: van den Brink

full_name: Wimar van den Brink

given: Wimar

family: Bostelmann

full_name: Richard Bostelmann

given: Richard

family: Hegewald

full_name: Aldemar Hegewald

given: Aldemar

family: Heidecke

full_name: Volkmar Heidecke

given: Volkmar

family: Vajkoczy

full_name: Peter Vajkoczy

family: Fröhlich

full_name: Susanne Fröhlich

given: Susanne

family: Wolfs

full_name: Jasper Wolfs

given: Jasper

family: Assaker

full_name: Richard Assaker

family: Van de Kelft

full_name: Erik Van de Kelft

given: Erik

family: Köhler

full_name: Hans-Peter Köhler

given: Hans-Peter

family: Jadik

full_name: Senol Jadik

given: Senol

family: Eustacchio

full_name: Sandro Eustacchio

given: Sandro

family: Hes

full_name: Robert Hes

given: Robert

family: Martens

full_name: Frederic Martens

given: Frederic

journal_name: The Spine Journal

repository_institution: Augsburg University - OPUS - Augsburg University Publication Server

ReferenceCitation: Martens F, Vajkoczy P, Jadik S, Hegewald A, Stieber J, Hes R. Patients at the Highest Risk for Reherniation Following Lumbar Discectomy in a Multicenter Randomized Controlled Trial. JB JS Open Access. 2018 Apr 16;3(2):e0037. doi: 10.2106/JBJS.OA.17.00037. eCollection 2018 Jun 28.

ReferenceCitation: Klassen PD, Hsu WK, Martens F, Inzana JA, van den Brink WA, Groff MW, Thomé C. Post-lumbar discectomy reoperations that are associated with poor clinical and socioeconomic outcomes can be reduced through use of a novel annular closure device: results from a 2-year randomized controlled trial. Clinicoecon Outcomes Res. 2018 Jun 26;10:349-357. doi: 10.2147/CEOR.S164129. eCollection 2018.

ReferenceCitation: Ament J, Thaci B, Yang Z, Kulubya E, Hsu W, Bouma G, Kim KD. Cost-effectiveness of a Bone-anchored Annular Closure Device Versus Conventional Lumbar Discectomy in Treating Lumbar Disc Herniations. Spine (Phila Pa 1976). 2019 Jan 1;44(1):5-16. doi: 10.1097/BRS.0000000000002746.

ReferenceCitation: Kuršumović A, Kienzler JC, Bouma GJ, Bostelmann R, Heggeness M, Thomé C, Miller LE, Barth M; Annular Closure RCT study group. Morphology and Clinical Relevance of Vertebral Endplate Changes Following Limited Lumbar Discectomy With or Without Bone-anchored Annular Closure. Spine (Phila Pa 1976). 2018 Oct 15;43(20):1386-1394. doi: 10.1097/BRS.0000000000002632.

ReferenceCitation: Klassen PD, Bernstein DT, Köhler HP, Arts MP, Weiner B, Miller LE, Thomé C. Bone-anchored annular closure following lumbar discectomy reduces risk of complications and reoperations within 90 days of discharge. J Pain Res. 2017 Aug 26;10:2047-2055. doi: 10.2147/JPR.S144500. eCollection 2017. Erratum in: J Pain Res. 2017 Dec 06;10 :2739.

ReferenceCitation: Kienzler JC, Fandino J, Van de Kelft E, Eustacchio S, Bouma GJ; Barricaid® Annular Closure RCT Study Group. Risk factors for early reherniation after lumbar discectomy with or without annular closure: results of a multicenter randomized controlled study. Acta Neurochir (Wien). 2021 Jan;163(1):259-268. doi: 10.1007/s00701-020-04505-4. Epub 2020 Oct 21.

name: The Barricaid® Annular Closure RCT Study Group

ReferenceCitation: van den Brink W, Flüh C, Miller LE, Klassen PD, Bostelmann R. Lumbar disc reherniation prevention with a bone-anchored annular closure device: 1-year results of a randomized trial. Medicine (Baltimore). 2019 Nov;98(44):e17760. doi: 10.1097/MD.0000000000017760.

