title: A Prospective Multicenter Randomized Study Comparing Photoselective Vaporization of the Prostate With the GreenLight XPS™ Laser System and Transurethral Resection of the Prostate for the Treatment of Benign Prostatic Hyperplasia

summary: The study compares procedural and post procedural outcomes for photoselective vaporization of the prostate (PVP) and transurethral resection of the prostate (TURP) for the treatment of lower urinary tract symptoms due to benign prostatic enlargement causing bladder outlet obstruction. The study requires use of the CE marked GreenLight XPS™ Laser System (GreenLight XPS) or a CE marked monopolar or bipolar loop TURP system for the treatment of benign prostatic hyperplasia (BPH).

ReferenceCitation: Thomas JA, Tubaro A, Barber N, d'Ancona F, Muir G, Witzsch U, Grimm MO, Benejam J, Stolzenburg JU, Riddick A, Pahernik S, Roelink H, Ameye F, Saussine C, Bruyère F, Loidl W, Larner T, Gogoi NK, Hindley R, Muschter R, Thorpe A, Shrotri N, Graham S, Hamann M, Miller K, Schostak M, Capitán C, Knispel H, Bachmann A. A Multicenter Randomized Noninferiority Trial Comparing GreenLight-XPS Laser Vaporization of the Prostate and Transurethral Resection of the Prostate for the Treatment of Benign Prostatic Obstruction: Two-yr Outcomes of the GOLIATH Study. Eur Urol. 2016 Jan;69(1):94-102. doi: 10.1016/j.eururo.2015.07.054. Epub 2015 Aug 15.

family: Thomas

full_name: James A. Thomas

given: James A.

family: Tubaro

full_name: Andrea Tubaro

given: Andrea

family: Barber

full_name: Neil Barber

given: Neil

family: d’Ancona

full_name: Frank d’Ancona

given: Frank

family: Muir

full_name: Gordon Muir

given: Gordon

family: Witzsch

full_name: Ulrich Witzsch

given: Ulrich

family: Grimm

full_name: Marc-Oliver Grimm

given: Marc-Oliver

family: Benejam

full_name: Joan Benejam

given: Joan

family: Stolzenburg

full_name: Jens-Uwe Stolzenburg

given: Jens-Uwe

family: Riddick

full_name: Antony Riddick

given: Antony

family: Pahernik

full_name: Sascha Pahernik

given: Sascha

family: Roelink

full_name: Herman Roelink

given: Herman

family: Ameye

full_name: Filip Ameye

given: Filip

family: Saussine

full_name: Christian Saussine

given: Christian

family: Bruyère

full_name: Franck Bruyère

given: Franck

family: Loidl

full_name: Wolfgang Loidl

given: Wolfgang

family: Larner

full_name: Tim Larner

given: Tim

family: Gogoi

full_name: Nirjan-Kumar Gogoi

given: Nirjan-Kumar

family: Hindley

full_name: Richard Hindley

given: Richard

family: Muschter

full_name: Rolf Muschter

given: Rolf

family: Thorpe

full_name: Andrew Thorpe

given: Andrew

family: Shrotri

full_name: Nitin Shrotri

given: Nitin

family: Graham

full_name: Stuart Graham

given: Stuart

family: Hamann

ReferenceCitation: Bachmann A, Tubaro A, Barber N, d'Ancona F, Muir G, Witzsch U, Grimm MO, Benejam J, Stolzenburg JU, Riddick A, Pahernik S, Roelink H, Ameye F, Saussine C, Bruyère F, Loidl W, Larner T, Gogoi NK, Hindley R, Muschter R, Thorpe A, Shrotri N, Graham S, Hamann M, Miller K, Schostak M, Capitán C, Knispel H, Thomas JA. 180-W XPS GreenLight laser vaporisation versus transurethral resection of the prostate for the treatment of benign prostatic obstruction: 6-month safety and efficacy results of a European Multicentre Randomised Trial--the GOLIATH study. Eur Urol. 2014 May;65(5):931-42. doi: 10.1016/j.eururo.2013.10.040. Epub 2013 Nov 11.

title: 180-W XPS GreenLight Laser Vaporisation Versus Transurethral Resection of the Prostate for the Treatment of Benign Prostatic Obstruction: 6-Month Safety and Efficacy Results of a European Multicentre Randomised Trial—The GOLIATH Study

location_country:
Italy
Austria
Switzerland
Netherlands
France
Belgium
Spain
Germany
United Kingdom

location_facility: Diakoniekrankenhaus Rotenburg (W.) gGmbH

location_facility: RadBound University Njmegen Medical Centre

location_facility: Universitatsklinikum Schleswig-Holstein, Campus Kiel

location_facility: UroForshungs GmbH, im St. hedwig Krankenhaus

location_facility: Unita di chirurgia Urologica mininvasiva,Azienda Ospedatiera Sant'Andrea Hospital

location_facility: Mid Yorkshire NHS Trust Dewsbury & District Hospital

location_facility: Brighton and Sussex University Hospitals NHS Trust

location_facility: Krankenhaus der Barmherzigen Schwestern Linz

location_facility: Basingstoke and North Hampshire NHS Foundation Trust

location_facility: Hospital Iniversitario Fundacion Alcorcon


title: A Phase I Open-Label Dose Escalation Study of the Focal Adhesion Kinase Inhibitor, GSK2256098, in Subjects With Solid Tumors

summary: This study is a Phase I dose escalation study in subjects with solid tumors. Part 1 will identify the maximum tolerated dose (MTD) using a dose-escalation procedure. Following identification of the MTD, enrollment into Parts 2, 3, 4 and 5 may be concurrent. Part 2 will explore further the safety, PK, tolerability, and anti-tumor activity of GSK2256098 in subjects with tumors known to overexpress focal adhesion kinase (FAK). Part 3 will characterize the range of biologically effective doses by assessing pharmacodynamic (PD) markers in hair, skin and tumor tissue at doses that will not go lower than 80 mg or above the MTD dose levels tested during the Phase 1 dose escalation. Part 4 will explore further the safety, PK, tolerability and anti-tumor activity of GSK2256098 in subjects with relapsed glioblastoma multiforme (GBM). The primary objective of this study is to determine the safety, tolerability, and MTD of GSK2256098. Secondary objectives are to characterize the pharmacokinetics (PK) of GSK2256098; to identify a range of biologically active doses; to explore the anti-tumor activity of GSK2256098, and to explore relationships between GSK2256098 PK, PD and clinical endpoints. Part 5 will investigate the time course, the extent of an apparent change in the PK of GSK2256098 following repeated dosing, and screen for potential CYP3A induction as a possible mechanism of reduced systemic exposure of GSK2256098 at Day 15 and later time points. The primary objective of this study is to determine the safety, tolerability, and MTD of GSK2256098.

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Non-Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Brown NF, Williams M, Arkenau HT, Fleming RA, Tolson J, Yan L, Zhang J, Singh R, Auger KR, Lenox L, Cox D, Lewis Y, Plisson C, Searle G, Saleem A, Blagden S, Mulholland P. A study of the focal adhesion kinase inhibitor GSK2256098 in patients with recurrent glioblastoma with evaluation of tumor penetration of [11C]GSK2256098. Neuro Oncol. 2018 Nov 12;20(12):1634-1642. doi: 10.1093/neuonc/noy078. Erratum in: Neuro Oncol. 2020 Jun 9;22(6):894. Swartz, Lisa [removed].

name: NIHR UCLH Clinical Research Facility, University College London Hospitals NHS Foundation Trust, London, UK

name: Department of Oncology, UCL Cancer Institute, London, UK

family: Brown

full_name: Nicholas F Brown

given: Nicholas F

sequence: first

name: Computational Oncology Lab, Institute of Global Health Innovation, South Kensington Campus, Imperial College, London, UK

name: Radiotherapy Department, Charing Cross Hospital, London, UK

family: Williams

full_name: Matthew Williams

given: Matthew

sequence: additional

name: Sarah Cannon Research Institute UK, London, UK

family: Arkenau

full_name: Hendrik-Tobias Arkenau

given: Hendrik-Tobias

name: GlaxoSmithKline, Research Triangle Park, Durham, North Carolina, USA

family: Fleming

full_name: Ronald A Fleming

given: Ronald A

family: Tolson

full_name: Jerry Tolson

given: Jerry

family: Yan

full_name: Li Yan

given: Li

name: PAREXEL International, Durham, North Carolina, USA

family: Zhang

full_name: Jianping Zhang

given: Jianping

name: GlaxoSmithKline, Collegeville, Pennsylvania, USA

family: Singh

full_name: Rajendra Singh

given: Rajendra

family: Auger

full_name: Kurt R Auger

given: Kurt R

family: Lenox

full_name: Laurie Lenox

given: Laurie

name: GlaxoSmithKline Research & Development Ltd, Uxbridge, UK

family: Cox

full_name: David Cox

given: David

name: Imanova Ltd, Centre for Imaging Sciences, London, UK

family: Lewis

full_name: Yvonne Lewis

given: Yvonne

family: Plisson

full_name: Christophe Plisson

given: Christophe

family: Searle

full_name: Graham Searle

given: Graham

family: Saleem

full_name: Azeem Saleem

given: Azeem

name: NIHR/Wellcome Trust Imperial CRF, Imperial Centre for Translational and Experimental Medicine, Hammersmith Hospital, London, UK

family: Blagden

full_name: Sarah Blagden

given: Sarah

family: Mulholland

full_name: Paul Mulholland

given: Paul

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Neuro-Oncology

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via free pdf)

host_type: publisher

license_normalized: no license

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

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

repository_institution: University College London - UCL Discovery

repository_institution: University of Oxford - Oxford University Research Archive (ORA)

repositories:
PubMed Central
UCL Discovery
ora.ox.ac.uk


title: A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

acronym: MARS

summary: The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the long-term effectiveness and safety of Vimizim®, including, but not limited to, the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes in antibody status; to help the medical community with the development of recommendations for monitoring MPS IVA patients and reports on patient outcomes to optimize patient care; to collect data on other treatment paradigms, and evaluate the prevalences of their use and their effectiveness; to characterize the effects and safety of Vimizim treatment 5 years from enrollment in the Registry for patients under 5 years of age; to monitor pregnancy exposure, including maternal, neonatal, and infant outcomes; and to monitor patients who have completed the MOR-005 and MOR-007 clinical trials. These patients will be encouraged to enroll in the applicable Registry Substudy and will be monitored using the MOR-005 and MOR-007 assessment schedules, respectively.

