title: Predicting Acute-on-Chronic Liver Failure in Cirrhosis (PREDICT) Study

acronym: PREDICT

summary: The aim of this study is to assess prospectively the critical period prior to the development of Acute-on-Chronic Liver Failure (ACLF) (1), to uncover mechanistic and pathophysiological processes associated with the development and clinical course of ACLF (2) and to identify the precipitating events of ACLF (3).

ReferenceCitation: Trebicka J, Fernandez J, Papp M, Caraceni P, Laleman W, Gambino C, Giovo I, Uschner FE, Jansen C, Jimenez C, Mookerjee R, Gustot T, Albillos A, Bañares R, Jarcuska P, Steib C, Reiberger T, Acevedo J, Gatti P, Shawcross DL, Zeuzem S, Zipprich A, Piano S, Berg T, Bruns T, Danielsen KV, Coenraad M, Merli M, Stauber R, Zoller H, Ramos JP, Solé C, Soriano G, de Gottardi A, Gronbaek H, Saliba F, Trautwein C, Kani HT, Francque S, Ryder S, Nahon P, Romero-Gomez M, Van Vlierberghe H, Francoz C, Manns M, Garcia-Lopez E, Tufoni M, Amoros A, Pavesi M, Sanchez C, Praktiknjo M, Curto A, Pitarch C, Putignano A, Moreno E, Bernal W, Aguilar F, Clària J, Ponzo P, Vitalis Z, Zaccherini G, Balogh B, Gerbes A, Vargas V, Alessandria C, Bernardi M, Ginès P, Moreau R, Angeli P, Jalan R, Arroyo V; PREDICT STUDY group of the EASL-CLIF CONSORTIUM. PREDICT identifies precipitating events associated with the clinical course of acutely decompensated cirrhosis. J Hepatol. 2021 May;74(5):1097-1108. doi: 10.1016/j.jhep.2020.11.019. Epub 2020 Nov 20.

family: Trebicka

full_name: Jonel Trebicka

given: Jonel

family: Fernandez

full_name: Javier Fernandez

given: Javier

family: Papp

full_name: Maria Papp

given: Maria

family: Caraceni

full_name: Paolo Caraceni

given: Paolo

family: Laleman

full_name: Wim Laleman

given: Wim

family: Gambino

full_name: Carmine Gambino

given: Carmine

family: Giovo

full_name: Ilaria Giovo

given: Ilaria

family: Uschner

full_name: Frank Erhard Uschner

given: Frank Erhard

family: Jansen

full_name: Christian Jansen

given: Christian

family: Jimenez

full_name: Cesar Jimenez

given: Cesar

family: Mookerjee

full_name: Rajeshwar Mookerjee

given: Rajeshwar

family: Gustot

full_name: Thierry Gustot

given: Thierry

family: Albillos

full_name: Agustin Albillos

given: Agustin

family: Bañares

full_name: Rafael Bañares

given: Rafael

family: Jarcuska

full_name: Peter Jarcuska

given: Peter

family: Steib

full_name: Christian Steib

family: Reiberger

full_name: Thomas Reiberger

given: Thomas

family: Acevedo

full_name: Juan Acevedo

given: Juan

family: Gatti

full_name: Pietro Gatti

given: Pietro

family: Shawcross

full_name: Debbie L. Shawcross

given: Debbie L.

family: Zeuzem

full_name: Stefan Zeuzem

given: Stefan

family: Zipprich

full_name: Alexander Zipprich

given: Alexander

family: Piano

full_name: Salvatore Piano

given: Salvatore

repository_institution: Ghent University - Ghent University Academic Bibliography

repository_institution: University of Bern - Bern Open Repository and Information System

repository_institution: Université Libre de Bruxelles - Dépôt institutionnel de l'Université libre de Bruxelles

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repository_institution: University College London - UCL Discovery

ReferenceCitation: Trebicka J, Fernandez J, Papp M, Caraceni P, Laleman W, Gambino C, Giovo I, Uschner FE, Jimenez C, Mookerjee R, Gustot T, Albillos A, Bañares R, Janicko M, Steib C, Reiberger T, Acevedo J, Gatti P, Bernal W, Zeuzem S, Zipprich A, Piano S, Berg T, Bruns T, Bendtsen F, Coenraad M, Merli M, Stauber R, Zoller H, Ramos JP, Solè C, Soriano G, de Gottardi A, Gronbaek H, Saliba F, Trautwein C, Özdogan OC, Francque S, Ryder S, Nahon P, Romero-Gomez M, Van Vlierberghe H, Francoz C, Manns M, Garcia E, Tufoni M, Amoros A, Pavesi M, Sanchez C, Curto A, Pitarch C, Putignano A, Moreno E, Shawcross D, Aguilar F, Clària J, Ponzo P, Jansen C, Vitalis Z, Zaccherini G, Balogh B, Vargas V, Montagnese S, Alessandria C, Bernardi M, Ginès P, Jalan R, Moreau R, Angeli P, Arroyo V; PREDICT STUDY group of the EASL-CLIF Consortium. The PREDICT study uncovers three clinical courses of acutely decompensated cirrhosis that have distinct pathophysiology. J Hepatol. 2020 Oct;73(4):842-854. doi: 10.1016/j.jhep.2020.06.013. Epub 2020 Jul 13.

repository_institution: Universitat de Barcelona - Dipòsit Digital de la Universitat de Barcelona

repository_institution: University Library Bern - BORIS - Bern Open Repository and Information System

repository_institution: Library of the Hungarian Academy of Sciences - Repository of the Academy's Library

location_country:
Italy
Austria
Switzerland
Netherlands
Spain
France
Slovakia
Hungary
Belgium
Portugal
Turkey
Denmark
Germany
United Kingdom

location_facility: Derriford Hospital, Plymouth Hospitals Trust

location_facility: Hospital Clinic y Provencial de Barcelona

location_facility: Pavol Jozef Sfarik University Kosice/Roosevelt Hospital Bystrica

location_facility: Hospital General Universitario Gregorio Maranon


intervention_type: Drug

status: Terminated

study_start_date_type: Actual

study_type: Interventional

lead_sponsor: Teva Branded Pharmaceutical Products R&D, Inc.

enrollment_type: Actual

primary_purpose: Treatment

location_country:
United States
Germany
France
Czechia
Poland
Israel
Romania
Hungary
Belgium
Russian Federation
Spain
Canada
Ukraine

location_facility:
Teva Investigational Site 80132
Teva Investigational Site 14619
Teva Investigational Site 32676
Teva Investigational Site 11133
Teva Investigational Site 53401
Teva Investigational Site 53403
Teva Investigational Site 14655
Teva Investigational Site 35227
Teva Investigational Site 14646
Teva Investigational Site 58301
Teva Investigational Site 54150
Teva Investigational Site 58297
Teva Investigational Site 32677
Teva Investigational Site 14647
Teva Investigational Site 58299
Teva Investigational Site 51283
Teva Investigational Site 14623
Teva Investigational Site 58304
Teva Investigational Site 14639
Teva Investigational Site 58286
Teva Investigational Site 58293
Teva Investigational Site 53406
Teva Investigational Site 14638
Teva Investigational Site 14624
Teva Investigational Site 58288
Teva Investigational Site 50458
Teva Investigational Site 14645
Teva Investigational Site 14622
Teva Investigational Site 51280
Teva Investigational Site 14626
Teva Investigational Site 14652
Teva Investigational Site 14621
Teva Investigational Site 50454
Teva Investigational Site 58294
Teva Investigational Site 32674
Teva Investigational Site 53407
Teva Investigational Site 35229
Teva Investigational Site 58303
Teva Investigational Site 14644
Teva Investigational Site 52115
Teva Investigational Site 53405
Teva Investigational Site 52117
Teva Investigational Site 58287
Teva Investigational Site 51285
Teva Investigational Site 50457
Teva Investigational Site 11134
Teva Investigational Site 14635
Teva Investigational Site 35228
Teva Investigational Site 14629
Teva Investigational Site 32681
Teva Investigational Site 32679
Teva Investigational Site 32671
Teva Investigational Site 54148
Teva Investigational Site 52116
Teva Investigational Site 80130
Teva Investigational Site 14640
Teva Investigational Site 37082
Teva Investigational Site 58296
Teva Investigational Site 14628
Teva Investigational Site 31217
Teva Investigational Site 58282
Teva Investigational Site 58289
Teva Investigational Site 14649
Teva Investigational Site 32675
Teva Investigational Site 14620
Teva Investigational Site 50455
Teva Investigational Site 51291
Teva Investigational Site 14641
Teva Investigational Site 14651
Teva Investigational Site 14627
Teva Investigational Site 32670
Teva Investigational Site 50460
Teva Investigational Site 58291
Teva Investigational Site 14642
Teva Investigational Site 53402
Teva Investigational Site 50456
Teva Investigational Site 50461
Teva Investigational Site 32672
Teva Investigational Site 58284
Teva Investigational Site 54149
Teva Investigational Site 58292
Teva Investigational Site 58295
Teva Investigational Site 53408
Teva Investigational Site 52113
Teva Investigational Site 51292
Teva Investigational Site 51282
Teva Investigational Site 58283
Teva Investigational Site 80133
Teva Investigational Site 80135
Teva Investigational Site 51284
Teva Investigational Site 51287
Teva Investigational Site 58290
Teva Investigational Site 58302
Teva Investigational Site 80129
Teva Investigational Site 37083
Teva Investigational Site 50453
Teva Investigational Site 51290
Teva Investigational Site 37081
Teva Investigational Site 14631
Teva Investigational Site 32678
Teva Investigational Site 51281
Teva Investigational Site 14633
Teva Investigational Site 51286
Teva Investigational Site 14634
Teva Investigational Site 32680
Teva Investigational Site 58298
Teva Investigational Site 53399
Teva Investigational Site 51289
Teva Investigational Site 53404
Teva Investigational Site 80131
Teva Investigational Site 31218
Teva Investigational Site 58285
Teva Investigational Site 32673
Teva Investigational Site 80134
Teva Investigational Site 14650
Teva Investigational Site 14653
Teva Investigational Site 14648
Teva Investigational Site 14643
Teva Investigational Site 14632
Teva Investigational Site 54151
Teva Investigational Site 14630
Teva Investigational Site 51293
Teva Investigational Site 31219
Teva Investigational Site 80136
Teva Investigational Site 58300
Teva Investigational Site 14654

study_completion_date_type: Actual

type: EudraCT Number

source: euctr

title: An Open-Label Extension Study of Reslizumab 110-mg Fixed, Subcutaneous Dosing in Patients 12 Years of Age and Older With Severe Eosinophilic Asthma

summary: This is a multicenter, open-label (OL) extension study to obtain additional long-term safety data for subcutaneous (sc) administration of reslizumab treatment administered at a fixed dose of 110 mg in patients 12 years of age and older with severe eosinophilic asthma who completed the treatment period of a placebo-controlled Phase 3 trial of sc reslizumab. The study consists of a screening/baseline visit followed by a 36-week OL treatment period and a 15-week follow-up period.

lead_sponsor_type: industriel

status_simplified: Unknown


ReferenceType: derived

ReferenceCitation: Papp KA, Lebwohl MG, Puig L, Ohtsuki M, Beissert S, Zeng J, Rubant S, Sinvhal R, Zhao Y, Soliman AM, Alperovich G, Leonardi C. Long-term efficacy and safety of risankizumab for the treatment of moderate-to-severe plaque psoriasis: interim analysis of the LIMMitless open-label extension trial beyond 3 years of follow-up. Br J Dermatol. 2021 Jun 22. doi: 10.1111/bjd.20595. [Epub ahead of print]

name: K Papp Clinical Research and Probity Medical Research Waterloo ON Canada

family: Papp

full_name: K.A. Papp

sequence: first

name: Icahn School of Medicine at Mount Sinai New York NY USA

family: Lebwohl

full_name: M.G. Lebwohl

sequence: additional

name: Hospital de la Santa Creu i Sant Pau Barcelona Spain

family: Puig

full_name: L. Puig

given: L.

family: Ohtsuki

full_name: M. Ohtsuki

given: M.

name: Department of Dermatology University Hospital Carl Gustav Carus, TU Dresden Dresden Germany

family: Beissert

full_name: S. Beissert

given: S.

family: Zeng

full_name: J. Zeng

given: J.

family: Rubant

full_name: S. Rubant

family: Sinvhal

full_name: R. Sinvhal

given: R.

family: Zhao

full_name: Y. Zhao

given: Y.

family: Soliman

full_name: A.M. Soliman

family: Alperovich

full_name: G. Alperovich

given: G.

name: Central Dermatology Richmond Heights MO USA

family: Leonardi

full_name: C. Leonardi

given: C.