ReferenceCitation: Kienzler JC, Klassen PD, Miller LE, Assaker R, Heidecke V, Fröhlich S, Thomé C; Annular Closure RCT Study Group. Three-year results from a randomized trial of lumbar discectomy with annulus fibrosus occlusion in patients at high risk for reherniation. Acta Neurochir (Wien). 2019 Jul;161(7):1389-1396. doi: 10.1007/s00701-019-03948-8. Epub 2019 May 15.

name: On behalf of the Annular Closure RCT Study Group

location_country: Austria Switzerland Netherlands France Belgium Germany

location_facility: Orthopädische Klinik und Poliklinik - Universitätsmedizin Rostock

location_facility: Neurochirurgische Universitätsklinik Knappschafts-Krankenhaus Bochum-Langendreer


title: AZA PH GL 2003 CL 001 - Extension A Multicenter, Randomized, Open-label, Parallel-group, Phase 3 Trial of Subcutaneous Azacitidine Plus Best Supportive Care Versus Conventional Care Regimens Plus Best Supportive Care for the Treatment of Myelodysplastic Syndromes (MDS)

summary: At the conclusion of study AZA PH GL 2003 CL 001 (NCT00071799), eligible participants could be enrolled in an optional extension phase in order to continue treatment with azacitidine until it became commercially available; the continued treatment was for ethical and safety reasons only and not to provide additional efficacy data.

ReferenceCitation: Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Epub 2009 Feb 21.

family: Fenaux

full_name: Pierre Fenaux

given: Pierre

family: Mufti

full_name: Ghulam J Mufti

given: Ghulam J

family: Hellstrom-Lindberg

full_name: Eva Hellstrom-Lindberg

given: Eva

family: Santini

full_name: Valeria Santini

given: Valeria

family: Finelli

full_name: Carlo Finelli

given: Carlo

family: Giagounidis

full_name: Aristoteles Giagounidis

given: Aristoteles

family: Schoch

full_name: Robert Schoch

family: Gattermann

full_name: Norbert Gattermann

given: Norbert

family: Sanz

full_name: Guillermo Sanz

given: Guillermo

family: List

full_name: Alan List

given: Alan

family: Gore

full_name: Steven D Gore

given: Steven D

family: Seymour

full_name: John F Seymour

given: John F

family: Bennett

full_name: John M Bennett

given: John M

family: Byrd

full_name: John Byrd

given: John

family: Backstrom

full_name: Jay Backstrom

given: Jay

family: Zimmerman

full_name: Linda Zimmerman

given: Linda

family: McKenzie

full_name: David McKenzie

given: David

family: Beach

full_name: CL Beach

given: CL

family: Silverman

full_name: Lewis R Silverman

given: Lewis R

journal_name: The Lancet Oncology

oa_host_type: repository

ReferenceCitation: Fenaux P, Mufti GJ, Hellström-Lindberg E, Santini V, Gattermann N, Germing U, Sanz G, List AF, Gore S, Seymour JF, Dombret H, Backstrom J, Zimmerman L, McKenzie D, Beach CL, Silverman LR. Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia. J Clin Oncol. 2010 Feb 1;28(4):562-9. doi: 10.1200/JCO.2009.23.8329. Epub 2009 Dec 21.

name: From the Avicenne, Assistance Publique-Hôpitaux de Paris (AP-HP), Université Paris XIII, Bobigny; Hôpital Saint-Louis, AP-HP, Université Paris 7, Paris, France; King's College Hospital, London, United Kingdom; Karolinska University Hospital (Huddinge), Stockholm, Sweden; Azienda Ospedaliera Universitaria Careggi, Florence, Italy; Universitätsklinikum Düsseldorf, Düsseldorf, Germany; Hospital Universitario La Fe, Valencia, Spain; Peter MacCallum Cancer Centre and University of Melbourne, Melbourne,...

publisher_dissemination: American Society of Clinical Oncology (ASCO)

title: Azacitidine Prolongs Overall Survival Compared With Conventional Care Regimens in Elderly Patients With Low Bone Marrow Blast Count Acute Myeloid Leukemia