status: Recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: BioMarin Pharmaceutical

location_country:
Italy
Austria
United States
Netherlands
Germany
France
Czechia
Malaysia
Poland
Ireland
Australia
Belgium
Puerto Rico
Portugal
Denmark
Canada
United Kingdom
Taiwan

location_facility: Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust

location_facility: University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit

location_facility: Zentrum für seltene Erkrankungen, Horst-Schmidt-Kliniken

location_facility: Temple Street Children's University Hospital

location_facility: Clinical Study Center, University Pediatric Clinic

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital

location_facility: Azienda Ospedaliero San Gerardo di Monza

location_facility: San Jorge Children's Hospital, Centro de Investigaciones Clinicas

location_facility: National Hospital for Neurology and Neurosurgery

location_facility: Chidren's Heatlh Queensland Hospital and Health Service

location_facility: Great Ormond Street Hospital NHS Foundation Trust

location_facility: Kapiolani Medical Center for Women and Children

location_facility: Academic Medical Center - University of Amsterdam

location_facility: University Hospital Birmingham - Selly Oaks

location_facility: Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania

location_facility: Chang Gung Memorial Hospital Kaohsiung Branch

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

location_facility: University Medical Center, Johannes Gutenberg-University Mainz

location_facility: Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Childrens Hospitals and Clinics of Minnesota

location_facility: NYU-Langone Medical Center School of Medicine

location_facility: Children's Hospital of the King's Daughters

location_facility: Copenhagen University Hospital, Klinik For Sjaeldne Handicap

location_facility: Klinika Dětského a Dorostového Lékařstvi

location_facility: Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra)

location_facility: Rotterdam University Hospital Medical Centre

location_facility: National Cheng Kung University (NCKU) Hospital

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

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Ongoing


type: EudraCT Number

title: A Multicenter, Open Label, Phase I /Randomised Phase II Study to Evaluate Safety, Pharmacokinetics and Efficacy of BIBF 1120 in Comparison With Oral Sorafenib for Advanced Hepatocellular Carcinoma Patients.

summary: The study aim is to determine maximally tolerated dose (MTD) of BIBF 1120 in HCC (hepatocellular cancer) and compare efficacy of BIBF 1120 to Sorafenib in HCC patients

design_allocation: Randomized

lead_sponsor: Boehringer Ingelheim

location_country:
Austria
Netherlands
France
Poland
Romania
Hungary
Germany
United Kingdom

location_facility: 1199.37.49006 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.31001 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.49005 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.44003 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.44004 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.40002 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.48002 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.48003 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.44005 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.49009 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.44001 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.49001 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.43002 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.33001 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.31002 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.44006 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.40003 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.33002 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.48001 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.44008 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.43001 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.49004 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.44002 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.49003 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.36001 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.49010 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.49008 Boehringer Ingelheim Investigational Site

location_facility: 1199.37.49002 Boehringer Ingelheim Investigational Site

status_simplified: Completed


ReferenceType: result

ReferenceCitation: Atrafi F, Groen HJM, Byers LA, Garralda E, Lolkema MP, Sangha RS, Viteri S, Chae YK, Camidge DR, Gabrail NY, Hu B, Tian T, Nuthalapati S, Hoening E, He L, Komarnitsky P, Calles A. A Phase I Dose-Escalation Study of Veliparib Combined with Carboplatin and Etoposide in Patients with Extensive-Stage Small Cell Lung Cancer and Other Solid Tumors. Clin Cancer Res. 2019 Jan 15;25(2):496-505. doi: 10.1158/1078-0432.CCR-18-2014. Epub 2018 Oct 16.

family: Atrafi

full_name: Florence Atrafi

given: Florence

family: Groen

full_name: Harry J.M. Groen

given: Harry J.M.

family: Byers

full_name: Lauren A. Byers

given: Lauren A.

family: Garralda

full_name: Elena Garralda

given: Elena

family: Lolkema

full_name: Martijn P. Lolkema

given: Martijn P.

family: Sangha

full_name: Randeep S. Sangha

given: Randeep S.

family: Viteri

full_name: Santiago Viteri

given: Santiago

family: Chae

full_name: Young Kwang Chae

given: Young Kwang

family: Camidge

full_name: D. Ross Camidge

given: D. Ross

family: Gabrail

full_name: Nashat Y. Gabrail

given: Nashat Y.

family: Hu

full_name: Beibei Hu

given: Beibei

family: Tian

full_name: Tian Tian

given: Tian

family: Nuthalapati

full_name: Silpa Nuthalapati

given: Silpa

family: Hoening

full_name: Elizabeth Hoening

given: Elizabeth

family: He

full_name: Lei He

given: Lei

family: Komarnitsky

full_name: Philip Komarnitsky

given: Philip

family: Calles

full_name: Antonio Calles

given: Antonio

journal_name: Clinical Cancer Research

oa_host_type: publisher

publisher: American Association for Cancer Research (AACR)

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:
United States
Netherlands
France
Czechia
Romania
Hungary
Belgium
Australia
Russian Federation
Spain
Canada
Korea, Republic of

location_facility: Chonnam National University Hwasun Hospital /ID# 153188

location_facility: Belgorod Oncology Dispensary /ID# 152330

location_facility: University of Texas MD Anderson Cancer Center /ID# 129213

location_facility: Gabrail Cancer Center Research /ID# 129216

location_facility: S.C. Radiotherapy Center Cluj /ID# 165137

location_facility: Hopital du Sacre Coeur Montreal /ID# 154436

location_facility: Fejer Megyei Szent Gyorgy Korh /ID# 151352

location_facility: Sverdlovsk Regional Oncology Center Dispensary /ID# 152328

location_facility: Univercity Headache Clynic,LTD /ID# 161708

location_facility: Oncocenter Oncologie Clinica S /ID# 151694

location_facility: Hosp Univ Puerta de Hierro Maj /ID# 151253

location_facility: Centre Hosp Intercommunal de Creteil /ID# 157970

location_facility: Orszagos Koranyi Pulmonologiai Intezet /ID# 151351

location_facility: Hosp Univ Madrid Sanchinarro /ID# 130301

location_facility: Northwestern University Feinberg School of Medicine /ID# 137088

location_facility: Univ of Colorado Cancer Center /ID# 129220

location_facility: Southern Medical Day Care Ctr /ID# 155498

location_facility: Veszprem Megyei Tudogyogyintez /ID# 158807

location_facility: Hospital Stanta Creu i Sant Pau /ID# 151254

location_facility: Road Hospital Open Joint Stock Company Russian Railways /ID# 152731

location_facility: S.C. Centrul de Oncologie Sf. Nectarie S.R.L. /ID# 161137

location_facility: Herbert Herman Cancer Center /ID# 167020

location_facility: Debreceni Egyetem Klinikai Központ /ID# 151354

location_facility: Atrium-Orbis Zuyderland Medisch Centrum /ID# 149830

location_facility: Markusovszky Egyetemi Oktatókórház /ID# 158806

location_facility: Hospital Universitario Gregori /ID# 164982

location_facility: Petz Aladar Megyei Oktato Korh /ID# 155352

location_facility: Peninsula & South Eastern Haem /ID# 155497

location_facility: Universitair Medisch Centrum Groningen /ID# 131252

location_facility: Cliniques Universitaires Saint Luc /ID# 151024

ipd_sharing: Yes

source: euctr

title: A Phase 1 Dose Escalation and Phase 2 Randomized Double-Blind Study of Veliparib in Combination With Carboplatin and Etoposide as a Therapy of Treatment-Naïve Extensive Stage Disease Small Cell Lung Cancer

summary: The study seeks to assess the efficacy of veliparib (ABT-888) in combination with carboplatin and etoposide in participants with extensive disease small cell lung cancer (ED SCLC).


status: Active, not recruiting

lead_sponsor: Celgene

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

location_facility: Centre Hospitalier Universitaire de Liege

location_facility: Hospital Universitario Virgen De La Victoria

location_facility: H Lee Moffitt Cancer Center and Research Institute

location_facility: Medizinische Klinik III Klinikum der Universität München-Großhadern

location_facility: Cancer Care Centers of South Texas - HOAST

location_facility: University of Miami Miller School of Medicine

location_facility: King's College HospitalSchool of Medicine

location_facility: John Theurer Cancer Center at Hackensack University Medical Center

location_facility: Universitatsklinikum Carl Gustav Carus an der TU Dresden

location_facility: Institut Calatà d'Oncologia, L'Hospitalet

location_facility: Azienda Ospedaliera Universitaria Policlinico Sant Orsola Malpighi

location_facility: University of Iowa Hospitals and Clinics

location_facility: Zaklad Opieki Zdrowotnej MSW z Warminsko-Mazurskim Centrum Onkologii

location_facility: Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital

location_facility: Azienda Ospedaliera Santi Antonio Biagio E Cesare Arrigo

title: A Phase 2, International, Multicenter, Randomized, Open-label, Parallel Group to Evaluate the Efficacy and Safety of Cc-486 (Oral Azacitidine) Alone in Combination With Durvalumab (MEDI4736) in Subjects With Myelodysplastic Syndromes Who Fail to Achieve an Objective Response to Treatment With Azacitidine for Injection or Decitabine

summary:
Evaluate the safety and efficacy of oral azacitidne (CC-486) twice daily (BID) in subjects with myelodysplastic syndromes who failed to achieve an objective response post injectable hypomethylating agent (iHMA) treatment

Reason for removing the combination arm: Due to difficulties with dose-finding, the durvalumab plus CC-486 combination arm was closed to enrollment.

Extension:

An Extension Phase (EP) has been added to allow subjects who are currently receiving oral azacitidine BID and who are demonstrating clinical benefit as assessed by the Investigator, to continue receiving oral azacitidine until the subject meets the criteria for study discontinuation.