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: British Journal of Dermatology

oa_host_type: publisher

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc

license_normalized: cc-by-nc

version: publishedVersion

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: Long‐term efficacy and safety of risankizumab for the treatment of moderate‐to‐severe plaque psoriasis: interim analysis of the LIMMitless open‐label extension trial beyond 3 years of follow‐up

intervention_type: Biological

status: Active, not recruiting

ipd_sharing_description: AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols and clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.

lead_sponsor: AbbVie

location_country:
Austria
United States
Sweden
Mexico
Spain
Germany
Finland
France
Czechia
Japan
Poland
Australia
Belgium
Portugal
Canada
Korea, Republic of
Taiwan

acronym: LIMMITLESS

location_facility: CHU Toulouse - Hopital Larrey /ID# 153951

location_facility: Osaka City University Hospital /ID# 153869

location_facility: The Guenther Dermatology Research Centre /ID# 153841

location_facility: Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz /ID# 154092

location_facility: National Cheng Kung University Hospital /ID# 154135

location_facility: Progressive Clinical Research /ID# 154149

location_facility: Derm Clin Res Ctr San Antonio /ID# 153978

location_facility: Charite Universitaetsmedizin Berlin - Campus Mitte /ID# 153896

location_facility: ActivMed Practices and Research, LLC. /ID# 154044

location_facility: Nippon Medical School Hospital /ID# 154084

location_facility: Jichi Medical University Hospital /ID# 154286

location_facility: University of Michigan Comprehensive Cancer Center Michigan Medicine /ID# 154111

location_facility: Northshore University Health System Dermatology Clinical Trials Unit /ID# 154011

location_facility: The Jikei University Hospital /ID# 154080

location_facility: Seoul National University Hospital /ID# 153484

location_facility: The Centre for Clinical Trials /ID# 153871

location_facility: Nagasaki University Hospital /ID# 170219

location_facility: University of Alabama at Birmingham - Main /ID# 153983

location_facility: Huntington Medical Foundation /ID# 154002

location_facility: Centre de recheche dermatologique du Quebec Metropolitain /ID# 153856

location_facility: Japan Organization of Occupational Health and Society Kanto Rosai Hospital /ID# 169172

location_facility: St George Dermatology & Skin Cancer Centre /ID# 154308

location_facility: Sahlgrenska University Hospital /ID# 154132

location_facility: Dermatology Research Institute Inc. /ID# 153850

location_facility: York Dermatology Clinic and Research Centre /ID# 153860

location_facility: Forest Hills Dermatology Group /ID# 154113

location_facility: St.Luke's International Hospital /ID# 153863

location_facility: Dermatology Research Associates /ID# 153977

location_facility: Hospital Universitario Dr. Jose Eleuterio Gonzalez /ID# 153882

location_facility: IMTR - Grand Hopital de Charleroi /ID# 154298

location_facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu /ID# 153473

location_facility: DermResearchCenter of New York, Inc. /ID# 154012

location_facility: Hautarztpraxis Dr. Niesmann und Dr. Othlinghaus /ID# 154289

location_facility: Austin Dermatology Associates /ID# 154042

location_facility: Medical Cooperation Kojinkai Sapporo Skin Clinic /ID# 154083

location_facility: North Bay Dermatology Centre /ID# 153845

location_facility: Eastern Canada Cutaneous Resea /ID# 153870

location_facility: Epiphany Dermatology of Kansas LLC /ID# 153993

location_facility: Universitaetsklinikum Hamburg-Eppendorf (UKE) /ID# 154089

location_facility: Klinikum rechts der Isar - Technische Universitaet Muenchen /ID# 153915

location_facility: Chonnam National University Hospital /ID# 153488

location_facility: Wilmington Dermatology Center /ID# 154109

location_facility: Coastal Carolina Research Center /ID# 153992

location_facility: Dermatology Associates of Seattle /ID# 153988

location_facility: The Indiana Clinical Trials Center /ID# 153976

location_facility: KonKuk University Medical Center /ID# 153483

location_facility: Center for Dermatology Clinical Research /ID# 154009

location_facility: Virginia Clinical Research, Inc. /ID# 154016

location_facility: Rush University Medical Center /ID# 154099

location_facility: Universitaetsklinik Heidelberg /ID# 153910

location_facility: Massachusetts General Hospital /ID# 153990

location_facility: Hospital Santa Creu i Sant Pau /ID# 154125

location_facility: Suzanne Bruce and Associates /ID# 154026

location_facility: Taipei Medical University Shuang Ho Hospital /ID# 154134

location_facility: Florida Academic Centers Research /ID# 154045

location_facility: New England Research Associates, LLC /ID# 154004

location_facility: Leavitt Medical Associates of Florida /ID# 154046

location_facility: Dermatology Consulting Service /ID# 154010

location_facility: Hospital Universitario Infanta Leonor /ID# 154121

location_facility: MUDr. Blanka Havlickova - dermatovenerologie /ID# 153888

location_facility: Tokyo Medical University Hospital /ID# 153874

location_facility: Fukushima Medical University Hospital /ID# 153873

location_facility: National Taiwan University Hospital /ID# 154136

location_facility: Iwate Medical University Hospital /ID# 170843

location_facility: The Dermatology Center PSC - New Albany /ID# 154075

location_facility: Dr. Chih-ho Hong Medical Inc. /ID# 153842

location_facility: Hokkaido University Hospital /ID# 154285

location_facility: Modern Research Associates, PL /ID# 154043

location_facility: Japan Community Health care Organization Tokyo Yamate Medical Center /ID# 153868

location_facility: CCA Braga - Hospital de Braga /ID# 156323

location_facility: National Hospital Organization Sagamihara National Hospital /ID# 153867

location_facility: Oregon Medical Res Center PC /ID# 154003

location_facility: Karolinska University Hospital Solna /ID# 154131

location_facility: CHP, EPE- Hospital Geral de Sa /ID# 153464

location_facility: Nippon Life Saiseikai Public Interest Foundation Nippon Life Hospital /ID# 156319

location_facility: Centro de Dermatologia de Monterrey /ID# 153881

location_facility: Paddington Testing Co., Inc. /ID# 154022

location_facility: North Eastern Health Specialists /ID# 154309

location_facility: Korea Universtiy Guro Hospital /ID# 153490

location_facility: Nihon University Itabashi Hospital /ID# 154081

location_facility: Klinika Ambroziak Sp. z o.o. /ID# 153467

location_facility: Minnesota Clinical Study Center /ID# 154015

location_facility: Northwestern University Feinberg School of Medicine /ID# 153974

location_facility: DermaKiel Allergie und Haut Centrum /ID# 201316

location_facility: Nagoya City University Hospital /ID# 153866

location_facility: Juntendo University Hospital /ID# 200292

location_facility: Hospital Universitario Germans Trias i Pujol /ID# 153465

location_facility: Hospital General Universitario de Valencia /ID# 154126

location_facility: Universitaetsklinikum Erlangen /ID# 154093

location_facility: The Catholic University of Korea, Seoul St. Mary's Hospital /ID# 153486

location_facility: Dartmouth-Hitchcock Medical Center /ID# 154095

location_facility: Klinische Forschung Hamburg GmbH /ID# 154290

location_facility: Michigan Center for Skin Care /ID# 153986

location_facility: KO-MED Centra Kliniczne Lublin /ID# 153469

location_facility: Oregon Dermatology and Research Center /ID# 153982

location_facility: Windsor Clinical Research, Inc /ID# 153877

location_facility: Kaiser Permanente Los Angeles /ID# 154079

location_facility: Synexus Clinical Research US, Inc. /ID# 154127

location_facility: Medizinische Universitaet Wien /ID# 154302

location_facility: Klinische Forschung Schwerin GmbH /ID# 154291

location_facility: Yokohama City University Hospital /ID# 168831

location_facility: Universitaetsklinikum Tuebingen Medizinische Klinik /ID# 153912

location_facility: CMS3 Company for Medical Study /ID# 201318

location_facility: Universitaetsmedizin Goettingen /ID# 154091

location_facility: Dermatology Ottawa Research Centre /ID# 153862

location_facility: Bakersfield Derma & Skin Cance /ID# 154008

location_facility: Dermatologische Gemeinschaftspraxis Mahlow /ID# 153892

location_facility: Psoriasis Treatment Center of Central New Jersey /ID# 154001

location_facility: Pusan National University Hospital /ID# 153485

location_facility: Southern California Derma. Inc /ID# 154006

location_facility: Hautarztpraxis Dr. med. Matthias Hoffmann /ID# 153917

location_facility: High-Med Przychodnia Specjalistyczna /ID# 153468

location_facility: Hamamatsu University Hospital /ID# 170132

location_facility: Centro Hospitalar Universitario de Sao Joao, EPE /ID# 153463

location_facility: Universitaetsklinikum Frankfurt /ID# 153891

location_facility: Great Lakes Research Group, Inc. /ID# 153998

location_facility: Universitaetsklinikum Schleswig-Holstein Campus Kiel /ID# 153916

location_facility: Total Skin and Beauty Derm Ctr /ID# 154000

location_facility: University Hospital Kyoto Prefectural University of Medicine /ID# 169415

location_facility: NW Arkansas Clinical Trials Center /ID# 154014

location_facility: ISA Interdisciplinary Study Association GmbH /ID# 153907

location_facility: Kansai Medical University Hospital /ID# 170884

location_facility: Center for Clinical Studies - Webster TX /ID# 154150

location_facility: Gachon University Gil Medical Center /ID# 153489

location_facility: Uniwersyteckie Centrum Kliniczne /ID# 153471

location_facility: Universitaetsklinikum Leipzig /ID# 153909

location_facility: Coastal Clinical Research Center of the Carolinas /ID# 154112

location_facility: Centrum Badan Klinicznych PI-House sp. z o.o. /ID# 153884

location_facility: Seoul National University Hospital /ID# 153491

location_facility: Cliniques Universitaires de Bruxelles Hopital Erasme /ID# 154293

location_facility: UNISON Center for Clinical Tri /ID# 153979

location_facility: Universitatsklinikum Munster /ID# 153895

location_facility: Centro Hospitalar Universitário de Lisboa Norte, EPE - Hospital de Santa Maria /ID# 153462

location_facility: California Dermatology and Research Institute /ID# 154007

location_facility: Dermatology Specialists, Inc /ID# 153984

location_facility: Chu de Nice-Hopital L'Archet Ii /Id# 153948

location_facility: Progressive Medical Research /ID# 154027

location_facility: Yamaguchi University Hospital /ID# 156322

location_facility: The South Bend Clinic Center /ID# 154147

location_facility: Wake Radiology UNC REX Healthcare - Raleigh Office /ID# 202490

location_facility: Universitaetsklinikum Carl Gustav Carus an der TU Dresden /ID# 153906

ipd_sharing: Yes

study_completion_date_type: Anticipated

type: Other Grant/Funding Number

title: A Multicenter, Open Label Study to Assess the Safety and Efficacy of Risankizumab for Maintenance in Moderate to Severe Plaque Type Psoriasis (LIMMITLESS)

summary: The purpose of this study is to investigate the long-term safety and efficacy of risankizumab in the treatment of moderate to severe chronic plaque psoriasis.