ReferenceCitation: Gore SD, Fenaux P, Santini V, Bennett JM, Silverman LR, Seymour JF, Hellström-Lindberg E, Swern AS, Beach CL, List AF. A multivariate analysis of the relationship between response and survival among patients with higher-risk myelodysplastic syndromes treated within azacitidine or conventional care regimens in the randomized AZA-001 trial. Haematologica. 2013 Jul;98(7):1067-72. doi: 10.3324/haematol.2012.074831. Epub 2013 Apr 12.

repository_institution: University of Florence - Florence Research

publisher_dissemination: Ferrata Storti Foundation (Haematologica)

ReferenceCitation: Savona MR, Kolibaba K, Conkling P, Kingsley EC, Becerra C, Morris JC, Rifkin RM, Laille E, Kellerman A, Ukrainskyj SM, Dong Q, Skikne BS. Extended dosing with CC-486 (oral azacitidine) in patients with myeloid malignancies. Am J Hematol. 2018 Oct;93(10):1199-1206. doi: 10.1002/ajh.25216. Epub 2018 Sep 3.

ReferenceCitation: Seymour JF, Fenaux P, Silverman LR, Mufti GJ, Hellström-Lindberg E, Santini V, List AF, Gore SD, Backstrom J, McKenzie D, Beach CL. Effects of azacitidine compared with conventional care regimens in elderly (≥ 75 years) patients with higher-risk myelodysplastic syndromes. Crit Rev Oncol Hematol. 2010 Dec;76(3):218-27. doi: 10.1016/j.critrevonc.2010.04.005. Epub 2010 May 6.

title: Effects of azacitidine compared with conventional care regimens in elderly (≥75 years) patients with higher-risk myelodysplastic syndromes

ReferenceCitation: Garcia-Manero G, Scott BL, Cogle CR, Boyd TE, Kambhampati S, Hetzer J, Dong Q, Kumar K, Ukrainskyj SM, Beach CL, Skikne BS. CC-486 (oral azacitidine) in patients with myelodysplastic syndromes with pretreatment thrombocytopenia. Leuk Res. 2018 Sep;72:79-85. doi: 10.1016/j.leukres.2018.08.001. Epub 2018 Aug 3.

ReferenceCitation: Ma X, Steensma DP, Scott BL, Kiselev P, Sugrue MM, Swern AS. Selection of patients with myelodysplastic syndromes from a large electronic medical records database and a study of the use of disease-modifying therapy in the United States. BMJ Open. 2018 Jul 23;8(7):e019955. doi: 10.1136/bmjopen-2017-019955.

ReferenceCitation: de Lima M, Oran B, Champlin RE, Papadopoulos EB, Giralt SA, Scott BL, William BM, Hetzer J, Laille E, Hubbell B, Skikne BS, Craddock C. CC-486 Maintenance after Stem Cell Transplantation in Patients with Acute Myeloid Leukemia or Myelodysplastic Syndromes. Biol Blood Marrow Transplant. 2018 Oct;24(10):2017-2024. doi: 10.1016/j.bbmt.2018.06.016. Epub 2018 Jun 20.

ReferenceCitation: Wehmeyer J, Zaiss M, Losem C, Schmitz S, Niemeier B, Harde J, Hannig CV, Harich HD, Müller J, Klausmann M, Tessen HW, Potthoff K. Impact of performance status and transfusion dependency on outcome of patients with myelodysplastic syndrome, acute myeloid leukemia and chronic myelomonocytic leukemia treated with azacitidine (PIAZA study). Eur J Haematol. 2018 Dec;101(6):766-773. doi: 10.1111/ejh.13160. Epub 2018 Oct 25.

ReferenceCitation: Platzbecker U, Middeke JM, Sockel K, Herbst R, Wolf D, Baldus CD, Oelschlägel U, Mütherig A, Fransecky L, Noppeney R, Bug G, Götze KS, Krämer A, Bochtler T, Stelljes M, Groth C, Schubert A, Mende M, Stölzel F, Borkmann C, Kubasch AS, von Bonin M, Serve H, Hänel M, Dührsen U, Schetelig J, Röllig C, Kramer M, Ehninger G, Bornhäuser M, Thiede C. Measurable residual disease-guided treatment with azacitidine to prevent haematological relapse in patients with myelodysplastic syndrome and acute myeloid leukaemia (RELAZA2): an open-label, multicentre, phase 2 trial. Lancet Oncol. 2018 Dec;19(12):1668-1679. doi: 10.1016/S1470-2045(18)30580-1. Epub 2018 Nov 12.