ReferenceCitation: Benjamin R, Graham C, Yallop D, Jozwik A, Mirci-Danicar OC, Lucchini G, Pinner D, Jain N, Kantarjian H, Boissel N, Maus MV, Frigault MJ, Baruchel A, Mohty M, Gianella-Borradori A, Binlich F, Balandraud S, Vitry F, Thomas E, Philippe A, Fouliard S, Dupouy S, Marchiq I, Almena-Carrasco M, Ferry N, Arnould S, Konto C, Veys P, Qasim W; UCART19 Group. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020 Dec 12;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5.

family: Benjamin

full_name: Reuben Benjamin

given: Reuben

family: Graham

full_name: Charlotte Graham

given: Charlotte

family: Yallop

full_name: Deborah Yallop

given: Deborah

family: Jozwik

full_name: Agnieszka Jozwik

given: Agnieszka

family: Mirci-Danicar

full_name: Oana C Mirci-Danicar

given: Oana C

family: Lucchini

full_name: Giovanna Lucchini

given: Giovanna

family: Pinner

full_name: Danielle Pinner

given: Danielle

family: Jain

full_name: Nitin Jain

given: Nitin

family: Kantarjian

full_name: Hagop Kantarjian

given: Hagop

family: Boissel

full_name: Nicolas Boissel

given: Nicolas

family: Maus

full_name: Marcela V Maus

given: Marcela V

family: Frigault

full_name: Matthew J Frigault

given: Matthew J

family: Baruchel

full_name: André Baruchel

given: André

family: Mohty

full_name: Mohamad Mohty

given: Mohamad

family: Gianella-Borradori

full_name: Athos Gianella-Borradori

given: Athos

family: Binlich

full_name: Florence Binlich

given: Florence

family: Balandraud

full_name: Svetlana Balandraud

given: Svetlana

family: Vitry

full_name: Fabien Vitry

given: Fabien

family: Thomas

full_name: Elisabeth Thomas

given: Elisabeth

family: Philippe

full_name: Anne Philippe

given: Anne

family: Fouliard

full_name: Sylvain Fouliard

given: Sylvain

family: Dupouy

full_name: Sandra Dupouy

given: Sandra

family: Marchiq

full_name: Ibtissam Marchiq

given: Ibtissam

family: Almena-Carrasco

full_name: Maria Almena-Carrasco

given: Maria

family: Ferry

full_name: Nicolas Ferry

ipd_sharing_description:
Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

sponsored by Servier
with a first patient enrolled as of 1 January 2004 onwards
for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

lead_sponsor: Institut de Recherches Internationales Servier

location_country:
United States
United Kingdom
France
Japan

location_facility: Hospital of the University of Pennsylvania

location_facility: King's College Hospital NHS Foundation Trust

location_facility: University of Texas MD Anderson Cancer Center

title: Phase I, Open Label, Dose-escalation Study Followed by a Safety Expansion Part to Evaluate the Safety, Expansion and Persistence of a Single Dose of UCART19 (Allogeneic Engineered T-cells Expressing Anti-CD19 Chimeric Antigen Receptor), Administered Intravenously in Patients With Relapsed or Refractory CD19 Positive B-cell Acute Lymphoblastic Leukaemia (B-ALL)

summary: The study is in two parts: a dose escalation then a safety dose expansion. The purpose of the dose escalation part is to evaluate the safety and tolerability of ascending doses of UCART19 (dose-escalation part) given as a single infusion in patients with relapsed / refractory (R/R) B-cell acute lymphoblastic leukaemia (B-ALL), to determine the maximum tolerated dose (MTD), the recommended dose and the lymphodepletion regimen. The purpose of the safety dose expansion is to assess the safety and tolerability of the RD for UCART19.


title: Prospective, Controlled, Double-Blind, Randomized Multicentric Study On The Efficacy And Safety Of An Early Target Controlled Plasma Volume Replacement Therapy With A Balanced Gelatine Solution vs A Balanced Electrolyte Solution In Patients With Severe Sepsis

acronym: GENIUS

summary: This prospective, double-blind randomized controlled trial evaluates the differences in terms of efficacy and safety of gelatin based resuscitation as compared to crystalloid based resuscitation in two parallel groups of patients with severe sepsis / septic shock.

ReferenceCitation: Marx G, Zacharowski K, Ichai C, Asehnoune K, Černý V, Dembinski R, Ferrer Roca R, Fries D, Molnar Z, Rosenberger P, Sanchez-Sanchez M, Schürholz T, Dehnhardt T, Schmier S, von Kleist E, Brauer U, Simon TP. Efficacy and safety of early target-controlled plasma volume replacement with a balanced gelatine solution versus a balanced electrolyte solution in patients with severe sepsis/septic shock: study protocol, design, and rationale of a prospective, randomized, controlled, double-blind, multicentric, international clinical trial : GENIUS-Gelatine use in ICU and sepsis. Trials. 2021 Jun 2;22(1):376. doi: 10.1186/s13063-021-05311-8.

family: Marx

full_name: Gernot Marx

given: Gernot

family: Zacharowski

full_name: Kai Zacharowski

given: Kai

family: Ichai

full_name: Carole Ichai

given: Carole

family: Asehnoune

full_name: Karim Asehnoune

given: Karim

family: Černý

full_name: Vladimír Černý

given: Vladimír

family: Dembinski

full_name: Rolf Dembinski

given: Rolf

family: Ferrer Roca

full_name: Ricard Ferrer Roca

given: Ricard

family: Fries

full_name: Dietmar Fries

given: Dietmar

family: Molnar

full_name: Zsolt Molnar

given: Zsolt

family: Rosenberger

full_name: Peter Rosenberger

given: Peter

family: Sanchez-Sanchez

full_name: Manuel Sanchez-Sanchez

given: Manuel

family: Schürholz

full_name: Tobias Schürholz

given: Tobias

family: Dehnhardt

full_name: Tamara Dehnhardt

given: Tamara

family: Schmier

full_name: Sonja Schmier

given: Sonja

family: von Kleist

full_name: Elke von Kleist

given: Elke

family: Brauer

full_name: Ute Brauer

given: Ute

family: Simon

full_name: Tim-Philipp Simon

given: Tim-Philipp

currency_apc_doaj: GBP

journal_name: Trials

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

lead_sponsor: B. Braun Melsungen AG

location_country: Austria France Czechia Hungary Spain Germany

location_facility: Masarykova nemocnice v Ústí nad Labem, o.z.

location_facility: Szegedi Tudományegyetem - Általános Orvostudományi Kar

location_facility: Universitätsinstitut - Johannes Wesling Klinikum MInden


title: A Phase 2, Multicenter, International, Single Arm Study To Assess The Safety And Efficacy Of Single Agent Cc-486 (Oral Azacitidine) In Previously Treated Subjects With Locally Advanced Or Metastatic Nasopharyngeal Carcinoma

summary: The purpose of this study is to evaluate the safety and efficacy of CC-486 in previously treated patients with locally advanced or metastatic nasopharyngeal carcinoma having failed one to two previous regimens, including platinum-based chemotherapy. Participants will be enrolled according to a Simon two-stage design; if the predefined activity is met (>4 responses [complete response; partial response {CR/PR}] out of the first 17 evaluable participants based on independent radiological assessment), then the study will continue to enroll an additional 34 participants. If 4 or less responses out of 17 are observed, then the study enrollment will be stopped.

ReferenceCitation: Von Hoff DD, Rasco DW, Heath EI, Munster PN, Schellens JHM, Isambert N, Le Tourneau C, O'Neil B, Mathijssen RHJ, Lopez-Martin JA, Edenfield WJ, Martin M, LoRusso PM, Bray GL, DiMartino J, Nguyen A, Liu K, Laille E, Bendell JC. Phase I Study of CC-486 Alone and in Combination with Carboplatin or nab-Paclitaxel in Patients with Relapsed or Refractory Solid Tumors. Clin Cancer Res. 2018 Sep 1;24(17):4072-4080. doi: 10.1158/1078-0432.CCR-17-3716. Epub 2018 May 15.

family: Von Hoff

full_name: Daniel D. Von Hoff

given: Daniel D.

family: Rasco

full_name: Drew W. Rasco

given: Drew W.

family: Heath

full_name: Elisabeth I. Heath

given: Elisabeth I.

family: Munster

full_name: Pamela N. Munster

given: Pamela N.

family: Schellens

full_name: Jan H.M. Schellens

given: Jan H.M.

family: Isambert

full_name: Nicolas Isambert

given: Nicolas

family: Tourneau

full_name: Christophe Le Tourneau

given: Christophe Le

family: O'Neil

full_name: Bert O'Neil

given: Bert

family: Mathijssen

full_name: Ron H.J. Mathijssen

given: Ron H.J.

family: Lopez-Martin

full_name: Jose A. Lopez-Martin

given: Jose A.

family: Edenfield

full_name: W. Jeff Edenfield

given: W. Jeff

family: Martin

full_name: Miguel Martin

given: Miguel

family: LoRusso

full_name: Patricia M. LoRusso

given: Patricia M.

family: Bray

full_name: Gordon L. Bray

given: Gordon L.

family: DiMartino

full_name: Jorge DiMartino

given: Jorge

family: Nguyen

full_name: Aaron Nguyen

given: Aaron

family: Liu

full_name: Kejian Liu

given: Kejian

family: Laille

full_name: Eric Laille

given: Eric

family: Bendell

full_name: Johanna C. Bendell

given: Johanna C.

publisher_dissemination: American Association for Cancer Research

location_country:
Italy
France
Greece
Tunisia
Singapore
Spain
United States
Canada
Taiwan

location_facility: Hopital Abderrahman Mami de Pneumo-Phtisiologie de l'Ariana

location_facility: Hospital Universitario Madrid Sanchinarro

location_facility: Winship Cancer Institute of Emory University

location_facility: Chang Gung Medical Foundation, Kaohsiung Memorial Hospital

location_facility: University General Hospital of Heraklion

location_facility: Johns Hopkins Singapore International Medical Centre

location_facility: Columbia Comprehensive Cancer Care Clinic

location_facility: Instituto Catalan de Oncologia-Hospital Duran


title: A Modular Phase I, Open-Label, Multicentre Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Anti-tumour Activity of Ceralasertib in Combination With Cytotoxic Chemotherapy and/or DNA Damage Repair/Novel Anti-cancer Agents in Patients With Advanced Solid Malignancies.