status_simplified: Ongoing


ReferenceCitation: Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2021 May;8(5):e323-e333. doi: 10.1016/S2352-3026(21)00059-4. Epub 2021 Apr 7.

family: Howard

full_name: Jo Howard

given: Jo

family: Ataga

full_name: Kenneth I Ataga

given: Kenneth I

family: Brown

full_name: Robert C Brown

given: Robert C

family: Achebe

full_name: Maureen Achebe

given: Maureen

family: Nduba

full_name: Videlis Nduba

given: Videlis

family: El-Beshlawy

full_name: Amal El-Beshlawy

given: Amal

family: Hassab

full_name: Hoda Hassab

given: Hoda

family: Agodoa

full_name: Irene Agodoa

given: Irene

family: Tonda

full_name: Margaret Tonda

given: Margaret

family: Gray

full_name: Sarah Gray

given: Sarah

family: Lehrer-Graiwer

full_name: Joshua Lehrer-Graiwer

given: Joshua

family: Vichinsky

full_name: Elliott Vichinsky

given: Elliott

journal_name: The Lancet Haematology

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

ReferenceCitation: Minniti CP, Knight-Madden J, Tonda M, Gray S, Lehrer-Graiwer J, Biemond BJ. The impact of voxelotor treatment on leg ulcers in patients with sickle cell disease. Am J Hematol. 2021 Apr 1;96(4):E126-E128. doi: 10.1002/ajh.26101. Epub 2021 Feb 19.

name: Department of Hematology Montefiore Medical Center/Albert Einstein College of Medicine  Bronx New York

family: Minniti

full_name: Caterina P. Minniti

given: Caterina P.

name: Sickle Cell Unit, Caribbean Institute for Health Research University of the West Indies, Mona  Kingston Jamaica

family: Knight‐Madden

full_name: Jennifer Knight‐Madden

given: Jennifer

name: Global Blood Therapeutics  South San Francisco California

family: Lehrer‐Graiwer

full_name: Joshua Lehrer‐Graiwer

name: Department of Hematology, Amsterdam University Medical Centers University of Amsterdam  Amsterdam The Netherlands

family: Biemond

full_name: Bart J. Biemond

given: Bart J.

journal_name: American Journal of Hematology

oa_colors: green hybrid

oa_host_type: publisher;repository

license: cc-by

license_normalized: cc-by

evidence: oa repository (via pmcid lookup)

host_type: repository

license_normalized: no license

repository_normalized: PubMed Central

ReferenceCitation: Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.

name: From the University of California, San Francisco (UCSF) Benioff Children’s Hospital Oakland, Oakland (E.V.), and Global Blood Therapeutics, South San Francisco (C.C.H., J.L.-G., M.T., A.I., B.T.) — both in California; the University of Tennessee Health Science Center at Memphis, Memphis (K.I.A.); Cincinnati Children’s Hospital and University of Cincinnati, Cincinnati (R.E.W.); Kenya Medical Research Institute, Kisumu, Kenya (V.N.); Cairo University, Cairo (A.E.-B.), and the Pediatric Department and...

ReferenceCitation: Metcalf B, Chuang C, Dufu K, Patel MP, Silva-Garcia A, Johnson C, Lu Q, Partridge JR, Patskovska L, Patskovsky Y, Almo SC, Jacobson MP, Hua L, Xu Q, Gwaltney SL 2nd, Yee C, Harris J, Morgan BP, James J, Xu D, Hutchaleelaha A, Paulvannan K, Oksenberg D, Li Z. Discovery of GBT440, an Orally Bioavailable R-State Stabilizer of Sickle Cell Hemoglobin. ACS Med Chem Lett. 2017 Jan 23;8(3):321-326. doi: 10.1021/acsmedchemlett.6b00491. eCollection 2017 Mar 9.

name:
Global Blood Therapeutics, Inc., South
San Francisco, California 94080, United States

name:
Cytokinetics, Inc., South
San Francisco, California 94080, United States

name: Albert Einstein College of Medicine, Bronx, New York 10461, United States

name:
Department
of Pharmaceutical Chemistry, University of California, San Francisco, California 94158, United States

name: Tandem Sciences, Inc., Menlo Park, California 94025, United States

license: acs-specific: authorchoice/editors choice usage agreement

location_country:
Italy
Lebanon
Netherlands
Jamaica
France
Egypt
Turkey
Oman
United States
United Kingdom
Canada
Kenya

title: A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease


type: Other Identifier

title: Phase 1 Open-label, Multicenter Study of MK-1454 Administered by Intratumoral Injection as Monotherapy and in Combination With Pembrolizumab for Patients With Advanced/Metastatic Solid Tumors or Lymphomas

summary:
The purpose of this study is to identify a maximum tolerated dose (MTD) or maximum administered dose (MAD) of ulevostinag alone and of ulevostinag in combination with pembrolizumab in participants with advanced/metastatic solid tumors or lymphomas in Part 1, and to evaluate the safety and efficacy of ulevostinag via intratumoral (IT) injection in combination with pembrolizumab in selected solid tumors in Part 2.

Ulevostinag will be administered IT; pembrolizumab (pembro) will be administered via intravenous (IV) infusion. In Part 1, participants will be allocated to one of three treatment arms: ulevostinag monotherapy (cutaneous/subcutaneous [cut/subcut] lesions), ulevostinag +pembro (cut/subcut lesions), or ulevostinag +pembro (visceral lesions).

In Part 2, participants with head and neck squamous cell carcinoma (HNSCC) who are anti-programmed cell death-protein 1 or anti-programmed cell death-ligand 1 (anti-PD-1/PD-L1) refractory or with anti-PD-1/PD-L1 treatment (TrT)-naïve triple-negative breast cancer (TNBC) or with anti-PD-1/PD-L1 TrT-naïve solid tumors with liver metastases/lesions will receive ulevostinag via IT injection at the RP2D determined in Part 1 PLUS pembrolizumab via IV infusion for up 35 cycles (up approximately 2 years).

design_allocation: Non-Randomized

enrollment_type: Anticipated

ReferenceCitation: Gogoi H, Mansouri S, Jin L. The Age of Cyclic Dinucleotide Vaccine Adjuvants. Vaccines (Basel). 2020 Aug 13;8(3). pii: E453. doi: 10.3390/vaccines8030453. Review.

family: Gogoi

full_name: Himanshu Gogoi

given: Himanshu

family: Mansouri

full_name: Samira Mansouri

given: Samira

family: Jin

full_name: Lei Jin

given: Lei

currency_apc_doaj: CHF

journal_name: Vaccines

oa_colors: green gold

evidence: oa journal (via doaj)

publisher: MDPI AG

publisher_dissemination: MDPI

publisher_group: MDPI

publisher_normalized: MDPI

lead_sponsor: Merck Sharp & Dohme Corp.

location_country:
France
Israel
United States
United Kingdom
Korea, Republic of

location_facility: Mary Crowley Cancer Research Center ( Site 0001)

location_facility: University of California San Francisco ( Site 0007)

location_facility: The Royal Marsden Foundation Trust ( Site 0031)

location_facility: The Royal Marsden NHS Foundation Trust. ( Site 0032)


title: A Phase II, Open-label, Multicentre, Randomised Study of the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of CAM2029 in Patients With Acromegaly and Neuroendocrine Tumours (NETs) Previously Treated With Sandostatin® LAR®

summary: This is a Phase II, open-label multicentre, randomised study to assess the PK, PD, efficacy, and safety of two dosing regimens of CAM2029 in adult patients with acromegaly or a functional, well-differentiated NET, with carcinoid symptoms.

status: Completed

design_allocation: Randomized

ReferenceCitation: Pavel M, Borson-Chazot F, Cailleux A, Hörsch D, Lahner H, Pivonello R, Tauchmanova L, Darstein C, Olsson H, Tiberg F, Ferone D. Octreotide SC depot in patients with acromegaly and functioning neuroendocrine tumors: a phase 2, multicenter study. Cancer Chemother Pharmacol. 2019 Feb;83(2):375-385. doi: 10.1007/s00280-018-3734-1. Epub 2018 Dec 8.

family: Pavel

full_name: Marianne Pavel

given: Marianne

family: Borson-Chazot

full_name: Françoise Borson-Chazot

given: Françoise

family: Cailleux

full_name: Anne Cailleux

given: Anne

family: Hörsch

full_name: Dieter Hörsch

given: Dieter

family: Lahner

full_name: Harald Lahner

given: Harald

family: Pivonello

full_name: Rosario Pivonello

given: Rosario

family: Tauchmanova

full_name: Libuse Tauchmanova

given: Libuse

family: Darstein

full_name: Christelle Darstein

given: Christelle

family: Olsson

full_name: Håkan Olsson

given: Håkan

family: Tiberg

full_name: Fredrik Tiberg

given: Fredrik

family: Ferone

full_name: Diego Ferone

given: Diego

journal_name: Cancer Chemotherapy and Pharmacology

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

ipd_sharing: No

ipd_sharing_description: No plan to share IPD

lead_sponsor: Camurus AB

location_country: Italy Germany Sweden France

location_facility: RCCS Azienda Ospedaliera Universitaria San Martino IST

location_facility: Università degli Studi di Napoli Federico II

status_simplified: Completed


title: Phase I-II Prospective Trial, Multicenter, Open Label, Exploring the Combination of Trabectedin Plus Radiotherapy in Soft Tissue Sarcoma Patients

acronym: TRASTS

summary:
Phase I-II trial that combines trabectedin plus radiotherapy for tumor reduction response measure in two cohorts of patients:

Cohort A: Patients with diagnosis of non-operable or unresectable or not oncologically recommended metastasectomy of limited to lung metastases soft tissue sarcoma.

Cohort B: Patients with locally advanced resectable Myxoid Liposarcoma. Cohort C: Patients with retroperitoneal and resectable soft tissue sarcoma (liposarcoma and leiomyosarcoma) Phase I: escalating dose of 1.3 or 1.5 mg/m2. Phase I for cohort C: de-escalating dose of 1.5 or 1.3mg/m2 Radiotherapy for cohort A: 30Gy in 10 fractions (3Gy/fraction) Radiotherapy for cohort B: 45Gy in 25 fractions (1.8Gy/fraction) Radiotherapy for cohort C: 45Gy in 25 fractions (1.8Gy/fraction) A translational substudy is developed to analyse different biomarkers predictive value.

status: Recruiting

ReferenceCitation: Vatner R, James CD, Sathiaseelan V, Bondra KM, Kalapurakal JA, Houghton PJ. Radiation therapy and molecular-targeted agents in preclinical testing for immunotherapy, brain tumors, and sarcomas: Opportunities and challenges. Pediatr Blood Cancer. 2021 May;68 Suppl 2:e28439. doi: 10.1002/pbc.28439. Epub 2020 Aug 22. Review.

name: Radiation Oncology University of Cincinnati and Cincinnati Children's Hospital  Cincinnati Ohio

family: Vatner

full_name: Ralph Vatner

given: Ralph

name: Neurosurgery Northwestern University  Chicago Illinois

family: James

full_name: Charles D. James

given: Charles D.

name: Radiation Oncology Northwestern University  Chicago Illinois

family: Sathiaseelan

full_name: Vythialingam Sathiaseelan

given: Vythialingam

name: Greehey Children's Cancer Research Institute University of Texas  San Antonio Texas

family: Bondra

full_name: Kathryn M. Bondra

given: Kathryn M.

family: Kalapurakal

full_name: John A. Kalapurakal

given: John A.

family: Houghton

full_name: Peter J. Houghton

given: Peter J.

journal_name: Pediatric Blood & Cancer

title: Radiation therapy and molecular‐targeted agents in preclinical testing for immunotherapy, brain tumors, and sarcomas: Opportunities and challenges