ReferenceCitation: Boutault R, Peterlin P, Boubaya M, Sockel K, Chevallier P, Garnier A, Guillaume T, Le Bourgeois A, Debord C, Godon C, Le Bris Y, Theisen O, Kroschinsky F, Moreau P, Béné MC, Platzbecker U, Eveillard M. A novel complete blood count-based score to screen for myelodysplastic syndrome in cytopenic patients. Br J Haematol. 2018 Dec;183(5):736-746. doi: 10.1111/bjh.15626. Epub 2018 Nov 8.

ReferenceCitation: Wenk C, Garz AK, Grath S, Huberle C, Witham D, Weickert M, Malinverni R, Niggemeyer J, Kyncl M, Hecker J, Pagel C, Mulholland CB, Müller-Thomas C, Leonhardt H, Bassermann F, Oostendorp RAJ, Metzeler KH, Buschbeck M, Götze KS. Direct modulation of the bone marrow mesenchymal stromal cell compartment by azacitidine enhances healthy hematopoiesis. Blood Adv. 2018 Dec 11;2(23):3447-3461. doi: 10.1182/bloodadvances.2018022053.

ReferenceCitation: Zeidan AM, Klink AJ, McGuire M, Feinberg B. Treatment sequence of lenalidomide and hypomethylating agents and the impact on clinical outcomes for patients with myelodysplastic syndromes. Leuk Lymphoma. 2019 Aug;60(8):2050-2055. doi: 10.1080/10428194.2018.1551538. Epub 2019 Jan 14.

ReferenceCitation: Leung KK, Nguyen A, Shi T, Tang L, Ni X, Escoubet L, MacBeth KJ, DiMartino J, Wells JA. Multiomics of azacitidine-treated AML cells reveals variable and convergent targets that remodel the cell-surface proteome. Proc Natl Acad Sci U S A. 2019 Jan 8;116(2):695-700. doi: 10.1073/pnas.1813666116. Epub 2018 Dec 24.

ReferenceCitation: Craddock C, Slade D, De Santo C, Wheat R, Ferguson P, Hodgkinson A, Brock K, Cavenagh J, Ingram W, Dennis M, Malladi R, Siddique S, Mussai F, Yap C. Combination Lenalidomide and Azacitidine: A Novel Salvage Therapy in Patients Who Relapse After Allogeneic Stem-Cell Transplantation for Acute Myeloid Leukemia. J Clin Oncol. 2019 Mar 1;37(7):580-588. doi: 10.1200/JCO.18.00889. Epub 2019 Jan 17.

ReferenceCitation: Silverman LR, Fenaux P, Mufti GJ, Santini V, Hellström-Lindberg E, Gattermann N, Sanz G, List AF, Gore SD, Seymour JF. Continued azacitidine therapy beyond time of first response improves quality of response in patients with higher-risk myelodysplastic syndromes. Cancer. 2011 Jun 15;117(12):2697-702. doi: 10.1002/cncr.25774. Epub 2011 Jan 10.

location_country:
Italy
Netherlands
France
Bulgaria
Greece
Poland
Hungary
Australia
Spain
Germany
United Kingdom


title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study of the Safety, Tolerability and Efficacy of E2007 in Parkinson's Disease Patients With "Wearing Off" Motor Fluctuations and "On" Period Dyskinesias

summary: The purpose of this study is to investigate the safety, tolerability and efficacy of E2007 in Parkinson's Disease patients who have "wearing off" motor fluctuations and "on" period dyskenisias.