summary: This is a modular, phase I/ phase 1 b, open-label, multicentre study of ceralasertib administered orally in combination with cytotoxic chemotherapy regimens and/or novel anti-cancer agents, to patients with advanced malignancies. The study design allows an investigation of optimal combination dose of ceralasertib with other anti-cancer treatments, with intensive safety monitoring to ensure the safety of the patients. The initial combination to be investigated is ceralasertib with carboplatin. The second combination to be investigated is ceralasertib with Olaparib. The third combination to be investigated is ceralasertib with durvalumab

ipd_sharing_description: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

lead_sponsor: AstraZeneca

location_country:
United States
United Kingdom
Korea, Republic of
France


title: DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis

summary: The purpose of this study is to characterize the frequency of TTR mutations in subjects suspected of having cardiac amyloidosis

ipd_sharing: Undecided

lead_sponsor: Alnylam Pharmaceuticals

location_country:
Brazil
France
Belgium
Spain
United States
United Kingdom


title: A Randomised Controlled Trial to Compare Seizure Remission Outcome Following Resective Surgery With or Without Prior Treatment With Ketogenic Diet in Children With Epilepsy the Result of Focal Cortical Dysplasia Type II

acronym: EDIBLE

summary: The investigators are undertaking the first European Randomised Controlled Trial (RCT) for epilepsy surgery in children with FCD type II, to prospectively evaluate the role of the KD prior to surgery in improving seizure outcome. The investigators will evaluate the role of KD as a disease-modifying treatment to achieve seizure control and improve neurodevelopment and quality of life. Children age 3 - 15 years with pharmacoresistant epilepsy believed to be the result of focal cortical dysplasia type II, considered to be surgically treatable, will be randomised to either receive 6m treatment presurgery with a ketogenic diet, or to proceed direct to surgery (no pretreatment). Primary outcome will be the time to achieve a period of 6 months of seizure freedom from the date of randomisation. Tissue resected at surgery will also be evaluated with regard to the degree of any methylation of DNA.

status: Terminated

ReferenceType: background

ReferenceCitation: Wiebe S, Blume WT, Girvin JP, Eliasziw M; Effectiveness and Efficiency of Surgery for Temporal Lobe Epilepsy Study Group. A randomized, controlled trial of surgery for temporal-lobe epilepsy. N Engl J Med. 2001 Aug 2;345(5):311-8.

ReferenceCitation: Engel J Jr, McDermott MP, Wiebe S, Langfitt JT, Stern JM, Dewar S, Sperling MR, Gardiner I, Erba G, Fried I, Jacobs M, Vinters HV, Mintzer S, Kieburtz K; Early Randomized Surgical Epilepsy Trial (ERSET) Study Group. Early surgical therapy for drug-resistant temporal lobe epilepsy: a randomized trial. JAMA. 2012 Mar 7;307(9):922-30. doi: 10.1001/jama.2012.220.

ReferenceCitation: Harvey AS, Cross JH, Shinnar S, Mathern GW; ILAE Pediatric Epilepsy Surgery Survey Taskforce. Defining the spectrum of international practice in pediatric epilepsy surgery patients. Epilepsia. 2008 Jan;49(1):146-55. Epub 2007 Nov 27. Erratum in: Epilepsia. 2013 Jun;54(6):1140. Mathern, Bary W [corrected to Mathern, Gary W].

ReferenceCitation: Lerner JT, Salamon N, Hauptman JS, Velasco TR, Hemb M, Wu JY, Sankar R, Donald Shields W, Engel J Jr, Fried I, Cepeda C, Andre VM, Levine MS, Miyata H, Yong WH, Vinters HV, Mathern GW. Assessment and surgical outcomes for mild type I and severe type II cortical dysplasia: a critical review and the UCLA experience. Epilepsia. 2009 Jun;50(6):1310-35. doi: 10.1111/j.1528-1167.2008.01998.x. Epub 2009 Jan 21. Review.

ReferenceCitation: Blümcke I, Thom M, Aronica E, Armstrong DD, Vinters HV, Palmini A, Jacques TS, Avanzini G, Barkovich AJ, Battaglia G, Becker A, Cepeda C, Cendes F, Colombo N, Crino P, Cross JH, Delalande O, Dubeau F, Duncan J, Guerrini R, Kahane P, Mathern G, Najm I, Ozkara C, Raybaud C, Represa A, Roper SN, Salamon N, Schulze-Bonhage A, Tassi L, Vezzani A, Spreafico R. The clinicopathologic spectrum of focal cortical dysplasias: a consensus classification proposed by an ad hoc Task Force of the ILAE Diagnostic Methods Commission. Epilepsia. 2011 Jan;52(1):158-74. doi: 10.1111/j.1528-1167.2010.02777.x. Epub 2010 Nov 10.

ReferenceCitation: Levy RG, Cooper PN, Giri P. Ketogenic diet and other dietary treatments for epilepsy. Cochrane Database Syst Rev. 2012 Mar 14;(3):CD001903. doi: 10.1002/14651858.CD001903.pub2. Review. Update in: Cochrane Database Syst Rev. 2016;2:CD001903.

ReferenceCitation: Neal EG, Chaffe H, Schwartz RH, Lawson MS, Edwards N, Fitzsimmons G, Whitney A, Cross JH. The ketogenic diet for the treatment of childhood epilepsy: a randomised controlled trial. Lancet Neurol. 2008 Jun;7(6):500-6. doi: 10.1016/S1474-4422(08)70092-9. Epub 2008 May 2.

ReferenceCitation: Neal EG, Chaffe H, Schwartz RH, Lawson MS, Edwards N, Fitzsimmons G, Whitney A, Cross JH. A randomized trial of classical and medium-chain triglyceride ketogenic diets in the treatment of childhood epilepsy. Epilepsia. 2009 May;50(5):1109-17. doi: 10.1111/j.1528-1167.2008.01870.x. Epub 2008 Nov 19.

ReferenceCitation: Sharma S, Sankhyan N, Gulati S, Agarwala A. Use of the modified Atkins diet for treatment of refractory childhood epilepsy: a randomized controlled trial. Epilepsia. 2013 Mar;54(3):481-6. doi: 10.1111/epi.12069. Epub 2013 Jan 7.

ReferenceCitation: Jung DE, Kang HC, Kim HD. Long-term outcome of the ketogenic diet for intractable childhood epilepsy with focal malformation of cortical development. Pediatrics. 2008 Aug;122(2):e330-3. doi: 10.1542/peds.2008-0012.

ReferenceCitation: Hahnen E, Hauke J, Tränkle C, Eyüpoglu IY, Wirth B, Blümcke I. Histone deacetylase inhibitors: possible implications for neurodegenerative disorders. Expert Opin Investig Drugs. 2008 Feb;17(2):169-84. doi: 10.1517/13543784.17.2.169. Review.

ReferenceCitation: Göttlicher M, Minucci S, Zhu P, Krämer OH, Schimpf A, Giavara S, Sleeman JP, Lo Coco F, Nervi C, Pelicci PG, Heinzel T. Valproic acid defines a novel class of HDAC inhibitors inducing differentiation of transformed cells. EMBO J. 2001 Dec 17;20(24):6969-78.

ReferenceCitation: Feil R, Fraga MF. Epigenetics and the environment: emerging patterns and implications. Nat Rev Genet. 2012 Jan 4;13(2):97-109. doi: 10.1038/nrg3142. Review.

ReferenceCitation: Feng J, Zhou Y, Campbell SL, Le T, Li E, Sweatt JD, Silva AJ, Fan G. Dnmt1 and Dnmt3a maintain DNA methylation and regulate synaptic function in adult forebrain neurons. Nat Neurosci. 2010 Apr;13(4):423-30. doi: 10.1038/nn.2514. Epub 2010 Mar 14.

ReferenceCitation: Kobow K, Kaspi A, Harikrishnan KN, Kiese K, Ziemann M, Khurana I, Fritzsche I, Hauke J, Hahnen E, Coras R, Mühlebner A, El-Osta A, Blümcke I. Deep sequencing reveals increased DNA methylation in chronic rat epilepsy. Acta Neuropathol. 2013 Nov;126(5):741-56. doi: 10.1007/s00401-013-1168-8. Epub 2013 Sep 5.

lead_sponsor: University College, London

location_country:
Italy
Austria
Switzerland
Germany
France
Czechia
United States
United Kingdom

location_facility: Great Ormond Street Hospital for Children NHS Trust

intervention_type: Dietary Supplement

lead_sponsor_type: academique

status_simplified: Unknown


title: Clinical Study of the BreathID® LF System to Train the Algorithm for the ¹³C-Methacetin Breath Test (MBT) in Assessment of Portal Hypertension in Patients With Compensated Liver Cirrhosis

summary: This study will be used to train an algorithm using Methacetin breath test (MBT) measures and to select a cut-off to determine presence or absence of Clinically Significant Portal Hypertension (CSPH) as defined by Hepatic Venous Gradient Pressure (HVPG) ≥ 10mmHg,

primary_purpose: Diagnostic

lead_sponsor: Exalenz Bioscience LTD.

location_country:
United States
France
Switzerland
Spain

location_facility: (University of Barcelona) Hospital Clinic

intervention_type: Device


title: A Phase 2 Multi-center, Randomized, Double-blind, Comparator-Controlled Dose Finding Study to Evaluate MP4CO for the Acute Treatment of Vaso-occlusive Crises in Subjects With Sickle Cell Disease

summary:
Sickle Cell disease is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body. In Sickle Cell disease, as red blood cells circulate and oxygen is released, the deoxygenated abnormal Hemoglobin S can begin to polymerize and cause red cells to become sticky and elongated. These "sickled" red cells are less flexible and will obstruct small blood vessels and prevent normal red cells from circulating freely, which limits oxygen delivery to tissues and organs. This is known as a "sickling crisis" or "vaso-occlusive crisis" and is the leading cause of hospitalization in patients with Sickle Cell disease.