ReferenceCitation: Martin-Broto J, Hindi N, Lopez-Pousa A, Peinado-Serrano J, Alvarez R, Alvarez-Gonzalez A, Italiano A, Sargos P, Cruz-Jurado J, Isern-Verdum J, Dolado MC, Rincon-Pérez I, Sanchez-Bustos P, Gutierrez A, Romagosa C, Morosi C, Grignani G, Gatti M, Luna P, Alastuey I, Redondo A, Belinchon B, Martinez-Serra J, Sunyach MP, Coindre JM, Dei Tos AP, Romero J, Gronchi A, Blay JY, Moura DS. Assessment of Safety and Efficacy of Combined Trabectedin and Low-Dose Radiotherapy for Patients With Metastatic Soft-Tissue Sarcomas: A Nonrandomized Phase 1/2 Clinical Trial. JAMA Oncol. 2020 Apr 1;6(4):535-541. doi: 10.1001/jamaoncol.2019.6584. Erratum in: JAMA Oncol. 2020 Oct 1;6(10):1641.

name: Department of Medical Oncology, University Hospital Virgen del Rocío, Sevilla, Spain

name: TERABIS Group, IBiS (Instituto de Biomedicina de Sevilla), Sevilla, Spain

family: Martin-Broto

full_name: Javier Martin-Broto

given: Javier

family: Hindi

full_name: Nadia Hindi

given: Nadia

name: Department of Medical Oncology, Santa Creu i Sant Pau Hospital, Barcelona, Spain

family: Lopez-Pousa

full_name: Antonio Lopez-Pousa

given: Antonio

name: CIBERONC (Centro de Investigación Biomédica en Red de Cáncer), Instituto de Salud Carlos III, Madrid, Spain

name: Department of Radiation Oncology, University Hospital Virgen del Rocío, Sevilla, Spain

family: Peinado-Serrano

full_name: Javier Peinado-Serrano

name: Department of Medical Oncology, Gregorio Marañon University Hospital, Madrid, Spain

family: Alvarez

full_name: Rosa Alvarez

given: Rosa

name: Radiotherapy Department, Gregorio Marañon University Hospital, Madrid, Spain

family: Alvarez-Gonzalez

full_name: Ana Alvarez-Gonzalez

given: Ana

name: Department of Medical Oncology, Institut Bergonié, Bordeaux, France

family: Italiano

full_name: Antoine Italiano

given: Antoine

name: Department of Radiotherapy, Institut Bergonié, Bordeaux, France

family: Sargos

full_name: Paul Sargos

given: Paul

name: Department of Medical Oncology, University Hospital of the Canary Islands, Tenerife, Spain

family: Cruz-Jurado

full_name: Josefina Cruz-Jurado

given: Josefina

name: Radiation Department, Santa Creu i Sant Pau Hospital, Barcelona, Spain

family: Isern-Verdum

full_name: Josep Isern-Verdum

given: Josep

name: Department of Radiation Oncology, University Hospital of the Canary Islands, Tenerife, Spain

family: Dolado

full_name: Maria Carmen Dolado

given: Maria Carmen

family: Rincon-Pérez

full_name: Inmaculada Rincon-Pérez

given: Inmaculada

family: Sanchez-Bustos

full_name: Paloma Sanchez-Bustos

given: Paloma

name: Department of Hematology, University Hospital Son Espases, Mallorca, Spain

family: Gutierrez

full_name: Antonio Gutierrez

name: Department of Pathology, Vall d’Hebron University Hospital, Barcelona, Spain

family: Romagosa

full_name: Cleofe Romagosa

name: Department of Radiology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy

name: Division of Medical Oncology, Candiolo Cancer Institute, FPO-IRCCS, Candiolo, Italy

name: Division of Radiotherapy, Candiolo Cancer Institute, FPO-IRCCS, Candiolo, Italy

name: Department of Medical Oncology, University Hospital Son Espases, Mallorca, Spain

name: Radiotherapy Department, University Hospital Son Espases, Mallorca, Spain

name: Medical Oncology Department, University Hospital La Paz, Madrid, Spain

name: Health Research Institute of La Paz Hospital (IdiPAZ), Madrid, Spain

name: Department of Radiotherapy, University Hospital La Paz, Madrid, Spain

name: Department of Radiotherapy, Centre Léon Bérard, Lyon, France

name: Department of Biopathology, Institut Bergonié, Bordeaux, France

name: Department of Biopathology, Bordeaux University, Talence, France

name: Department of Medicine, University of Padua School of Medicine, Padua, Italy

name: Department of Radiation Oncology, University Hospital Puerta de Hierro, Madrid, Spain

name: Department of Surgery, Fondazione IRCCS, Istituto Nazionale dei Tumori, Milan, Italy

name: Medical Oncology Department, Centre Léon Bérard, Lyon, France

name: Département of Medicine, Université Claude Bernard Lyon I, Lyon, France

ReferenceCitation: Gronchi A, Hindi N, Cruz J, Blay JY, Lopez-Pousa A, Italiano A, Alvarez R, Gutierrez A, Rincón I, Sangalli C, Pérez Aguiar JL, Romero J, Morosi C, Sunyach MP, Sanfilippo R, Romagosa C, Ranchere-Vince D, Dei Tos AP, Casali PG, Martin-Broto J. Trabectedin and RAdiotherapy in Soft Tissue Sarcoma (TRASTS): Results of a Phase I Study in Myxoid Liposarcoma from Spanish (GEIS), Italian (ISG), French (FSG) Sarcoma Groups. EClinicalMedicine. 2019 Mar 11;9:35-43. doi: 10.1016/j.eclinm.2019.03.007. eCollection 2019 Mar.

repository_institution: Universita Degli Studi Di Milano - Archivio Istituzionale della Ricerca

lead_sponsor: Grupo Espanol de Investigacion en Sarcomas

location_facility:
Centre Léon Berard
Istituto Clinico Humanitas
Istituto Nazionale dei Tumori
Hospital Son Espases
Istituto Ortopedico Rizzoli
Hospital Vall d'Hebrón
Hospital Puerta de Hierro
Candiolo Cancer Institute
Hospital Virgen del Rocío
Hospital Uniersitario La Paz
Hospital Miguel Servet
Centro di Referimento Ocologico
Hospital Universitario Canarias
Hospital Universitario Gregorio Marañón
Institut Bergonié
Hospital Sant Pau


title: Effects of F17464 in Acute Exacerbation of Schizophrenia

acronym: FAST

summary: The purpose of this study is to evaluate the potential efficacy of oral F17464 in comparison to placebo over 6 weeks in patients with acute exacerbation of schizophrenia. Study design: double-blind, randomized, placebo-controlled, parallel-groups, fixed-dose design, multicentre study.

lead_sponsor: Pierre Fabre Medicament

location_country:
France
Romania
Hungary
Russian Federation
Latvia


title: Post-Market Registry of the ACIST Cardiovascular Pressure Measurement (CPM) System and Navvus Catheter in Clinical Practice-ADVANCE-EU

acronym: ADVANCE-EU

summary: This registry will collect real-world clinical evidence on the performance, safety, and usability of the ACIST CPM System and NAVVUS Catheter when used in accordance with approved labeling in a European commercial setting. Up to 60 subjects will have Fractional Flow Reserve (FFR) measurements of coronary lesions attempted with the CPM System and NAVVUS Catheter. All subjects will receive diagnostic treatment according to clinical indications and center standard practice.

study_type: Observational

time_perspective: Prospective

lead_sponsor: Acist Medical Systems

location_country: Italy Germany France Spain

location_facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz

location_facility: Hospital Universitario San Juan de Alicante

intervention_type: Device


title: An Parallel Phase II Study of Tarceva (Erlotinib) in Patients With Advanced Non-small Cell Lung Cancer (Stage IIIB/IV) Not Pre-treated by Chemotherapy Including Dose Escalation to Toxicity in Current and Former Smokers

summary: This study will evaluate the efficacy and safety of Tarceva in two groups of patients with non-small cell lung cancer who have not been pre-treated with chemotherapy. One group, consisting of patients who have never smoked, will receive Tarceva 150 mg/day, and the other group, consisting of current/former smokers, will receive Tarceva 150 mg/day increasing to a maximum of 300 mg/day. The anticipated time on study treatment is 1-2 years.

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
Netherlands
France
Spain
Germany
United Kingdom


title: PENTA15: Plasma Pharmacokinetic Study of Once Versus Twice Daily Abacavir as Part of Combination Antiretroviral Therapy in Children With HIV-1 Infection Aged 3 Months to <36 Months

summary:
To compare the plasma pharmacokinetic (PK) parameters of q24h versus q12h dosing of abacavir in HIV-1-infected infants and children aged 3 months to 36 months

The secondary objectives of PENTA15 were:

To compare the plasma PK parameters of q24h versus q12h dosing of lamivudine in HIV-1-infected infants and children aged 3 months to 36 months who were receiving lamivudine in combination with abacavir

To compare age-related differences in the PK parameters of q24h versus q12h dosing of abacavir and lamivudine infants and children in 3 age groups (≥3 to <12 months, ≥12 to <24 months and ≥24 to <36 months)

To describe child and family acceptability of and adherence to q24h compared to q12h dosage regimens of abacavir and lamivudine

ReferenceType: background

ReferenceCitation: Paediatric European Network for Treatment of AIDS (PENTA). Pharmacokinetic study of once-daily versus twice-daily abacavir and lamivudine in HIV type-1-infected children aged 3-<36 months. Antivir Ther. 2010;15(3):297-305. doi: 10.3851/IMP1532.

lead_sponsor: PENTA Foundation

location_country:
Italy
France
Spain
Germany
United Kingdom

location_facility: Immundefekt-Ambulanz, Dr. von Haunersches Kinderspital

location_facility: Clinica Pediatrica, Università di Padova

intervention_type: Other


title: An Open Label Phase I Dose Escalation Trial to Investigate the Maximum Tolerated Dose, Safety, Pharmacokinetics and Efficacy of Intravenous Volasertib in Combination With Subcutaneous Azacitidine in Patients With Previously Untreated High-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) and Not Candidates for Haematopoetic Stem Cell Transplant

summary: To investigate the maximum tolerated dose (MTD), safety, pharmacokinetics, and efficacy of volasertib in combination with azacitidine in patients with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) and not candidates for hematopoietic stem cell transplant

lead_sponsor: Boehringer Ingelheim

location_country: Germany France

location_facility: 1230.33.49001 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49012 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49003 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49014 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49002 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49006 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49005 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49009 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49011 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.33001 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49010 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49008 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.49004 Boehringer Ingelheim Investigational Site

location_facility: 1230.33.33002 Boehringer Ingelheim Investigational Site


title: A Phase IIa, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety and Efficacy of 28 Day Oral Administration of BAY85-8501 in Patients With Non-Cystic Fibrosis Bronchiectasis

summary:
The primary objective of this study is to assess the safety and tolerability of 28 day oral administration of BAY85-8501 versus placebo in subjects with non-CF Bronchiectasis (BE).

The secondary objectives are to examine the effect of BAY85-8501 on pulmonary function, biomarkers of inflammation and tissue damage, and the impact on overall health and perceived well-being and to evaluate the pharmacokinetics of BAY85-8501.

lead_sponsor: Bayer


summary: Phase 3 efficacy and safety study of CD07805/47 topical gel in subjects with facial erythema associated with rosacea. The study hypothesis is that CD07805/47 gel, applied topically once daily is more efficacious than vehicle and provides an acceptable safety profile in the treatment of facial erythema associated with rosacea

lead_sponsor: Galderma R&D

location_country:
Sweden
Russian Federation
France


title: The European Paediatric Network for Haemophilia Management and the PedNet Haemophilia Registry

acronym: PedNet

summary:
Rationale:

Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias.

Objective:

To collect data on bleeding during neonatal period, endogenous (genetic) and exogenous (treatment-related) determinants of inhibitor development and long term outcome.