lead_sponsor: Eisai Limited

location_country:
Italy
Serbia
France
Spain
Germany
Czech Republic

location_facility: Department of Neurology, Regional Hospital Pardubice

location_facility: Zentralkrankenhaus Reinkenheide Neurologische Klinik

location_facility: Hospital Clinic I Provincial de Barcelona

location_facility: Reparto di Neurologia - Ospedale Misericordia

location_facility: Clinic of Neurology, Faculty Hospital Olomouc

location_facility: First Faculty of Medicine Charles University

location_facility: Universit?tsklinikum Rostock Klinik f?r Neurologie

location_facility: Universit?tskrankenh aus Hamburg Eppendorf

location_facility: Parkinson's Competence Network Germany Dept. of Neurology - Philipps-University Marburg

location_facility: Dept. of Neurology - Second Faculty of Medicine Charles University

location_facility: Unit? Operativa Parkinson e Disordini del Movimento

location_facility: Centre D'Investigation Clinique Pavillon Riser - Hopital Purpan

location_facility: Klinikum der Heinrich-Heine- Universit?t

location_facility: Humboldt Universit?t Charite Neurologische Klinik

location_facility: Klinikum der Friedrich-Wilhelms- Univerit?t Bonn


ReferenceCitation: Violari A, Masenya M, Blanche S, Vanveggel S, Hufkens V, Chetty P, Opsomer M. The DIANA Study: Continued Access to Darunavir/Ritonavir (DRV/r) and Long-Term Safety Follow-Up in HIV-1-Infected Pediatric Patients Aged 3 to < 18 Years. Drug Saf. 2021 Apr;44(4):439-446. doi: 10.1007/s40264-020-01032-0. Epub 2020 Dec 24.

family: Violari

full_name: Avy Violari

given: Avy

family: Masenya

full_name: Maysseb Masenya

given: Maysseb

family: Blanche

full_name: Stephane Blanche

given: Stephane

family: Vanveggel

full_name: Simon Vanveggel

given: Simon

family: Hufkens

full_name: Veerle Hufkens

given: Veerle

family: Chetty

full_name: Polan Chetty

given: Polan

family: Opsomer

full_name: Magda Opsomer

given: Magda

journal_name: Drug Safety

lead_sponsor: Janssen Sciences Ireland UC

location_country:
Brazil
South Africa
India
France
Argentina
Spain
United Kingdom
Ukraine

title: Continued Access to DRV/Rtv in HIV-1 Infected Children and Adolescents (Rollover Patients From C212, C228, C230)

summary: The primary objective of this study is to continue to provide Darunavir (DRV) to pediatric patients who previously received DRV in any of three pediatric clinical studies sponsored by Tibotec Pharmaceuticals and continue to benefit from using it, in countries where DRV is not yet commercially available for the pediatric patient, is not reimbursed or cannot be accessed through another source (like access program or government program).


title: A Phase I/II Trial of Temsirolimus Plus Neratinib for Patients With Metastatic HER2-Amplified or Triple Negative Breast Cancer

summary: This is an open-label, single arm, multi-center, multi-national, adaptive design, dose-escalation Phase 1/2 study to determine the maximum tolerated dose (MTD) of temsirolimus with daily neratinib, and to determine the safety and efficacy of this combination when given to patients with advanced breast carcinoma, specifically trastuzumab-refractory HER2-amplified disease or triple-negative disease.

lead_sponsor: Puma Biotechnology, Inc.

location_country:
Hong Kong
France
Spain
Denmark
United States
United Kingdom

location_facility: Hospital Universitari Arnau de Vilanova de Lleida

location_facility: Weill Cornell Medical College - New York - Presbyterian Hospital

location_facility: Memorial Sloan Kettering Cancer Center (MSKCC)

location_facility: Hospital Universitario Sant Joan de Reus

location_facility: Edinburgh Cancer Center, Western General Hospital


title: NordiNet® International Outcome Study-Observational Prospective Study on Patients Treated With Norditropin®

summary: This observational study is conducted globally. The aim of the study is to investigate the effectiveness and safety of real-life treatment with Norditropin®. The study population will consist of children and adults who are on treatment with Norditropin® in accordance with normal clinical practice.