Patients suffering from a sickle crisis experience severe pain and are at risk of stroke, heart attack or even death. Current therapy is limited to hydration and symptomatic pain relief. The administration of MP4CO as an adjunct treatment to standard therapy may alleviate pain associated with a sickling crisis and potentially reduce the severity and duration of a crisis. This may shorten the time in hospital and potentially improve the quality of life for patients with sickle cell anemia.

status: Withdrawn

ReferenceCitation: Vandegriff KD, Young MA, Lohman J, Bellelli A, Samaja M, Malavalli A, Winslow RM. CO-MP4, a polyethylene glycol-conjugated haemoglobin derivative and carbon monoxide carrier that reduces myocardial infarct size in rats. Br J Pharmacol. 2008 Aug;154(8):1649-61. doi: 10.1038/bjp.2008.219. Epub 2008 Jun 9.

ReferenceCitation: Vandegriff KD, Bellelli A, Samaja M, Malavalli A, Brunori M, Winslow RM. Kinetics of NO and O2 binding to a maleimide poly(ethylene glycol)-conjugated human haemoglobin. Biochem J. 2004 Aug 15;382(Pt 1):183-9.

ReferenceCitation: Tsai AG, Vandegriff KD, Intaglietta M, Winslow RM. Targeted O2 delivery by low-P50 hemoglobin: a new basis for O2 therapeutics. Am J Physiol Heart Circ Physiol. 2003 Oct;285(4):H1411-9. Epub 2003 Jun 12.

ReferenceCitation: Cole RH, Vandegriff KD. MP4, a vasodilatory PEGylated hemoglobin. Adv Exp Med Biol. 2011;701:85-90. doi: 10.1007/978-1-4419-7756-4_12.

ReferenceCitation: Vandegriff KD, Malavalli A, Mkrtchyan GM, Spann SN, Baker DA, Winslow RM. Sites of modification of hemospan, a poly(ethylene glycol)-modified human hemoglobin for use as an oxygen therapeutic. Bioconjug Chem. 2008 Nov 19;19(11):2163-70. doi: 10.1021/bc8002666.

ReferenceCitation: Svergun DI, Ekström F, Vandegriff KD, Malavalli A, Baker DA, Nilsson C, Winslow RM. Solution structure of poly(ethylene) glycol-conjugated hemoglobin revealed by small-angle X-ray scattering: implications for a new oxygen therapeutic. Biophys J. 2008 Jan 1;94(1):173-81. Epub 2007 Sep 7.

ReferenceCitation: Tsai AG, Cabrales P, Manjula BN, Acharya SA, Winslow RM, Intaglietta M. Dissociation of local nitric oxide concentration and vasoconstriction in the presence of cell-free hemoglobin oxygen carriers. Blood. 2006 Nov 15;108(10):3603-10. Epub 2006 Jul 20.

ReferenceCitation: Vandegriff KD, McCarthy M, Rohlfs RJ, Winslow RM. Colloid osmotic properties of modified hemoglobins: chemically cross-linked versus polyethylene glycol surface-conjugated. Biophys Chem. 1997 Nov;69(1):23-30.

ReferenceCitation: Ballas SK, Gupta K, Adams-Graves P. Sickle cell pain: a critical reappraisal. Blood. 2012 Nov 1;120(18):3647-56. doi: 10.1182/blood-2012-04-383430. Epub 2012 Aug 24. Review.

ReferenceCitation: Belcher JD, Young M, Chen C, Nguyen J, Burhop K, Tran P, Vercellotti GM. MP4CO, a pegylated hemoglobin saturated with carbon monoxide, is a modulator of HO-1, inflammation, and vaso-occlusion in transgenic sickle mice. Blood. 2013 Oct 10;122(15):2757-64. doi: 10.1182/blood-2013-02-486282. Epub 2013 Aug 1.

ReferenceCitation: Belcher JD, Mahaseth H, Welch TE, Otterbein LE, Hebbel RP, Vercellotti GM. Heme oxygenase-1 is a modulator of inflammation and vaso-occlusion in transgenic sickle mice. J Clin Invest. 2006 Mar;116(3):808-16. Epub 2006 Feb 16.

ReferenceCitation: Bilban M, Haschemi A, Wegiel B, Chin BY, Wagner O, Otterbein LE. Heme oxygenase and carbon monoxide initiate homeostatic signaling. J Mol Med (Berl). 2008 Mar;86(3):267-79. Epub 2007 Nov 22. Review.

ReferenceCitation: Piantadosi CA, Withers CM, Bartz RR, MacGarvey NC, Fu P, Sweeney TE, Welty-Wolf KE, Suliman HB. Heme oxygenase-1 couples activation of mitochondrial biogenesis to anti-inflammatory cytokine expression. J Biol Chem. 2011 May 6;286(18):16374-85. doi: 10.1074/jbc.M110.207738. Epub 2011 Mar 18.

ReferenceCitation: Powars DR, Chan LS, Hiti A, Ramicone E, Johnson C. Outcome of sickle cell anemia: a 4-decade observational study of 1056 patients. Medicine (Baltimore). 2005 Nov;84(6):363-376. doi: 10.1097/01.md.0000189089.45003.52.

lead_sponsor: Sangart

location_country:
Brazil
Bahrain
Lebanon
Netherlands
France
Belgium
Turkey
United Kingdom
Qatar

location_facility: Rio de Janerio Instituto Estadual de Hematologie

location_facility: Georges Pompidou European University Hospital


title: PHASE ANGLE PROJECT

acronym: PAP

summary:
Critically ill patients feature a loss of fat-free mass (FFM) up to 440 g/day, which is associated with increased morbidity and prolonged recovery. In several clinical conditions, FFM or phase angle (PhA)derived from BIA have been associated with clinical outcome. However, solid data to support this association in ICU patients are lacking. Only one retrospective study of 51 ICU patients with acute respiratory failure correlated loss of active cell mass with mortality. In a pilot study performed in 55 ICU patients, the investigators observed that five kHz BIA PhA was significantly related to SOFA (r=0.38, P=0.03). The relation between PhA and mortality remains to be determined in ICU patients.

Classic ICU validated severity scores (e.g. Acute Physiology and Chronic Health Evaluation (APACHE), Sequential Organ Failure Assessment (SOFA), Simplified Acute Physiology Score (SAPS)) and recent nutritional scores have been developed to foresee the clinical outcome of ICU patients. Most of these scores are time consuming and suffer some degree of discriminative power (i.e. APACHE II and SAPS II are not validated in cardiovascular surgery patients).

PhA is reflecting intracellular status: altered intracellular water (ICW) to extracellular water (ECW) distribution is suggested by low PhA. PhA measurement does not require anamnestic parameters, body weight, and lab tests. It could easily, accurately and repeatedly measured at bedside. PhA has been correlated with the disease prognosis in HIV infection, hemodialysis, peritoneal dialysis, chronic renal failure and liver cirrhosis patients. These studies suggest that PhA may be useful in determining increased risk of morbidity in the ICU.

Computerized tomography (CT) images targeted on the 3rd lumbar vertebrae (L3) could accurately measure FFM13 and predict survival in cancer patients. Body composition evaluation by CT presents great practical significance due to its routine ICU use in the initial diagnosis or follow-up. The usefulness of measuring FFM with L3-targeted CT has never been evaluated in ICU patients.

Therefore, the investigators aim to conduct an international multicentre prospective observational study in ICU patients to assess the prognosis value of BIA PhA at admission, and to compare the performances of BIA and L3-targeted CT for FFM measurement.

ReferenceCitation: Thibault R, Makhlouf AM, Mulliez A, Cristina Gonzalez M, Kekstas G, Kozjek NR, Preiser JC, Rozalen IC, Dadet S, Krznaric Z, Kupczyk K, Tamion F, Cano N, Pichard C; Phase Angle Project Investigators. Fat-free mass at admission predicts 28-day mortality in intensive care unit patients: the international prospective observational study Phase Angle Project. Intensive Care Med. 2016 Sep;42(9):1445-53. doi: 10.1007/s00134-016-4468-3. Epub 2016 Aug 11.

family: Thibault

full_name: Ronan Thibault

given: Ronan

family: Makhlouf

full_name: Anne-Marie Makhlouf

given: Anne-Marie

family: Mulliez

full_name: Aurélien Mulliez

given: Aurélien

family: Cristina Gonzalez

full_name: M. Cristina Gonzalez

given: M.

family: Kekstas

full_name: Gintautas Kekstas

given: Gintautas

family: Kozjek

full_name: Nada Rotovnik Kozjek

given: Nada Rotovnik

family: Preiser

full_name: Jean-Charles Preiser

given: Jean-Charles

family: Rozalen

full_name: Isabel Ceniceros Rozalen

given: Isabel Ceniceros

family: Dadet

full_name: Sylvain Dadet

given: Sylvain

family: Krznaric

full_name: Zeljko Krznaric

given: Zeljko

family: Kupczyk

full_name: Kinga Kupczyk

given: Kinga

family: Tamion

full_name: Fabienne Tamion

given: Fabienne

family: Cano

full_name: Noël Cano

given: Noël

family: Pichard

full_name: Claude Pichard

given: Claude

family: Graf

full_name: Séverine Graf

given: Séverine

family: Heidegger

full_name: Claudia P. Heidegger

given: Claudia P.

family: Guerreiro

full_name: Marcio Guerreiro

given: Marcio

family: Orlandi

full_name: Silvana Orlandi

given: Silvana

family: Klimasauskas

full_name: Andrius Klimasauskas

given: Andrius

family: Sostakaite

full_name: Gintare Sostakaite

given: Gintare

family: Gorjup

full_name: Kristina Gorjup

given: Kristina

family: Kogovšek

full_name: Katja Kogovšek

given: Katja

family: Pirtovšek

full_name: Katja Kopriva Pirtovšek

given: Katja Kopriva

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

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

location_country: Brazil Switzerland Croatia France Lithuania Israel Poland Belgium Spain

location_facility:
Rabin Medical Center
Clermont-Ferrand University Hospital
Univ. Hospital Center Zagreb
Rouen University Hospital
Stanley Dudrick's Memorial Hospital
Erasme University Hospital
Geneva University Hospital
Clinica USP Palmaplanas
Universidade Federal de Pelotas
Vilnius University Hospital


title: The "DeSScipher" Project - to Decipher the Best Treatment for Systemic Sclerosis - Observational Trial 2: Improvement of Hand Dysfunction by Arthritis in Systemic Sclerosis

summary:
Systemic sclerosis (SSc) is an orphan, multiorgan disease affecting the connective tissue of the skin and several internal organs. Beside skin involvement, digital ulcers, tendinitis, calcinosis and flexion contractures, the presence of hand arthritis is a major contributor to impairment of hand function in systemic sclerosis. Several immunomodulatory drugs used in other rheumatic diseases (including methotrexate, leflunomide, azathioprine, mycophenolate mofetil and low-dose corticosteroids) can potentially improve arthritis and consequently hand function in systemic sclerosis. For the assessment of arthritis, the CDAI (clinical disease activity index) is validated in rheumatoid arthritis, and may be useful for SSc-related arthritis, too.