ReferenceType: result

ReferenceCitation: van den Berg HM, Gouw SC, van der Bom JG. Factor VIII products and inhibitors in severe hemophilia A. N Engl J Med. 2013 Apr 11;368(15):1457. doi: 10.1056/NEJMc1301995.

journal_name: New England Journal of Medicine

publisher: Massachusetts Medical Society

publisher_dissemination: Massachusetts Medical Society

publisher_group: Massachusetts Medical Society

publisher_normalized: Massachusetts Medical Society

title: Factor VIII Products and Inhibitors in Severe Hemophilia A

ReferenceCitation: Gouw SC, van den Berg HM, Fischer K, Auerswald G, Carcao M, Chalmers E, Chambost H, Kurnik K, Liesner R, Petrini P, Platokouki H, Altisent C, Oldenburg J, Nolan B, Garrido RP, Mancuso ME, Rafowicz A, Williams M, Clausen N, Middelburg RA, Ljung R, van der Bom JG; PedNet and Research of Determinants of INhibitor development (RODIN) Study Group. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013 May 16;121(20):4046-55. doi: 10.1182/blood-2012-09-457036. Epub 2013 Apr 3.

name: Department of Paediatrics, Wilhelmina Children’s Hospital, University Medical Center Utrecht, Utrecht, The Netherlands;

family: Gouw

full_name: Samantha C. Gouw

given: Samantha C.

name: Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands;

family: van den Berg

full_name: H. Marijke van den Berg

given: H. Marijke

name: Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands;

family: Fischer

full_name: Kathelijn Fischer

given: Kathelijn

name: Gesundheit Nord, Klinikum Bremen Mitte, Prof.-Hess-Kinderklinik, Bremen, Germany;

family: Auerswald

full_name: Günter Auerswald

given: Günter

name: Division of Haematology/Oncology, Hospital for Sick Children, Toronto, Canada;

family: Carcao

full_name: Manuel Carcao

given: Manuel

name: Department of Haematology, Royal Hospital for Sick Children, Yorkhill, Glasgow, UK;

family: Chalmers

full_name: Elizabeth Chalmers

given: Elizabeth

name: Service d’hématologie pédiatrique, Hôpital La Timone & Aix-Marseille Univ, Marseille, France;

family: Chambost

full_name: Hervé Chambost

given: Hervé

name: Dr. v. Haunersches Kinderspital, University of Munich, Munich, Germany;

family: Kurnik

full_name: Karin Kurnik

given: Karin

name: Hemophilia Center, Department of Haematology, Great Ormond Street Hospital for children, London, UK;

family: Liesner

full_name: Ri Liesner

given: Ri

name: Department of Pediatrics, Clinic of Coagulation Disorders, Karolinska Hospital, Stockholm, Sweden;

family: Petrini

full_name: Pia Petrini

given: Pia

name: St. Sophia Children’s Hospital, Haemophilia-Haemostasis Unit, Athens, Greece;

family: Platokouki

full_name: Helen Platokouki

given: Helen

name: Unitat Hemofilia, Hospital Traumatologica, Hospital Vall d’Hebron, Barcelona, Spain;

family: Altisent

full_name: Carmen Altisent

given: Carmen

name: Institut für Experimentelle Hämatologie und Transfusionsmedizin, Universitätsklinikum Bonn, Bonn, Germany;

family: Oldenburg

full_name: Johannes Oldenburg

given: Johannes

name: Department of Paediatric Haematology, St. James's Hospital, Dublin, Ireland;

family: Nolan

full_name: Beatrice Nolan

given: Beatrice

name: Hospital General Unidad de Hemofilia, Hospitales Universitarios Virgen del Rocio, Sevilla, Spain;

family: Garrido

full_name: Rosario Pérez Garrido

given: Rosario Pérez

name: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy;

family: Mancuso

full_name: M. Elisa Mancuso

given: M. Elisa

name: Centre Régional de Traitement de l'Hémophilie et Autres Maladies Bicetre, Service Hématologique, Paris, France;

family: Rafowicz

full_name: Anne Rafowicz

given: Anne

name: Department of Haematology, The Children’s Hospital, Birmingham, UK;

family: Williams

full_name: Mike Williams

given: Mike

name: Department of Pediatrics, University Hospital of Aarhus at Skejby, Aarhus, Denmark;

family: Clausen

full_name: Niels Clausen

given: Niels

name: Center for Clinical Transfusion Research, Sanquin Foundation, Leiden, The Netherlands;

family: Middelburg

full_name: Rutger A. Middelburg

given: Rutger A.

name: Department of Pediatrics and Malmö Centre for Thrombosis and Haemostasis, Skånes Universitetssjukhus, Malmö, Sweden; and

family: Ljung

full_name: Rolf Ljung

given: Rolf

name: Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, The Netherlands

ReferenceCitation: Carcao MD, van den Berg HM, Ljung R, Mancuso ME; PedNet and the Rodin Study Group. Correlation between phenotype and genotype in a large unselected cohort of children with severe hemophilia A. Blood. 2013 May 9;121(19):3946-52, S1. doi: 10.1182/blood-2012-11-469403. Epub 2013 Mar 12.

name: Child Health Evaluative Sciences, Research Institute, Division of Haematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, ON Canada;

name: Department of Laboratory and Pharmacy, University Hospital Utrecht, Utrecht, the Netherlands;

name: Lund University, Departments of Paediatrics and Mälmo Centre for Thrombosis and Haemostasis, Skåne University Hospital, Sweden; and

name: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy

ReferenceCitation: Clausen N, Petrini P, Claeyssens-Donadel S, Gouw SC, Liesner R; PedNet and Research of Determinants of Inhibitor development (RODIN) Study Group. Similar bleeding phenotype in young children with haemophilia A or B: a cohort study. Haemophilia. 2014 Nov;20(6):747-55. doi: 10.1111/hae.12470. Epub 2014 Jun 3.

name: Department of Pediatrics; University Hospital of Aarhus at Skejby; Aarhus Denmark

name: Department of Pediatrics; Clinic of Coagulation Disorders; Karolinska Hospital; Stockholm Sweden

name: Centre Regional d'Haemophilie; Centre Hospitale Universitaire; Toulouse France

name: Department of Pediatric Hematology; Emma Children's Hospital; Academic Medical Center; Amsterdam The Netherlands

name: Haemophilia Comprehensive Care Centre; Great Ormond Street Hospital for Children NHS Foundation Trust; London UK

name: PedNet and Research of Determinants of Inhibitor development (RODIN) Study Group

title: Similar bleeding phenotype in young children with haemophilia A or B: a cohort study

ReferenceCitation: Fischer K, Ljung R, Platokouki H, Liesner R, Claeyssens S, Smink E, van den Berg HM. Prospective observational cohort studies for studying rare diseases: the European PedNet Haemophilia Registry. Haemophilia. 2014 Jul;20(4):e280-6. doi: 10.1111/hae.12448. Epub 2014 May 2.

name: Julius Center for Health Sciences and Primary Care; University Medical Center Utrecht; Utrecht the Netherlands

name: Van Creveldkliniek; University Medical Center Utrecht; Utrecht the Netherlands

name: Paediatric Clinic and Malmö Centre for Thrombosis and Haemostasis; Lund University; Skånes Universitetssjukhus; Malmö Sweden

name: Haemophilia Centre and Haemostasis Unit; St. Sophia Children's Hospital; Athens Greece

name: Department of Haematology; Haemophilia Centre; Great Ormond Street Hospital for Children; Toulouse France

name: Haemophilia Centre; Purpan Hospital; Toulouse France

ReferenceCitation: Gouw SC, van der Bom JG, Ljung R, Escuriola C, Cid AR, Claeyssens-Donadel S, van Geet C, Kenet G, Mäkipernaa A, Molinari AC, Muntean W, Kobelt R, Rivard G, Santagostino E, Thomas A, van den Berg HM; PedNet and RODIN Study Group. Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med. 2013 Jan 17;368(3):231-9. doi: 10.1056/NEJMoa1208024.

title: Factor VIII Products and Inhibitor Development in Severe Hemophilia A

ReferenceCitation: Andersson NG, Auerswald G, Barnes C, Carcao M, Dunn AL, Fijnvandraat K, Hoffmann M, Kavakli K, Kenet G, Kobelt R, Kurnik K, Liesner R, Mäkipernaa A, Manco-Johnson MJ, Mancuso ME, Molinari AC, Nolan B, Perez Garrido R, Petrini P, Platokouki HE, Shapiro AD, Wu R, Ljung R. Intracranial haemorrhage in children and adolescents with severe haemophilia A or B - the impact of prophylactic treatment. Br J Haematol. 2017 Oct;179(2):298-307. doi: 10.1111/bjh.14844. Epub 2017 Jul 12.

name: Centre for Thrombosis and Haemostasis; Skåne University Hospital; Malmö Sweden

name: Klinikum Bremen-Mitte; Professor Hess Children's Hospital; Bremen Germany

name: Haematology; Royal Children's Hospital; Melbourne Vic. Australia

name: Division of Haematology/Oncology; Hospital for Sick Children; Toronto ON Canada

name: Division of Hematology, Oncology and Bone Marrow Transplant; Nationwide Children's Hospital and Ohio State University School of Medicine; Columbus OH USA

name: Department of Paediatric Haematology; Academic Medical Centre; University of Amsterdam; Amsterdam The Netherlands

name: Department of Pediatrics and Adolescent Medicine; University Hospital Rigshospitalet; Copenhagen Denmark

name: Department of Haematology; Ege University Children's Hospital; Izmir Turkey

name: National Haemophilia Centre; Tel Hashomer and the Sackler Medical School; Tel Aviv University; Tel Aviv Israel

name: Hämophiliezentrum; Wabern and Children's Hospital of the University of Bern; Bern Switzerland

name: Dr. von Hauner Children's Hospital; University of Munich; Munich Germany

name: Great Ormond Street Hospital for Children NHS Foundation Trust; Haemophilia Centre; London UK

name: Children's Hospital and Haematology; Cancer Center; Helsinki University Hospital; University of Helsinki; Helsinki Finland

name: Department of Pediatrics; University of Colorado; Aurora CO USA

name: Angelo Bianchi Bonomi Haemophilia and Thrombosis Centre; Fondazione IRCCS Ca' Granda; Milan Italy

name: Dipartimento di Ematologia ed Oncologia; Unità Trombosi ed Emostasi; Ospedale Pediatrico Giannina Gaslini; Genoa Italy

name: Our Lady's Children's Hospital; Dublin Ireland

name: Haemophilia Unit; Hospital Universitario Virgen del Rocío; Sevilla Spain

name: Department of Paediatrics; Clinic of Coagulation Disorders; Karolinska Hospital; Stockholm Sweden

name: Haemophilia-Haemostasis Unit; St. Sophia Children's Hospital; Athens Greece

name: Indiana Hemophilia and Thrombosis Center; Indianapolis IN USA

name: Hematology-Oncology Center; Beijing Children's Hospital; Capital Medical University; National Center for Children's Health; Beijing China

name: Department of Clinical Sciences Lund; Lund University; Lund Sweden

ReferenceCitation: Mancuso ME, Fischer K, Santagostino E, Oldenburg J, Platokouki H, Königs C, Escuriola-Ettingshausen C, Rivard GE, Cid AR, Carcao M, Ljung R, Petrini P, Altisent C, Kenet G, Liesner R, Kurnik K, Auerswald G, Chambost H, Mäkipernaa A, Molinari AC, Williams M, van den Berg HM; European Pediatric Network for Haemophilia Management (PedNet) the REMAIN (REal life MAnagement of children with INhibitors) Study Group. Risk Factors for the Progression from Low to High Titres in 260 Children with Severe Haemophilia A and Newly Developed Inhibitors. Thromb Haemost. 2017 Dec;117(12):2274-2282. doi: 10.1160/TH17-01-0059. Epub 2017 Dec 6.

name: on behalf of the European Pediatric Network for Haemophilia Management (PedNet) the REMAIN (REal life MAnagement of children with INhibitors) Study Group

ReferenceCitation: Andersson NG, Chalmers EA, Kenet G, Ljung R, Mäkipernaa A, Chambost H; PedNet Haemophilia Research Foundation. Mode of delivery in hemophilia: vaginal delivery and Cesarean section carry similar risks for intracranial hemorrhages and other major bleeds. Haematologica. 2019 Oct;104(10):2100-2106. doi: 10.3324/haematol.2018.209619. Epub 2019 Feb 21.