ReferenceCitation: Weber MM, Biller BM, Pedersen BT, Pournara E, Christiansen JS, Höybye C. The effect of growth hormone (GH) replacement on blood glucose homeostasis in adult nondiabetic patients with GH deficiency: real-life data from the NordiNet(®) International Outcome Study. Clin Endocrinol (Oxf). 2017 Feb;86(2):192-198. doi: 10.1111/cen.13256. Epub 2016 Nov 21.

name: Department of Endocrinology and Metabolism; Medical Clinic; University of Mainz; Mainz Germany

family: Weber

full_name: Matthias M. Weber

given: Matthias M.

name: Neuroendocrine Unit; Massachusetts General Hospital; Boston MA USA

family: Biller

full_name: Beverly M.K. Biller

given: Beverly M.K.

name: Epidemiology; Novo Nordisk A/S; Søborg Denmark

family: Pedersen

full_name: Birgitte Tønnes Pedersen

given: Birgitte Tønnes

name: Novo Nordisk Health Care AG; Zurich Switzerland

family: Pournara

full_name: Effie Pournara

given: Effie

name: Department of Endocrinology and Diabetes; MEA, Aarhus University Hospital, NBG; Aarhus Denmark

family: Christiansen

full_name: Jens Sandahl Christiansen

given: Jens Sandahl

name: Department of Endocrinology, Metabolism and Diabetology; Department of Molecular Medicine and Surgery; Karolinska Institute; Karolinska University Hospital; Stockholm Sweden

family: Höybye

full_name: Charlotte Höybye

given: Charlotte

journal_name: Clinical Endocrinology

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: The effect of growth hormone (GH) replacement on blood glucose homeostasis in adult nondiabetic patients with GH deficiency: real-life data from the NordiNet®International Outcome Study

ReferenceCitation: Sävendahl L, Pournara E, Pedersen BT, Blankenstein O. Is safety of childhood growth hormone therapy related to dose? Data from a large observational study. Eur J Endocrinol. 2016 May;174(5):681-91. doi: 10.1530/EJE-15-1017. Epub 2016 Feb 22.

family: Sävendahl

full_name: Lars Sävendahl

given: Lars

family: Blankenstein

full_name: Oliver Blankenstein

given: Oliver

journal_name: European Journal of Endocrinology

evidence: open (via free pdf)

publisher: Bioscientifica

publisher_dissemination: Bioscientifica

publisher_group: Bioscientifica

publisher_normalized: Bioscientifica

ReferenceCitation: Lee PA, Sävendahl L, Oliver I, Tauber M, Blankenstein O, Ross J, Snajderova M, Rakov V, Pedersen BT, Christesen HT. Comparison of response to 2-years' growth hormone treatment in children with isolated growth hormone deficiency, born small for gestational age, idiopathic short stature, or multiple pituitary hormone deficiency: combined results from two large observational studies. Int J Pediatr Endocrinol. 2012 Jul 12;2012(1):22. doi: 10.1186/1687-9856-2012-22.

family: Lee

full_name: Peter A Lee

given: Peter A

family: Oliver

full_name: Isabelle Oliver

given: Isabelle

family: Tauber

full_name: Maithé Tauber

given: Maithé

family: Ross

journal_name: International Journal of Pediatric Endocrinology

repository_institution: Thomas Jefferson University - Jefferson Digital Commons

title: Comparison of response to 2-years’ growth hormone treatment in children with isolated growth hormone deficiency, born small for gestational age, idiopathic short stature, or multiple pituitary hormone deficiency: combined results from two large observational studies

ReferenceCitation: Höybye C, Sävendahl L, Christesen HT, Lee P, Pedersen BT, Schlumpf M, Germak J, Ross J. The NordiNet® International Outcome Study and NovoNet® ANSWER Program®: rationale, design, and methodology of two international pharmacoepidemiological registry-based studies monitoring long-term clinical and safety outcomes of growth hormone therapy (Norditropin®). Clin Epidemiol. 2013 Apr 26;5:119-27. doi: 10.2147/CLEP.S42602. Print 2013.