This observational trial is part of the collaborative project "DeSScipher", one out of five observational trials to decipher the optimal management of systemic sclerosis. Aim of this observational trial is to:

investigate the efficacy and safety of different treatments on hand dysfunction in systemic sclerosis patients with hand arthritis and
to validate the CDAI for arthritis in systemic sclerosis.

status: Unknown status

lead_sponsor: Prof. Laszlo Czirjak

location_country:
Italy
Serbia
Switzerland
Croatia
France
Romania
Hungary
Belgium
Russian Federation
Egypt
Spain
Turkey
Germany
United Kingdom

location_facility: GR.T.Popa Center for Biomedical Research, European Center for Translational Research, "GR.T.Popa" University of Medicine and Pharmacy, Rehabilitation Hospital

location_facility: Department of Internal Medicine Hôpital Claude Huriez

location_facility: Institute of Rheumatology, Russian Academy of Medical Science

location_facility: University of Marmara, Department of Rheumatology

location_facility: Justus-Liebig-University Gießen, Kerckhoff Clinic, Departement of Rheumatology and Clinical Immunology

location_facility: Charité Universitätsmedizin Berlin, Charité Centrum 12 für Innere Medizin und Dermatologie, Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie

location_facility: Reumatologie, University of Medicine & Pharmacy,"Iuliu Hatieganu" Cluj

location_facility: Royal Free Hospital, University College London

location_facility: Université Paris Descartes, Hôpital Cochin, Service de Rhumatologie A & INSERM 1016

location_facility: Hospital Universitario Madrid Norte Sanchinarro

location_facility: University of Florence, Denothe Centre, Division of Rheumatology AOUC, Department of Biomedicine

location_facility: Clinic of Nephrology, Internal and Occupational Diseases

location_facility: University of Ghent, Department of Rheumatology

location_facility: Pecsi Tudomanyegyetem - University of Pecs

location_facility: Divisione di Reumatologia, Università di Roma La Sapienza, Dipartimento di Clinica e Terapia medica applicata, Policlinico Umberto I

location_facility: University of Manchester, Rheumatic Diseases Centre, Clinical Sciences

location_facility: Assiut and Sohage University Hospital Rheumatology Department Assiut University Hospital

location_facility: Department of Internal Medicine and Rheumatology Clinic, Ion Cantacuzino Clinical Hospital

location_facility: Istituto di Clinica Medica Generale, Ematologia ed Immunologia Clinica, Università Politecnica delle Marche, Polo Didattico, University of Ancona

location_facility: The Universitiy of Leeds, Division of Rheumatic and Musculoskeletal Disease, St James's University Hospital

location_facility: Centre for Pediatric Rheumatology, Klinikum Eilbek

location_facility: Department of Rheumatology, University of Cagliari-Policlinico Universitario

location_facility: University of Zurich, Department of Rheumatology


title: A Phase 2A Study of ALXN1007 in Subjects With Newly Diagnosed Acute Graft-Versus-Host Disease Involving the Lower Gastrointestinal Tract

acronym: GIGVHD

summary: The objectives of this trial were to evaluate the safety, tolerability, pharmacokinetics/pharmacodynamics (PK/PD) and efficacy of intravenous (IV) ALXN1007 in participants with acute graft-versus-host disease (GVHD) of the lower gastrointestinal (GI) tract.

lead_sponsor: Alexion Pharmaceuticals

location_country:
United States
France

location_facility: University of Minnesota Medicine - Hematology, Oncology and Transplantation Office

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Abramson Cancer Perelman Center for Advanced Medicine, University of Pennsylvania

location_facility: Groupe Hospitalier Sud - Hôpital Haut-Lévêque - Centre François

intervention_type: Biological


ReferenceCitation: Connor P, Sánchez van Kammen M, Lensing AWA, Chalmers E, Kállay K, Hege K, Simioni P, Biss T, Bajolle F, Bonnet D, Grunt S, Kumar R, Lvova O, Bhat R, Van Damme A, Palumbo J, Santamaria A, Saracco P, Payne J, Baird S, Godder K, Labarque V, Male C, Martinelli I, Morales Soto M, Motwani J, Shah S, Hooimeijer HL, Prins MH, Kubitza D, Smith WT, Berkowitz SD, Pap AF, Majumder M, Monagle P, Coutinho JM. Safety and efficacy of rivaroxaban in pediatric cerebral venous thrombosis (EINSTEIN-Jr CVT). Blood Adv. 2020 Dec 22;4(24):6250-6258. doi: 10.1182/bloodadvances.2020003244.

name: The Noah's Ark Children’s Hospital for Wales, Cardiff, United Kingdom;

family: Connor

full_name: Philip Connor

given: Philip

name: Department of Neurology, Amsterdam University Medical Center (UMC), University of Amsterdam, Amsterdam, The Netherlands;

family: Sánchez van Kammen

full_name: Mayte Sánchez van Kammen

given: Mayte

family: Lensing

full_name: Anthonie W. A. Lensing

given: Anthonie W. A.

name: Royal Hospital for Children, Glasgow, United Kingdom;

family: Chalmers

full_name: Elizabeth Chalmers

given: Elizabeth

name: Department of Pediatric Hematology and Stem Cell Transplantation, Central Hospital of Southern Pest, National Institute of Hematology and Infectious Diseases, Budapest, Hungary;

family: Kállay

full_name: Krisztián Kállay

given: Krisztián

name: Riley Hospital For Children at Indiana University (IU) Health, Indianapolis, IN;

family: Hege

full_name: Kerry Hege

given: Kerry

name: Thrombotic and Hemorrhagic Diseases Unit, Department of Medicine (DIMED), Padua University Hospital, Padua, Italy;

family: Simioni

full_name: Paolo Simioni

given: Paolo

name: Department of Haematology, The Newcastle upon Tyne Hospitals, National Health Service (NHS) Foundation Trust, Newcastle upon Tyne, United Kingdom;

family: Biss

full_name: Tina Biss

given: Tina

name: M3C-Necker Enfants Malades, Université Paris Descartes, Sorbonne Paris Cité, Paris, France;

family: Bajolle

full_name: Fanny Bajolle

given: Fanny

family: Bonnet

full_name: Damien Bonnet

given: Damien

name: Division of Neuropediatrics, Development, and Rehabilitation, University Children’s Hospital, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland;

family: Grunt

full_name: Sebastian Grunt

given: Sebastian

name: Nationwide Children’s Hospital, The Ohio State University, Columbus, OH;

family: Kumar

full_name: Riten Kumar

given: Riten

name: Ural Federal University, Ural State Medical University, Yekaterinburg, Russia;

family: Lvova

full_name: Olga Lvova

given: Olga

name: Ann and Robert H. Lurie Children’s Hospital of Chicago, Feinberg School of Medicine, Northwestern University, Chicago, IL;

family: Bhat

full_name: Rukhmi Bhat

given: Rukhmi

name: Department of Pediatric Hematology/Oncology, Saint Luc University Hospital, Brussels, Belgium;

family: Van Damme

full_name: An Van Damme

given: An

name: Cancer and Blood Diseases Institute, Cincinnati Children’s Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH;

family: Palumbo

full_name: Joseph Palumbo

given: Joseph

name: Hemostasis and Thrombosis Unit, Department of Hematology, University Hospital Vall d´Hebron, Barcelona, Spain;

family: Santamaria

full_name: Amparo Santamaria

given: Amparo

name: Pediatric Hematology, University Hospital Città della Salute e della Scienza Torino, Turin, Italy;

family: Saracco

full_name: Paola Saracco

given: Paola

name: Department of Paediatric Haematology, Sheffield Children’s Hospital, Sheffield, United Kingdom;

family: Payne

full_name: Jeanette Payne

given: Jeanette

name: Royal Hospital for Sick Children, Edinburgh, United Kingdom;

family: Baird

full_name: Susan Baird

given: Susan

name: Nicklaus Children’s Hospital, Miami, FL;

family: Godder

full_name: Kamar Godder

given: Kamar

name: Department of Pediatric Hematology and Oncology, University Hospital Leuven Gasthuisberg, Leuven, Belgium;

family: Labarque

full_name: Veerle Labarque

given: Veerle

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

family: Male

full_name: Christoph Male

given: Christoph

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

family: Martinelli

full_name: Ida Martinelli

given: Ida

name: Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde, Guadalajara, Mexico;

family: Morales Soto

full_name: Michelle Morales Soto

given: Michelle

name: Birmingham Children’s Hospital, Birmingham, United Kingdom;

name: University of Arizona School of Medicine and Phoenix Children’s Hospital, Phoenix, AZ;

name: Department of Hematology and Oncology, University Medical Center Groningen, Beatrix Children’s Hospital, Groningen, The Netherlands;

name: Department of Clinical Epidemiology and Medical Technology Assessment, Maastricht University Medical Center, Maastricht, The Netherlands;

name: Haematology Research Murdoch's Children's Research Institute, and

name: Department of Paediatrics, University of Melbourne, Melbourne, Australia

repository_institution: Ural Federal University - Electronic scientific archive of UrFU

ReferenceCitation: Thom K, Lensing AWA, Nurmeev I, Bajolle F, Bonnet D, Kenet G, Massicotte MP, Karakas Z, Palumbo JS, Saracco P, Amedro P, Chain J, Chan AK, Ikeyama T, Lam JCM, Gauger C, Pap ÁF, Majumder M, Kubitza D, Smith WT, Berkowitz SD, Prins MH, Monagle P, Young G, Male C. Safety and efficacy of anticoagulant therapy in pediatric catheter-related venous thrombosis (EINSTEIN-Jr CVC-VTE). Blood Adv. 2020 Oct 13;4(19):4632-4639. doi: 10.1182/bloodadvances.2020002637.