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

repository_normalized: HELDA - Digital Repository of the University of Helsinki

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

repositories: HELDA - Digital Repository of the University of Helsinki

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

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

publisher: Ferrata Storti Foundation (Haematologica)

publisher_dissemination: Ferrata Storti Foundation (Haematologica)

publisher_group: Ferrata Storti Foundation (Haematologica)

publisher_normalized: Ferrata Storti Foundation (Haematologica)

ReferenceCitation: van den Berg HM, Fischer K, Carcao M, Chambost H, Kenet G, Kurnik K, Königs C, Male C, Santagostino E, Ljung R; PedNet Study Group. Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A. Blood. 2019 Jul 18;134(3):317-320. doi: 10.1182/blood.2019000658. Epub 2019 Jun 11.

name: PedNet Haemophilia Research Foundation, Baarn, The Netherlands;

name: Van Creveld Kliniek, University Medical Center Utrecht, Utrecht, The Netherlands;

name: Division of Haematology/Oncology, Hospital for Sick Children, Toronto, ON, Canada;

name: Centre de Recherche en Cardiovasculaire et Nutrition (C2VN), Aix Marseille University, Institut National de la Recherche Agronomique (INRA), INSERM, Marseille, France;

name: Centre for Bleeding Disorders, La Timone Children Hospital, Assistance Publique–Hopitaux de Marseille (APHM), Marseille, France;

name: National Hemophilia Center, Ministry of Health, Sheba Medical Center, Tel Hashomer, Israel;

name: Dr V. Haunersches Kinderspital, University of Munich, Munich, Germany;

name: Clinical and Molecular Hemostasis, Department of Pediatrics and Adolescent Medicine, J .W. Goethe University Hospital, Frankfurt, Germany;

name: Department of Paediatrics, Medical University Hospital of Vienna, Vienna, Austria;

name: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy; and

name: Department of Pediatrics, Malmö-Lund University, Lund, Sweden

ReferenceCitation: Male C, Andersson NG, Rafowicz A, Liesner R, Kurnik K, Fischer K, Platokouki H, Santagostino E, Chambost H, Nolan B, Königs C, Kenet G, Ljung R, Van den Berg M. Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study. Haematologica. 2021 Jan 1;106(1):123-129. doi: 10.3324/haematol.2019.239160.

ReferenceCitation: van den Berg HM, Mancuso ME, Königs C, D'Oiron R, Platokouki H, Mikkelsen TS, Motwani J, Nolan B, Santagostino E; European Pediatric Network for Haemophilia Management (PedNet). ITI Treatment is not First-Choice Treatment in Children with Hemophilia A and Low-Responding Inhibitors: Evidence from a PedNet Study. Thromb Haemost. 2020 Aug;120(8):1166-1172. doi: 10.1055/s-0040-1713097. Epub 2020 Jun 22.

name: PedNet Foundation, Baarn, The Netherlands

name: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Ospedale Maggiore Policlinico, Fondazione IRCCS Ca' Granda, Milan, Italy

name: Department of Paediatrics and Adolescent Medicine, University Hospital Frankfurt, Frankfurt, Germany

name: Centre de Référence pour le Traitement des Maladies Hémorragiques, Hôpital Bicêtre, Paris, France

name: Haemophilia Centre, Haemostasis Unit, “Aghia Sophia” Children's Hospital, Athens, Greece

name: Division of Pediatric and Adolescent Medicine, Department of Clinical Medicine, Aarhus University Hospital, Aarhus, Denmark

name: Haematology Department, Birmingham Women's and Children's Hospital, Birmingham, United Kingdom

name: Our Lady's Children's Hospital Crumlin, Dublin, Ireland

name: on behalf of the European Pediatric Network for Haemophilia Management (PedNet)

ReferenceCitation: Andersson NG, Wu R, Carcao M, Claeyssens-Donadel S, Kobelt R, Liesner R, Mäkipernaa A, Ranta S, Ljung R; ICH study group. Long-term follow-up of neonatal intracranial haemorrhage in children with severe haemophilia. Br J Haematol. 2020 Jul;190(2):e101-e104. doi: 10.1111/bjh.16740. Epub 2020 Jun 9.

name: Department of Clinical Sciences, Pediatrics Lund University Lund Sweden

name: Department for Thrombosis and Hemostasis Skåne University Hospital Malmö Sweden

name: Hematology‐Oncology Center Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China

name: Division of Haematology/Oncology and Child Health Evaluative Sciences Research Institute Hospital for Sick Children Toronto ON Canada

name: Chu Purpan Pav. Centre Hospitalier Lefebvre Centre Regional de l'Hemophilie Toulouse France

name: Hämophiliezentrum Wabern and Children's Hospital of the University of Bern Bern Switzerland

name: Great Ormond Hospital for Children NHS Trust Haemophilia Centre London UK

name: Children's Hospital and Haematology Helsinki University Hospital University of Helsinki Helsinki Finland

name: Childhood Cancer Research Unit Department of Women's and Children's Health Karolinska Institutet Stockholm Sweden

name: Astrid Lindgren Children´s Hospital Karolinska University Hospital Stockholm Sweden

title: Long‐term follow‐up of neonatal intracranial haemorrhage in children with severe haemophilia

ReferenceCitation: Platokouki H, Fischer K, Gouw SC, Rafowicz A, Carcao M, Kenet G, Liesner R, Kurnik K, Rivard GE, van den Berg HM. Vaccinations are not associated with inhibitor development in boys with severe haemophilia A. Haemophilia. 2018 Mar;24(2):283-290. doi: 10.1111/hae.13387. Epub 2017 Dec 15.

name: Van Creveldkliniek; University Medical Centre Utrecht; Utrecht The Netherlands

name: Department of Paediatric Haematology; Academic Medical Centre Amsterdam; Emma Children's Hospital; Amsterdam The Netherlands

name: Centre de Référence pour le Traitement des Maladies Hémorragiques (CRTH); Hôpital Bicêtre; Paris France

name: Division of Haematology/Oncology; Department of Paediatrics and Child Health Evaluative Sciences; Research Institute; Hospital for Sick Children; Toronto ON Canada

name: National Haemophilia Centre; Ministry of Health; Sheba Medical Center; Tel Hashomer Israel

name: Department of Haematology; Haemophilia Centre; Great Ormond Street Hospital for Children; London UK

name: Dr. v. Haunersches Kinderspital; University of Munich; Munich Germany

name: Division of Haematology/Oncology; Hôpital St. Justine; Montréal QC Canada

name: PedNet Haemophilia Research Foundation; Mollerusstraat1 3743BW Baarn The Netherlands

title: Vaccinations are not associated with inhibitor development in boys with severe haemophilia A

ReferenceCitation: Khair K, Ranta S, Thomas A, Lindvall K; PedNet study group. The impact of clinical practice on the outcome of central venous access devices in children with haemophilia. Haemophilia. 2017 Jul;23(4):e276-e281. doi: 10.1111/hae.13241. Epub 2017 May 24.

name: Great Ormond Street Hospital for Children NHS Foundation Trust; London UK

name: Karolinska University Hospital; Stockholm Sweden

name: Royal Hospital for Sick Children; Edinburgh UK

ReferenceCitation: Hashemi SM, Fischer K, Moons KGM, van den Berg HM; PedNet Study group. Validation of the prediction model for inhibitor development in PUPs with severe haemophilia A. Haemophilia. 2016 Mar;22(2):e116-e118. doi: 10.1111/hae.12895. Epub 2016 Feb 8.

name: Julius Center for Health Sciences and Primary Care; University Medical Center; Utrecht the Netherlands

name: Van Creveldkliniek; University Medical Center; Utrecht the Netherlands

title: Validation of the prediction model for inhibitor development in PUPs with severe haemophilia A

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
Spain
Germany
Finland
France
Czechia
Greece
Israel
Norway
Ireland
Belgium
Portugal
Denmark
Canada
United Kingdom

location_facility: Unidad de Coagulopatias Congenitas Hospital Universitario la Fe

location_facility: Children's Hospital Helsinki University Hospital

location_facility: Division of Haematology/Oncology Hospital for Sick Children

location_facility: Department of Haematology Royal Hospital for Sick Children

location_facility: Division of Hematology/Oncology Hôpital St Justine

location_facility: Haemophilia Comprehensive Care Centre, Centre for Thrombosis and Haemostasis Children's University Hospital Brno

location_facility: Medical University of Vienna - Department of Paediatrics

location_facility: The National Hemophilia Center Sheba Medical Center, Tel Hashomer

location_facility: Dept of Paediatric Haematology Our Lady's Children's Hospital for Sick Children Crumlin

location_facility: Department of Paediatric Haematology/oncology - University Hospital Motol

location_facility: Universitäts-Klinik für Kinder- und Jugendheilkunde

location_facility: Dr. v. Haunersches Kinderspital University of Munich

location_facility: Birmingham Children's Hospital NHS Trust - Department of Haematology

location_facility: Inselspital Bern, University Children's Hospital

location_facility: Hospital General Unidad de Hemofilia 1 Sur Hospitales Universitarios Virgen del Rocio

location_facility: Institut für Experimentelle Hämatologie und Transfusionsmedizin Universitätsklinikum Bonn

location_facility: Service Hématologique Centre Regional Traitement d'Hemophilie Bicetre

location_facility: Service of Pediatric Haematology University Hospital Leuven

location_facility: A. Bianchi Bonomi Hemophilia and Thrombosis Centre IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: Department of Pediatrics, Clinic of Coag. Disorders Karolinska Hospital

location_facility: University Hospital Frankfurt & Goethe University - Clinical and Molecular Hemostasis, Department of Pediatrics

location_facility: Department of Pediatrics Århus Kommunehospital Skejby Sygehus

location_facility: Haemophilia-Haemostasis Unit St. Sophia Children's Hospital

location_facility: Haemophila Center Great Ormond Street Hospital for Children

location_facility: Service d'Hématologie Pédiatrique Hôpital Universitaire La Timone

location_facility: Unidad de Coagulopatías Hospital Universitario La Paz

location_facility: Van Creveld Kliniek University Medical Center Utrecht

location_facility: Centro Hospitalar São João, S. Imuno-hemoterapia

location_facility: Centre de traitement des hémophiles Hôpital Universitaire Purpan


title: A Phase Ib, Open-label, Multicenter Study of Oral LXH254-centric Combinations in Adult Patients With Advanced or Metastatic KRAS or BRAF Mutant Non-Small Cell Lung Cancer or NRAS Mutant Melanoma

summary: To characterize safety and tolerability and identify a recommended dose and regimen for the LXH254 in combination with LTT462 or trametinib or ribociclib.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
Sweden
Germany
France
Poland
Israel
Belgium
Australia
Spain
United States
Korea, Republic of

location_facility: Sarah Cannon Research Institute Tennessee Oncology

location_facility: University of Texas MD Anderson Cancer Center


title: The Guard Control Trial - Randomized, Controlled, Muti-centric, Clinical Study to Assess Whether Type 1 Diabetic Patients in Poor Glycemic Control Can Improve Using the Real-time Values of the Guardian RT Versus Conventional Self-Monitoring Blood Glucose

summary: The purpose of this study is to determine whether Type 1 diabetic patients using the Guardian RT glucose sensor can improve glycemic control over a 12-week period, compared to patients using self-monitoring blood glucose testing (SMBG) alone.