ReferenceCitation: Sävendahl L, Blankenstein O, Oliver I, Christesen HT, Lee P, Pedersen BT, Rakov V, Ross J. Gender influences short-term growth hormone treatment response in children. Horm Res Paediatr. 2012;77(3):188-94. doi: 10.1159/000337570. Epub 2012 Apr 12.

title: Gender Influences Short-Term Growth Hormone Treatment Response in Children

ReferenceCitation: Blankenstein O, Snajderova M, Blair J, Pournara E, Pedersen BT, Petit IO. Real-life GH dosing patterns in children with GHD, TS or born SGA: a report from the NordiNet® International Outcome Study. Eur J Endocrinol. 2017 Aug;177(2):145-155. doi: 10.1530/EJE-16-1055. Epub 2017 May 18.

name: 1Center for Chronic Sick Children, Institute for Experimental Paediatric Endocrinology, Charité – Universitätsmedizin Berlin, Berlin, Germany

name: 22nd Faculty of Medicine, Charles University and University Hospital Motol, Prague, Czech Republic

name: 3Alder Hey Children’s NHS Foundation Trust, Liverpool, UK

name: 4Novo Nordisk Health Care AG, Zurich, Switzerland

name: 5Epidemiology, Novo Nordisk A/S, Søborg, Denmark

name: 6Department of Paediatric Endocrinology, Hôpital des Enfants, Toulouse, France

ReferenceCitation: Christesen HT, Pedersen BT, Pournara E, Petit IO, Júlíusson PB. Short Stature: Comparison of WHO and National Growth Standards/References for Height. PLoS One. 2016 Jun 9;11(6):e0157277. doi: 10.1371/journal.pone.0157277. eCollection 2016.

repository_institution: University of Southern Denmark - University of Southern Denmark Research Portal

ReferenceCitation: Biller BMK, Höybye C, Carroll P, Gordon MB, Birkegård AC, Kelepouris N, Nedjatian N, Weber MM. Pregnancy outcomes in women receiving growth hormone replacement therapy enrolled in the NordiNet® International Outcome Study (IOS) and the American Norditropin® Studies: Web-Enabled Research (ANSWER) Program. Pituitary. 2021 Aug;24(4):611-621. doi: 10.1007/s11102-021-01138-3. Epub 2021 Mar 12.

ReferenceCitation: Sävendahl L, Polak M, Backeljauw P, Blair JC, Miller BS, Rohrer TR, Hokken-Koelega A, Pietropoli A, Kelepouris N, Ross J. Long-Term Safety of Growth Hormone Treatment in Childhood: Two Large Observational Studies: NordiNet IOS and ANSWER. J Clin Endocrinol Metab. 2021 May 13;106(6):1728-1741. doi: 10.1210/clinem/dgab080.

name: Karolinska Institutet, Karolinska University Hospital, Solna, Sweden

name: Université de Paris, Hôpital Universitaire Necker Enfants Malades, Paris, France

name: Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA

name: University of Minnesota Masonic Children’s Hospital, Minneapolis, Minnesota, USA

name: University Children’s Hospital, Saarland University Medical Center, Homburg, Germany

name: Department of Pediatrics, Division of Endocrinology, Erasmus University Medical Center/Sophia Children’s Hospital, Rotterdam, the Netherlands

name: Novo Nordisk Inc, Plainsboro, New Jersey, USA

name: Thomas Jefferson University, Philadelphia, Pennsylvania, USA

name: Nemours/DuPont Hospital for Children, Wilmington, Delaware, USA

journal_name: The Journal of Clinical Endocrinology & Metabolism

ReferenceCitation: Weber MM, Gordon MB, Höybye C, Jørgensen JOL, Puras G, Popovic-Brkic V, Molitch ME, Ostrow V, Holot N, Pietropoli A, Biller BMK. Growth hormone replacement in adults: Real-world data from two large studies in US and Europe. Growth Horm IGF Res. 2020 Feb;50:71-82. doi: 10.1016/j.ghir.2019.09.002. Epub 2019 Oct 26.