name: Kazan State Medical University, Kazan, Russia;

name: Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel;

name: Israeli National Hemophilia Center and Thrombosis Unit and The Amalia Biron Thrombosis Research Institute, Sheba Medical Center, Tel Hashomer, Israel;

name: Department of Pediatrics, University of Alberta, Edmonton, AB, Canada;

name: Pediatric Hematology/Oncology, Istanbul Medical Faculty, Istanbul University, Istanbul, Turkey;

name: Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH;

name: Pediatric Hematology, University Hospital Città della Salute e della Scienza, Turin, Italy;

name: Paediatric and Congenital Cardiology Department, M3C Regional Reference Centre, Montpellier University Hospital, PhyMedExp, INSERM, Centre National de la Recherche Scientifique, Montpellier, France;

name: Hospital del Niño Jesus, San Miguel de Tucumán, Argentina;

name: McMaster Children’s Hospital, Hamilton, ON, Canada;

name: Aichi Children’s Health and Medical Center, Aichi, Japan;

name: Paediatric Haematology/Oncology Service, KK Women's and Children's Hospital, Singapore, Singapore;

name: Nemours Children’s Specialty Care, Jacksonville, FL;

name: Department of Clinical Haematology, Royal Children’s Hospital, Haematology Research Murdoch Children’s Research Institute, Department of Paediatrics, University of Melbourne, Melbourne, VIC, Australia; and

name: Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA

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

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

ReferenceCitation: Male C, Lensing AWA, Palumbo JS, Kumar R, Nurmeev I, Hege K, Bonnet D, Connor P, Hooimeijer HL, Torres M, Chan AKC, Kenet G, Holzhauer S, Santamaría A, Amedro P, Chalmers E, Simioni P, Bhat RV, Yee DL, Lvova O, Beyer-Westendorf J, Biss TT, Martinelli I, Saracco P, Peters M, Kállay K, Gauger CA, Massicotte MP, Young G, Pap AF, Majumder M, Smith WT, Heubach JF, Berkowitz SD, Thelen K, Kubitza D, Crowther M, Prins MH, Monagle P; EINSTEIN-Jr Phase 3 Investigators. Rivaroxaban compared with standard anticoagulants for the treatment of acute venous thromboembolism in children: a randomised, controlled, phase 3 trial. Lancet Haematol. 2020 Jan;7(1):e18-e27. doi: 10.1016/S2352-3026(19)30219-4. Epub 2019 Nov 5.

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

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

ReferenceCitation: Monagle P, Lensing AWA, Thelen K, Martinelli I, Male C, Santamaría A, Samochatova E, Kumar R, Holzhauer S, Saracco P, Simioni P, Robertson J, Grangl G, Halton J, Connor P, Young G, Molinari AC, Nowak-Göttl U, Kenet G, Kapsa S, Willmann S, Pap AF, Becka M, Twomey T, Beyer-Westendorf J, Prins MH, Kubitza D; EINSTEIN-Jr Phase 2 Investigators. Bodyweight-adjusted rivaroxaban for children with venous thromboembolism (EINSTEIN-Jr): results from three multicentre, single-arm, phase 2 studies. Lancet Haematol. 2019 Oct;6(10):e500-e509. doi: 10.1016/S2352-3026(19)30161-9. Epub 2019 Aug 13.

ReferenceCitation: Lensing AWA, Male C, Young G, Kubitza D, Kenet G, Patricia Massicotte M, Chan A, Molinari AC, Nowak-Goettl U, Pap ÁF, Adalbo I, Smith WT, Mason A, Thelen K, Berkowitz SD, Crowther M, Schmidt S, Price V, Prins MH, Monagle P. Rivaroxaban versus standard anticoagulation for acute venous thromboembolism in childhood. Design of the EINSTEIN-Jr phase III study. Thromb J. 2018 Dec 21;16:34. doi: 10.1186/s12959-018-0188-y. eCollection 2018.

repository_institution: University of Melbourne - Minerva Access

repository_normalized: University of Melbourne - Minerva Access

repositories: University of Melbourne - Minerva Access

repositories_institution: University of Melbourne - Minerva Access

location_country:
Finland
Australia
Belgium
Russian Federation
France
Japan
Slovakia
Argentina
Canada
Sweden
Hong Kong
Netherlands
Switzerland
Singapore
Hungary
Ireland
China
Portugal
Germany
United States
United Kingdom
Italy
Austria
Brazil
Mexico
Israel
Spain
Turkey

title: Multicenter, Open-label, Active-controlled, Randomized Study to Evaluate the Efficacy and Safety of an age-and Body Weight-adjusted Rivaroxaban Regimen Compared to Standard of Care in Children With Acute Venous Thromboembolism

summary: The purpose of this study is to evaluate comparative efficacy and safety of rivaroxaban to standard of care in children with acute venous thromboembolism.


title: An Open-label Extension Study of DS-5565 for 52 Weeks in Pain Associated With Fibromyalgia

summary: This is an open-label study of DS-5565 in subjects who either completed participation in a preceding Phase 3 study of DS-5565 in fibromyalgia (FM); i.e. DS5565-A-E309 (NCT02146430), DS5565-A-E310 (NCT02187471), or DS5565-A-E311 (NCT02187159) or are de novo subjects. Eligible subjects will be assigned to receive open-label DS-5565 for 52 weeks. All subjects will receive DS-5565 15 mg once daily (QD) for the first three weeks of the treatment period. After three weeks, subjects may be titrated to 15 mg twice daily (BID) based on protocol-specified criteria.

ReferenceCitation: Arnold LM, Whitaker S, Hsu C, Jacobs D, Merante D. Efficacy and safety of mirogabalin for the treatment of fibromyalgia: results from three 13-week randomized, double-blind, placebo- and active-controlled, parallel-group studies and a 52-week open-label extension study. Curr Med Res Opin. 2019 Oct;35(10):1825-1835. doi: 10.1080/03007995.2019.1629757. Epub 2019 Jul 9.

name: Women’s Health Research Program, University of Cincinnati College of Medicine, Cincinnati, OH, USA;

family: Arnold

full_name: Lesley M. Arnold

given: Lesley M.

name: Daiichi Sankyo Inc., Basking Ridge, NJ, USA;

family: Whitaker

full_name: Susan Whitaker

family: Hsu

full_name: Ching Hsu

given: Ching

family: Jacobs

full_name: David Jacobs

given: David

name: Global Clinical Development, Daiichi Sankyo Development, Gerrards Cross, United Kingdom

family: Merante

full_name: Domenico Merante

given: Domenico

journal_name: Current Medical Research and Opinion

publisher: Informa UK Limited

publisher_dissemination: Informa

publisher_group: Informa

publisher_normalized: Informa

ipd_sharing_description: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/

lead_sponsor: Daiichi Sankyo, Inc.

location_country:
Finland
Romania
Australia
Russian Federation
Denmark
Ukraine
France
Poland
Lithuania
Slovakia
Norway
Canada
Slovenia
South Africa
Chile
Bulgaria
Hungary
Puerto Rico
Portugal
United States
Germany
United Kingdom
Austria
Serbia
Czechia
New Zealand
Spain
Estonia
Latvia


title: A Phase 2, Dose-Ranging, Randomized, Double-Blind, Placebo-Controlled Study of GS-4997 in Subjects With Pulmonary Arterial Hypertension

acronym: ARROW

summary: The primary objective of this study is to evaluate the effect of selonsertib (GS-4997) on pulmonary vascular resistance (PVR), as measured by right heart catheterization (RHC) in adults with pulmonary arterial hypertension (PAH). The study will consist of a 24-week placebo-controlled treatment period and a long-term selonsertib treatment period. Participants completing the 24-week placebo-controlled period will be eligible to receive active treatment with selonsertib in the long-term treatment period.

ReferenceCitation: Rosenkranz S, Feldman J, McLaughlin VV, Rischard F, Lange TJ, White RJ, Peacock AJ, Gerhardt F, Ebrahimi R, Brooks G, Satler C, Frantz RP; ARROW Study Group. Selonsertib in adults with pulmonary arterial hypertension (ARROW): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Respir Med. 2021 Aug 20. pii: S2213-2600(21)00032-1. doi: 10.1016/S2213-2600(21)00032-1. [Epub ahead of print]

family: Rosenkranz

full_name: Stephan Rosenkranz

given: Stephan

family: Feldman

full_name: Jeremy Feldman

given: Jeremy

family: McLaughlin

full_name: Vallerie V McLaughlin

given: Vallerie V

family: Rischard

full_name: Franz Rischard

given: Franz

family: Lange

full_name: Tobias J Lange

given: Tobias J

family: White

full_name: R James White

given: R James

family: Peacock

full_name: Andrew J Peacock

given: Andrew J

family: Gerhardt

full_name: Felix Gerhardt

given: Felix

family: Ebrahimi

full_name: Ramin Ebrahimi

given: Ramin

family: Brooks

full_name: Gabriel Brooks

given: Gabriel

family: Satler

full_name: Carol Satler

given: Carol

family: Frantz

full_name: Robert P Frantz

given: Robert P

journal_name: The Lancet Respiratory Medicine

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

lead_sponsor: Gilead Sciences

location_country:
Italy
United States
Netherlands
France
Spain
Germany
United Kingdom
Canada

location_facility: Mary M. Parkes Center // University of Rochester Medical Center

location_facility: Klinik und Poliklinik fur Innere Medizin II, Universitatsklinikum Regensburg

location_facility: CHU de Grenoble Clinique Universitaire de Pneumologie

location_facility: Hopital Cardiologique-CHRU Lille, Service de cardiologie

location_facility: Scottish Pulmonary Vascular Unit, Golden Jubilee National Hospital

location_facility: Klinik III fur Innere Medizin, Herzzentrum Uniklinik Koln

location_facility: Vancouver General Hospital, The Lung Centre // Vancouver Coastal Health, Vancouver General Hospital

location_facility: Washington University School of Medicine

location_facility: CHU de Bicetre, Service de Pneumologie-Reanimation Respiratoire

location_facility: University of Arizona Clinical and Translational Science (CATS) Research Center

location_facility: University of South Alabama Medical Center

location_facility: VA Greater Los Angeles Healthcare System

location_facility: Victoria Hospital - London Health Sciences Centre

location_facility: University of Colorado Cardiac and Vascular Center, Anschutz Inpatient Pavillion

location_facility: Universita "Sapienza"-Azienda Policlinico Umberto 1

location_facility: Universitatsklinikul Giessen und Marburg GmbH

location_facility: Clinical Research Facility, Royal Hallamshrie Hospital

location_facility: Institut Universitaire de caridologie et de pneumologie de Quebee (IUCPQ)

location_facility: LA Biomedical Research Institute Harbor-UCLA Medical Center

location_facility: Hospital Universitario Doce (12) de Octubre


title: Multicenter Phase III Open Label Randomized Trial Comparing CPT-11 In Combination With A 5-FU/FA Infusional Regimen To The Same 5-FU/FA Infusional Regimen Alone As Adjuvant Treatment Of Stage III Colon Cancer

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug and giving them after surgery may kill more tumor cells.