lead_sponsor: Medtronic Diabetes

location_country:
Italy
Sweden
United Kingdom
France
Israel
Germany
Slovenia

location_facility:
Hospital Sud Francilien
Klinik fur Allgemeine Charite
CH Robert Debre
University Children's Hospital
Royal Bournemouth Hospital
Huddinge University Hospital
Schneider Children Centre
U.O. Medicina Generale


title: Topical Ophthalmic AGN-195263 for the Treatment of Evaporative Dry Eye

summary: The objectives of this study are twofold •To evaluate the safety and efficacy of 0.1% AGN-195263 administered twice daily compared to its vehicle in patients with evaporative dry eye (EDE) •To evaluate the systemic pharmacokinetics of 0.1% AGN-195263 administered twice daily in patients with EDE

lead_sponsor: Allergan

location_country:
Italy
United States
Philippines
France
Czechia
Poland
Hungary
Spain
Turkey
Germany
United Kingdom
Taiwan

location_facility: Rapallo Hospital, Opthalmology Department

location_facility: Uniwersyteck Szpital Kliniczny im. Jana Mikulicza-Radeckiego

location_facility: Gutenberg University Medical School, Dept of Ophthalmology

location_facility: Ospedale San Giuseppe, Universita di Milano

location_facility: University of Szeged Szent-Györgyi Albert Clinical Center, Faculty of Medicine, Dept of Ophthalmology

location_facility: CHU Hopital Morvan (Bat 4) Service Daviel Ophtalmologie

location_facility: Specjalistyczna Praktyka Lekarska prof. Edward Wylegala

location_facility: CHU de Bordeaux, Service d'Ophtalmologie

location_facility: Institto de Microcirugia Ocular C/Josep Maria Llado

location_facility: University of Debrecen, Dept of Opthalmology, (DE OEC Szemklinika)

location_facility: Newcastle University School of Medicine, Dept of Ophthamology

location_facility: Ludwig-Maximilians-University, Dept of Ophthalmology

location_facility: University of Pisa, Neurosciences - Section of Ophthalmology

location_facility: Clinico de Barcelona Casa de la Maternidad

location_facility: University Hospital of Cologne, Dept of Ophthalmology

location_facility: University of Valladolid, Facultad Medicina

location_facility: Istituto Di Ricovero E Cura A Carattere Scientifico (IRCCS) - Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)

location_facility: Dept. of Ophthalmology Semmelweis University


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

lead_sponsor: GlaxoSmithKline

location_country:
United States
France
Australia
Spain
Canada
United Kingdom
Korea, Republic of

title: A Phase I/II Dose Escalation and Expansion Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of GSK525762 in Combination With Fulvestrant in Subjects With Hormone Receptor-positive/HER2-negative (HR+/HER2-) Advanced or Metastatic Breast Cancer

summary: This is a combination Phase I and Phase II study, with an aim to evaluate the combination of GSK525762 and fulvestrant in women with HR+/HER2- advanced or metastatic breast cancer, who have disease that has progressed after prior treatment with at least one line of endocrine therapy. The objectives of the study are to first identify, in open-label single-arm Phase I, a recommended Phase II dose of GSK525762 that may be combined safely with fulvestrant. Phase I will follow a modified toxicity probability interval (mTPI) design, and a sentinel group will be evaluated first for dose-limiting toxicity and further expanded to collect additional safety data. This will be followed by a double-blind, randomized controlled Phase II, to identify the clinical activity of the two study treatments when given in combination. The composition of Phase II will be selected at the end of Phase I.


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: Baxalta now part of Shire

location_country:
Italy
Brazil
United States
Serbia
Mexico
Germany
France
Czechia
Greece
Poland
Slovakia
Argentina
Spain
Turkey
Denmark
Canada
United Kingdom
Colombia

location_facility: Dokuz Eylul University Faculty of Medicine

location_facility: Azienda Ospedaliera Universitaria Policlinico G. Martino

location_facility: Instituto de Neurologia de Curitiba - Hospital Ecoville

location_facility: Toronto General Hospital, University Health Network

location_facility: Azienda Ospedaliero Universitaria San Martino

location_facility: Institucion Prestadora de Servicios de Salud de la Universidad de Antioquia "IPS UNIVERSITARIA"

location_facility: Groupe Hospitalier Pellegrin - Hôpital Pellegrin

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie

location_facility: Selcuk Universitesi Selcuklu Tip Fakultesi Hastanesi

location_facility: Instituto Nacional de Ciencias Médicas y Nutricion Dr. Salvador Zubiran

location_facility: Fondazione Istituto Neurologico Casimiro Mondino

location_facility: Azienda Ospedaliero-Universitaria Santa Maria della Misericordia

location_facility: Azienda Ospedaliero Universitaria Pisana

location_facility: Uniwersytecki Szpital Kliniczny nr 1 im. Norberta Barlickiego

title: Long-Term Tolerability and Safety of Immune Globulin Infusion 10% (Human) With Recombinant Human Hyaluronidase (HYQVIA/HyQvia) for the Treatment of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

summary: The purpose of this phase IIIb study is to assess the long-term safety, tolerability, and immunogenicity of the subcutaneous (SC) treatment with Immune Globulin Subcutaneous (IGSC) facilitated with recombinant human hyaluronidase (rHuPH20) (HYQVIA/HyQvia) in participants with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) who have completed Baxalta Clinical Study Protocol 161403 Epoch 1 without CIDP worsening.


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

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

location_country:
Italy
Austria
United States
Netherlands
Germany
France
Japan
Australia
Belgium
Spain
Canada
United Kingdom

location_facility: University of TX MD Anderson Cancer Center

location_facility: H Lee Moffitt Cancer Center and Research Institute

title: An Open Label Phase II Study to Evaluate the Efficacy and Safety of PDR001 in Patients With Advanced or Metastatic, Well-differentiated, Non-functional Neuroendocrine Tumors of Pancreatic, Gastrointestinal (GI), or Thoracic Origin or Poorly-differentiated Gastroenteropancreatic Neuroendocrine Carcinoma (GEP-NEC), That Have Progressed on Prior Treatment.

summary: This study aimed to investigate the efficacy and safety of PDR001 in patients with advanced or metastatic, well-differentiated, non-functional neuroendocrine tumors of pancreatic, gastrointestinal (GI), or thoracic origin or poorly-differentiated gastroenteropancreatic neuroendocrine carcinoma (GEP-NEC) that progressed on prior treatment.


title: An Exploratory, Open Label Multicenter Study to Investigate Pharmacodynamic of RO5083945, a Human Monoclonal Antibody Antagonist of Epidermal Growth Factor Receptor (EGFR), Compared to Cetuximab in Patients With Operable Head and Neck Squamous Cell Carcinoma

summary: This open-label study will assess the pharmacodynamics, safety and efficacy of RO5083945 as compared to cetuximab in patients with head and neck squamous cell carcinoma. Patients will receive at least 2 infusions of either RO5083945 or cetuximab. Anticipated time on study treatment is up to 3 months, and target sample size is <50.

ReferenceCitation: Temam S, Spicer J, Farzaneh F, Soria JC, Oppenheim D, McGurk M, Hollebecque A, Sarini J, Hussain K, Soehrman Brossard S, Manenti L, Evers S, Delmar P, Di Scala L, Mancao C, Feuerhake F, Andries L, Ott MG, Passioukov A, Delord JP. An exploratory, open-label, randomized, multicenter study to investigate the pharmacodynamics of a glycoengineered antibody (imgatuzumab) and cetuximab in patients with operable head and neck squamous cell carcinoma. Ann Oncol. 2017 Nov 1;28(11):2827-2835. doi: 10.1093/annonc/mdx489.

family: Temam

full_name: S. Temam

given: S.

family: Spicer

full_name: J. Spicer

given: J.

family: Farzaneh

full_name: F. Farzaneh

given: F.

family: Soria

full_name: J.C. Soria

family: Oppenheim

full_name: D. Oppenheim

given: D.

family: McGurk

full_name: M. McGurk

given: M.

family: Hollebecque

full_name: A. Hollebecque

given: A.

family: Sarini

full_name: J. Sarini

family: Hussain

full_name: K. Hussain

given: K.

family: Soehrman Brossard

full_name: S. Soehrman Brossard

family: Manenti

full_name: L. Manenti

given: L.

family: Evers

full_name: S. Evers

family: Delmar

full_name: P. Delmar

given: P.

family: Di Scala

full_name: L. Di Scala

family: Mancao

full_name: C. Mancao

given: C.

family: Feuerhake

full_name: F. Feuerhake

family: Andries

full_name: L. Andries

family: Ott

full_name: M.G. Ott

family: Passioukov

full_name: A. Passioukov

family: Delord

full_name: J.P. Delord

journal_name: Annals of Oncology

license: elsevier-specific: oa user license

license_normalized: elsevier-specific

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

repository_institution: PubMed Central - Europe PMC

location_country:
Italy
Netherlands
France
Spain
United Kingdom


title: A Phase II Study to Compare the Effect of a Compression Device to That of a 4-layer Compression System on Subjects With Venous Leg Ulcers.

summary: A prospective, Phase II, stratified, randomized study to compare the safety, ulcer healing, patient compliance (concordance) and resource utilisation of a compression device with IPC mode to a Class 3(c) UK standard graduated compression regime (4- layer system) on subjects with venous leg ulcers.

lead_sponsor: ConvaTec Inc.

location_country:
Germany
Ireland
France
United Kingdom

location_facility: Department of Vascular Surgery, Good Hope Hospital

location_facility: Medical Physics & Bioengineering, Southampton University Hospital

location_facility: Wound Healing Research Unit; Cardiff University

location_facility: Dermatology Day Unit; Monklands Hospital

location_facility: Diving Diseases Research Centre, Hyperbaric Medical Centre

location_facility: The Wilson Practice, Alton Health Centre

location_facility: Institute of Wound Care, The University of Hull


title: Multicenter Phase II Safety and Preliminary Efficacy Study of 2 Dose Regimens of HepaStem in Patients With Acute on Chronic Liver Failure

summary: The study will assess the safety of different dose regimens of HepaStem in cirrhotic Patients with ACLF or with acute decompensation at risk of developing ACLF up to Day 28 of the active study period.

lead_sponsor: Promethera Biosciences

location_country: Belgium France

location_facility:
Cliniques St Luc
Hôpital Paul Brousse
CHU de Liège
UZ Gent
CHU Brugmann
Hôpital de la Croix Rousse
Hôpital Erasme
UZ Antwerpen
KU Leuven
Hôpital Beaujon


title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Safety and Efficacy of a Single Dose of ASN100 for the Prevention of Staphylococcus Aureus Pneumonia in Heavily Colonized, Mechanically Ventilated Subjects

summary: The purpose of this study is the prevention of Staphylococcus aureus pneumonia in mechanically ventilated subjects heavily colonized with S. aureus. Staphylococcus aureus is a human pathogenic bacterium that causes severe infections, including pneumonia and sepsis. Hospital-acquired bacterial pneumonia (HABP) caused by S. aureus, including ventilator-associated bacterial pneumonia (VABP) in mechanically ventilated subjects, is a significant public health threat despite efforts to optimize antibiotic treatment. ASN100 is an investigational monoclonal antibody product that targets the toxins produced by S. aureus to protect subjects from developing S. aureus pneumonia.

primary_purpose: Prevention

lead_sponsor: Arsanis, Inc.

location_country:
Austria
South Africa
India
Serbia
Spain
France
Czechia
Poland
Israel
Georgia
Romania
Hungary
Russian Federation
Portugal
United States
Ukraine

location_facility:
Research Site
Research Site 268-002
Research Site 268-011
Research Site 268-005
Research Site 268-006
Research Site 688-002
Research Site 268-010
Research Site 268-003
Research Site 688-004
Research Site 040-002
Research Site 040-001
Research Site 268-008
Research Site 268-001
Research Site 268-007
Research Site 688-003
Research Site 688-001
Research Site 688-005
Research Site 268-009
Research Site 268-004


title: Evaluation of PillCam™ Colon Capsule Endoscopy (PCCE) in the Visualization of the Colon

summary: To see if PillCam COLON will demonstrate diagnostic yield >80% in detecting significant colonic pathologies when compared to colonoscopy in the target population.