ReferenceCitation: Sävendahl L, Polak M, Backeljauw P, Blair J, Miller BS, Rohrer TR, Pietropoli A, Ostrow V, Ross J. Treatment of Children With GH in the United States and Europe: Long-Term Follow-Up From NordiNet® IOS and ANSWER Program. J Clin Endocrinol Metab. 2019 Oct 1;104(10):4730-4742. doi: 10.1210/jc.2019-00775. Erratum in: J Clin Endocrinol Metab. 2020 Jun 1;105(6):.

name: Hôpital Necker Enfants Malades, Paris, France

name: Cincinnati Center for Growth Disorders, Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio

name: Alder Hey Children’s NHS Foundation Trust, Liverpool, United Kingdom

name: Novo Nordisk Inc., Plainsboro, New Jersey

location_country:
Luxembourg
Finland
Belgium
Russian Federation
Denmark
France
Lithuania
Norway
Argentina
Slovenia
Sweden
Saudi Arabia
Switzerland
Netherlands
Hungary
Ireland
Germany
United Kingdom
Italy
Serbia
Czechia
Israel
Spain


title: Patient Characteristics in Advanced Renal Cell Carcinoma and Daily Practice Treatment With Nexavar

summary: Renal cell carcinoma accounts for roughly 3 % of all cancer. It is a rather aggressive cancer type, which means that patients who present with an advanced disease have a rather poor prognosis. When this study has been started the standard therapy for patients has been cytokines, which might be accompanied by significant toxicities or might fail the therapeutic goal. In these cases sorafenib can be a feasible therapeutic option. This non-interventional study has been created and is being conducted to collect clinical data on the patients' therapy with sorafenib in an everyday treatment schedule. The main goal of this study focuses on patient characteristics and tumor status in RCC treated with sorafenib as well as the treatment duration and safety of sorafenib under everyday treatment conditions.

lead_sponsor: Bayer

location_country:
Austria
Sweden
Mexico
Netherlands
Philippines
France
Poland
Greece
Slovakia
Slovenia
Argentina
Russian Federation
China
Indonesia
Germany
Czech Republic
Korea, Republic of
Colombia


title: Prophylactic Treatment With Recombinant Factor VIIa (rFVIIa, NovoSeven®) in Haemophilia Patients With Inhibitors

acronym: PROPACT

summary: This study is conducted in Europe and North and South America. The primary aim of this observational study is to evaluate the frequency and pattern of bleeding episodes in haemophilia patients receiving preventative treatment with activated recombinant human factor VII. The secondary aim is to evaluate which patients are selected for this treatment, the dose and dose intervals used, and the safety of activated recombinant human factor VII when used as prevention. The study also aims to increase understanding of the unmet medical need and clinical relevance of preventative treatment in haemophilia patients.

time_perspective: Retrospective

location_country:
Italy
United States
Sweden
Switzerland
United Kingdom
Germany
Croatia
France
Slovakia
Ireland
Argentina
Spain
Canada
Czech Republic


title: A Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Subcutaneous Methylnaltrexone for the Treatment of Opioid-Induced Constipation in Subjects With Cancer-Related Pain

summary: This purpose of this study is to evaluate the safety and effectiveness of a subcutaneous (injection beneath the skin) form of methylnaltrexone in subjects who have cancer-related pain and constipation from taking opioids. The length of participation will be up to 7 weeks.

status: Withdrawn

lead_sponsor: Bausch Health Americas, Inc.

location_country:
United States
Canada
France
Spain


title: A Multicentre, Randomised, Double-Blind, Placebo Controlled, Phase II Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable Implants of Afamelanotide (CUV1647) for the Prophylactic Treatment of Pre-Cancerous Skin Lesions of the Head, Forearms and Hands in Immune Compromised, Organ Transplant Patients.

summary:
The purpose of this study is to determine whether afamelanotide (CUV1647) is effective in reducing the number of actinic keratoses and squamous cell carcinomas developing in immune compromised organ transplant recipients, who are at particularly high risk, over a 24 month test period.

The number of lesions formed on the head, hands and forarms will be monitored over this 24 month test period.

primary_purpose: Prevention

lead_sponsor: Clinuvel Pharmaceuticals Limited

location_country: Italy Sweden Switzerland France Belgium Australia Germany

location_facility:
The Princess Alexandra Hospital
Universitätsspital Zürich
The Queen Elizabeth Hospital
Gent University Hospital
Charité Univeritätsklinikum
University Hospital
Karolinska University Hospital
Kiel University Hospital
Hospital Erasme
Ospedali Riuniti di Bergamo
University of Padua
The Royal Melbourne Hospital