primary_purpose: Diagnostic

ReferenceCitation: Van Gossum A, Munoz-Navas M, Fernandez-Urien I, Carretero C, Gay G, Delvaux M, Lapalus MG, Ponchon T, Neuhaus H, Philipper M, Costamagna G, Riccioni ME, Spada C, Petruzziello L, Fraser C, Postgate A, Fitzpatrick A, Hagenmuller F, Keuchel M, Schoofs N, Devière J. Capsule endoscopy versus colonoscopy for the detection of polyps and cancer. N Engl J Med. 2009 Jul 16;361(3):264-70. doi: 10.1056/NEJMoa0806347. Erratum in: N Engl J Med. 2009 Sep 17;361(12):1220. Navas, Miguel Munoz [corrected to Munoz-Navas, Miguel].

family: Van Gossum

full_name: André Van Gossum

given: André

family: Munoz-Navas

full_name: Miguel Munoz-Navas

given: Miguel

family: Fernandez-Urien

full_name: Iñaqui Fernandez-Urien

given: Iñaqui

family: Carretero

full_name: Cristina Carretero

given: Cristina

family: Gay

full_name: Gérard Gay

given: Gérard

family: Delvaux

full_name: Michel Delvaux

given: Michel

family: Lapalus

full_name: Marie Georges Lapalus

given: Marie Georges

family: Ponchon

full_name: Thierry Ponchon

given: Thierry

family: Neuhaus

full_name: Horst Neuhaus

given: Horst

family: Philipper

full_name: Michael Philipper

given: Michael

family: Costamagna

full_name: Guido Costamagna

given: Guido

family: Riccioni

full_name: Maria Elena Riccioni

given: Maria Elena

family: Spada

full_name: Cristiano Spada

given: Cristiano

family: Petruzziello

full_name: Lucio Petruzziello

given: Lucio

family: Fraser

full_name: Chris Fraser

given: Chris

family: Postgate

full_name: Aymer Postgate

given: Aymer

family: Fitzpatrick

full_name: Aine Fitzpatrick

given: Aine

family: Hagenmuller

full_name: Friedrich Hagenmuller

given: Friedrich

family: Keuchel

full_name: Martin Keuchel

given: Martin

family: Schoofs

full_name: Nathalie Schoofs

given: Nathalie

family: Devière

full_name: Jacques Devière

given: Jacques

title: Capsule Endoscopy versus Colonoscopy for the Detection of Polyps and Cancer

location_facility:
Ospedale Gemelli
CHU Lyon
St.Marks&Northwick Park hospital
Clinica Universitaria de Navarra
AK Altona
CHU Nancy
Evangelisches Krankenhaus


title: Placebo Controlled Trial on the Efficacy of Growth Hormone Replacement Therapy in Patients With Growth Hormone Deficiency After Traumatic Brain Injury.

summary: To establish the effects of genotropin replacement on cognitive function in patients with severe growth hormone deficiency after traumatic brain injury.

lead_sponsor: Pfizer

location_country:
Italy
Sweden
Netherlands
France
Spain
United Kingdom


lead_sponsor: Genzyme, a Sanofi Company

location_country:
France
Slovakia
Hungary
Spain
Korea, Republic of
Taiwan

title: A Phase II, Double-blind, Placebo Controlled, Randomized Study to Assess the Efficacy and Safety of 2 Doses of ZD6474 (Vandetanib) in Combination With FOLFOX vs FOLFOX Alone for the Treatment of Colorectal Cancer in Patients Who Have Failed Therapy With an Irinotecan and Fluoropyrimidine Regimen

summary: The purpose of this study is to determine whether treatment with ZACTIMA (vandetanib) in combination with FOLFOX is more effective than FOLFOX alone for colorectal cancer in patients who have failed therapy with an irinotecan and fluoropyrimidine containing regimen.


title: A Phase I, Open-label Study to Assess the Safety and Tolerability of AZD0530 in Combination With Carboplatin and/or Paclitaxel Chemotherapy in Patients With Solid Tumours

summary: This study will comprise 2 parts, a dose escalation phase and a dose expansion phase. The 2 phases are part of a single study and described by this single protocol. Patients entered into the dose escalation phase will not be entered into the expansion phase. All subjects must be suitable for treatment with either carboplatin and/or paclitaxel.

lead_sponsor: AstraZeneca

location_country:
Norway
United Kingdom
France
Netherlands


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

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

location_country:
Sweden
United Kingdom
Netherlands
Finland
France
Czechia
Bulgaria
Greece
Lithuania
Norway
Ireland
Belgium
Poland
Spain
Estonia
Denmark
Germany
Latvia
Iceland

acronym: OUTSTEP-HF

title: A Multi-center, Prospective, Randomized, Double-blind Study to Assess the Impact of Sacubitril/Valsartan vs. Enalapril on Daily Physical Activity Using a Wrist Worn Actigraphy Device in Adult Chronic Heart Failure Patients

summary: The purpose of this randomized, actively controlled, double-blind study with prospective data collection was to assess differences between sacubitril/valsartan versus enalapril in increasing exercise capacity and non-sedentary physical activity in HFrEF patients. Physical activity was assessed by the 6 minute walk test, and daily physical activity was continuously measured by means of a wrist-worn accelerometry device from 2 weeks before until 12 weeks after start of study therapy (sacubitril/valsartan or enalapril).


title: Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia and Comparison With Matched Historical Patients Treated With Horse ATG and Ciclosporin

summary: To assess the tolerability and effectiveness of rabbit antithymocyte globulin (ATG, Thymoglobuline) with ciclosporin in the first line treatment of patients with acquired severe aplastic anaemia, and patients with non-severe aplastic anaemia and who are transfusion dependent.

ReferenceCitation: Marsh JC, Bacigalupo A, Schrezenmeier H, Tichelli A, Risitano AM, Passweg JR, Killick SB, Warren AJ, Foukaneli T, Aljurf M, Al-Zahrani HA, Höchsmann B, Schafhausen P, Roth A, Franzke A, Brummendorf TH, Dufour C, Oneto R, Sedgwick P, Barrois A, Kordasti S, Elebute MO, Mufti GJ, Socie G; European Blood and Marrow Transplant Group Severe Aplastic Anaemia Working Party. Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. Blood. 2012 Jun 7;119(23):5391-6. doi: 10.1182/blood-2012-02-407684. Epub 2012 Apr 27. Erratum in: Blood. 2013 Jun 20;121(25):5104. Höchsmann, Britta [added].

name: Department of Haematological Medicine, King's College Hospital/King's College London, London, United Kingdom;

family: Marsh

full_name: Judith C. Marsh

given: Judith C.

name: Department of Hematology II, Azienda Ospedaliera Universitaria San Martino, Genoa, Italy;

family: Bacigalupo

full_name: Andrea Bacigalupo

given: Andrea

name: Institute for Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University of Ulm, Ulm, Germany;

family: Schrezenmeier

full_name: Hubert Schrezenmeier

given: Hubert

name: Department of Hematology, University Hospital, Basel, Switzerland;

family: Tichelli

full_name: Andre Tichelli

given: Andre

name: Department of Biochemistry and Medical Biotechnology, Hematology, Federico II University of Naples, Naples, Italy;

family: Risitano

full_name: Antonio M. Risitano

given: Antonio M.

family: Passweg

full_name: Jakob R. Passweg

given: Jakob R.

name: Department of Haematology, Royal Bournemouth Hospital, Bournemouth, United Kingdom;

family: Killick

full_name: Sally B. Killick

given: Sally B.

name: Department of Haematology, University of Cambridge, Cambridge, United Kingdom;

family: Warren

full_name: Alan J. Warren

given: Alan J.

family: Foukaneli

full_name: Theodora Foukaneli

given: Theodora

name: Adult Hematology/HSCT Oncology Centre, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia;

family: Aljurf

full_name: Mahmoud Aljurf

given: Mahmoud

family: Al-Zahrani

full_name: H. A. Al-Zahrani

given: H. A.

name: Department of Oncology and Hematology, University Cancer Centre Hamburg, Hamburg, Germany;

family: Schafhausen

full_name: Philip Schafhausen

given: Philip

name: Department of Hematology, West German Cancer Center, University Hospital Essen, Essen, Germany;

family: Roth

full_name: Alexander Roth

given: Alexander

name: Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany;

family: Franzke

full_name: Anke Franzke

given: Anke

name: Department of Hematology and Oncology, University Hospital of the Rheinisch-Westfälische Technische Hochschule Aachen, Aachen, Germany;

family: Brummendorf

full_name: Tim H. Brummendorf

given: Tim H.

name: Hematology Unit G. Gaslini Childrens' Research Hospital, Genova, Italy;

family: Dufour

full_name: Carlo Dufour

given: Carlo

family: Oneto

full_name: Rosi Oneto

given: Rosi

name: Centre for Medical and Healthcare Education, St George's, University of London, London, United Kingdom;

family: Sedgwick

full_name: Philip Sedgwick

name: European Blood and Marrow Transplant Clinical Trials Office, Department of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands; and

family: Barrois

full_name: Alain Barrois

given: Alain

family: Kordasti

full_name: Shahram Kordasti

given: Shahram

family: Elebute

full_name: Modupe O. Elebute

given: Modupe O.

family: Mufti

full_name: Ghulam J. Mufti

given: Ghulam J.

name: Hospital Saint Louis, Hematology-Transplantation and Université Paris VII, Paris, France

family: Socie

full_name: Gerard Socie

given: Gerard

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country:
Italy
Saudi Arabia
Switzerland
France
Germany
United Kingdom

location_facility: Universitätsklinikum - Institut für klinische Transfusionsmedizin

location_facility: St George's Hospital/ St George's University of London

location_facility: King Faisal Specialist Hospital & Research Cnetre


title: A Phase 2, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of GS-5745 Combined With Nivolumab Versus Nivolumab Alone in Subjects With Unresectable or Recurrent Gastric or Gastroesophageal Junction Adenocarcinoma

summary: The primary objective of this study is to evaluate and compare the efficacy of andecaliximab (GS-5745) in combination with nivolumab versus nivolumab alone in adults with recurrent gastric or gastroesophageal junction (GEJ) adenocarcinoma.

lead_sponsor: Gilead Sciences

location_country:
Italy
France
Poland
Hungary
Belgium
Australia
Spain
United States
United Kingdom


title: A Multicenter Study of the EZH2 Inhibitor Tazemetostat in Adult Subjects With Relapsed or Refractory Malignant Mesothelioma With BAP1 Loss of Function

summary:
This is a Phase 2, multicenter, open-label, 2-part, single-arm, 2-stage study of tazemetostat 800 mg two times a day (BID) administered orally. Screening of subjects to determine eligibility for the study will be performed within 21 days of the first planned dose of tazemetostat.

In Part 1: planned to enroll 12 subjects with relapsed or refractory malignant mesothelioma regardless of BAP1 status will be treated and undergo pharmacokinetics (PK) blood sample collection after a single tazemetostat 800 mg.

Part 2 plans to include 55 subjects with BAP1-deficient relapsed or refractory malignant mesothelioma.

Treatment with tazemetostat will continue until disease progression, unacceptable toxicity or withdrawal of consent, or termination of the study. Response assessment will be evaluated after 6 weeks of treatment and then every 12 weeks thereafter while on study.

ipd_sharing_description: To be determined.

lead_sponsor: Epizyme, Inc.

location_country:
United States
United Kingdom
France

location_facility: University of Leicester & Leicester University Hospitals


title: Randomised, Controlled, Multi-centric, Clinical Study to Assess Whether Type 1 Diabetic Patients in Poor Glycemic Control Can Improve Using the Real-time Values of Guardian T Versus Conventional Self-Monitoring Blood Glucose

summary: The objective of the study is to determine whether patients with poor glycemic control can improve metabolic control using the real-time values of the Guardian® RT compared to conventional self-monitoring blood glucose finger-sticks.