PURPOSE: Randomized phase III trial to compare the effectiveness of fluorouracil and leucovorin with or without irinotecan in treating patients who have undergone surgery for stage III colorectal cancer.

ReferenceCitation: Punt CJ, Buyse M, Köhne CH, Hohenberger P, Labianca R, Schmoll HJ, Påhlman L, Sobrero A, Douillard JY. Endpoints in adjuvant treatment trials: a systematic review of the literature in colon cancer and proposed definitions for future trials. J Natl Cancer Inst. 2007 Jul 4;99(13):998-1003. Epub 2007 Jun 27. Review.

ReferenceCitation: Delorenzi M, Budinska E, Popovici V, et al.: Molecular classes in CRC: characterization of MSI by expression profiling in the translational study of the PETACC 3-EORTC 40993- SAKK 60-00 trial. [Abstract] J Clin Oncol 28 (Suppl 15): A-3597, 2010.

ReferenceCitation: Roth AD, Tejpar S, Delorenzi M, Yan P, Fiocca R, Klingbiel D, Dietrich D, Biesmans B, Bodoky G, Barone C, Aranda E, Nordlinger B, Cisar L, Labianca R, Cunningham D, Van Cutsem E, Bosman F. Prognostic role of KRAS and BRAF in stage II and III resected colon cancer: results of the translational study on the PETACC-3, EORTC 40993, SAKK 60-00 trial. J Clin Oncol. 2010 Jan 20;28(3):466-74. doi: 10.1200/JCO.2009.23.3452. Epub 2009 Dec 14.

name: From Oncosurgery, Geneva University Hospital, Geneva; National Center of Competence in Research Molecular Oncology, Swiss Institute of Bioinformatics, Lausanne; Swiss Group for Clinical Cancer Research, Bern; Department of Pathology, Lausanne University, Lausanne, Switzerland; Digestive Oncology Unit, University Hospital Gasthuisberg; Center For Human Genetics, Katholieke Universiteit Leuven, Leuven, Belgium; Department of Surgical and Morphological Sciences, University of Genova, Genova; Medical...

family: Roth

full_name: Arnaud D. Roth

given: Arnaud D.

family: Tejpar

full_name: Sabine Tejpar

given: Sabine

family: Delorenzi

full_name: Mauro Delorenzi

given: Mauro

family: Yan

full_name: Pu Yan

given: Pu

family: Fiocca

full_name: Roberto Fiocca

given: Roberto

family: Klingbiel

full_name: Dirk Klingbiel

given: Dirk

family: Dietrich

full_name: Daniel Dietrich

given: Daniel

family: Biesmans

full_name: Bart Biesmans

given: Bart

family: Bodoky

full_name: György Bodoky

given: György

family: Barone

full_name: Carlo Barone

given: Carlo

family: Aranda

full_name: Enrique Aranda

given: Enrique

family: Nordlinger

full_name: Bernard Nordlinger

given: Bernard

family: Cisar

full_name: Laura Cisar

given: Laura

family: Labianca

full_name: Roberto Labianca

family: Cunningham

full_name: David Cunningham

family: Van Cutsem

full_name: Eric Van Cutsem

given: Eric

family: Bosman

full_name: Fred Bosman

given: Fred

journal_name: Journal of Clinical Oncology

publisher: American Society of Clinical Oncology (ASCO)

title: Prognostic Role of KRAS and BRAF in Stage II and III Resected Colon Cancer: Results of the Translational Study on the PETACC-3, EORTC 40993, SAKK 60-00 Trial

ReferenceCitation: Bosman FT, Yan P, Tejpar S, Fiocca R, Van Cutsem E, Kennedy RD, Dietrich D, Roth A. Tissue biomarker development in a multicentre trial context: a feasibility study on the PETACC3 stage II and III colon cancer adjuvant treatment trial. Clin Cancer Res. 2009 Sep 1;15(17):5528-33. doi: 10.1158/1078-0432.CCR-09-0741. Epub 2009 Aug 18.

full_name: Fred T. Bosman

given: Fred T.

publisher_group: American Association for Cancer Research

title: Tissue Biomarker Development in a Multicentre Trial Context: a Feasibility Study on the PETACC3 Stage II and III Colon Cancer Adjuvant Treatment Trial

ReferenceCitation: Tejpar S, Bosman F, Delorenzi M, et al.: Microsatellite instability (MSI) in stage II and III colon cancer treated with 5FU-LV or 5FU-LV and irinotecan (PETACC 3-EORTC 40993-SAKK 60/00 trial). [Abstract] J Clin Oncol 27 (Suppl 15): A-4001, 2009.

ReferenceCitation: Van Cutsem E, Labianca R, Bodoky G, Barone C, Aranda E, Nordlinger B, Topham C, Tabernero J, André T, Sobrero AF, Mini E, Greil R, Di Costanzo F, Collette L, Cisar L, Zhang X, Khayat D, Bokemeyer C, Roth AD, Cunningham D. Randomized phase III trial comparing biweekly infusional fluorouracil/leucovorin alone or with irinotecan in the adjuvant treatment of stage III colon cancer: PETACC-3. J Clin Oncol. 2009 Jul 1;27(19):3117-25. doi: 10.1200/JCO.2008.21.6663. Epub 2009 May 18.

name: From the Digestive Oncology Unit, University Hospital Gasthuisberg, Leuven; European Organisation for Research and Treatment of Cancer Headquarters, Statistics Department, Brussels, Belgium; Department of Oncology, Ospedali Riuniti, Bergamo; Medical Oncology, Catholic University of the Sacred Heart, Rome; Medical Oncology, Department of Pharmacology, Ospedale San Martino, Genova; Unit of Medical Oncology, Azienda Ospedaliero Universitaria Careggi; Medical Oncology, University of Florence, Florence,...

title: Randomized Phase III Trial Comparing Biweekly Infusional Fluorouracil/Leucovorin Alone or With Irinotecan in the Adjuvant Treatment of Stage III Colon Cancer: PETACC-3

ReferenceCitation: Roth AD, Yan P, Dietrich D, et al.: Does UGT1A1*28 homozygosity predict for severe toxicity in patients treated with 5-fluorouracil (5-FU)-irinotecan (IRI)? Results of the PETACC 3-EORTC 40993-SAKK 60/00 trial comparing IRI/5-FU/folinic acid (FA) to 5-FU/FA in stage II-III colon cancer. [Abstract] American Society of Clinical Oncology 2008 Gastrointestinal Cancers Symposium, 25-27 January 2008, Orlando, FL. A-277, 2008.

ReferenceCitation: Roth AD, Yan P, Dietrich D, et al.: Is UGT1A1*28 homozygosity the strongest predictor for severe hematotoxicity in patients treated with 5-fluorouracil (5-FU)-irinotecan (IRI)? Results of the PETACC 3 - EORTC 40993 -SAKK 60/00 trial comparing IRI/5-FU/folinic acid (FA) to 5-FU/FA in stage II- III colon cancer (COC) patients. [Abstract] J Clin Oncol 26 (Suppl 15): A-4036, 2008.

ReferenceCitation: van Cutsem E, Labianca R, Hossfeld D, et al.: Randomized phase III trial comparing infused irinotecan / 5-fluorouracil (5-FU)/folinic acid (IF) versus 5-FU/FA (F) in stage III colon cancer patients (pts). (PETACC 3). [Abstract] J Clin Oncol 23 (Suppl 16): A-LBA8, 3s, 2005.

ReferenceCitation: Taieb J, Shi Q, Pederson L, Alberts S, Wolmark N, Van Cutsem E, de Gramont A, Kerr R, Grothey A, Lonardi S, Yoshino T, Yothers G, Sinicrope FA, Zaanan A, André T. Prognosis of microsatellite instability and/or mismatch repair deficiency stage III colon cancer patients after disease recurrence following adjuvant treatment: results of an ACCENT pooled analysis of seven studies. Ann Oncol. 2019 Sep 1;30(9):1466-1471. doi: 10.1093/annonc/mdz208.

ReferenceCitation: Klingbiel D, Saridaki Z, Roth AD, Bosman FT, Delorenzi M, Tejpar S. Prognosis of stage II and III colon cancer treated with adjuvant 5-fluorouracil or FOLFIRI in relation to microsatellite status: results of the PETACC-3 trial. Ann Oncol. 2015 Jan;26(1):126-132. doi: 10.1093/annonc/mdu499. Epub 2014 Oct 30.

location_country:
Italy
Austria
Switzerland
Spain
France
Belgium
Egypt
Portugal
Germany
United Kingdom

location_facility: Universita Degli Studi di Firenze - Policlin. di Careggi

location_facility: Hopital Cantonal Universitaire de Geneva

location_facility: Instituto Portugues de Oncologia do Porto


title: A Feasibility Study Of Primary Chemotherapy Followed By Concomitant Chemoradiation With And Without Amifostine In Patients With Locally Advanced Undifferentiated Nasopharyngeal Cancer (UNPC)

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining radiation therapy with chemotherapy may kill more tumor cells. Giving chemoprotective drugs such as amifostine may protect normal cells from the side effects of chemotherapy and radiation therapy.

PURPOSE: Randomized phase II trial to compare the effectiveness of paclitaxel and carboplatin followed by cisplatin plus radiation therapy with or without amifostine in treating patients who have locally advanced cancer of the nasopharynx.

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country:
Italy
France
Belgium
Spain
Turkey
United Kingdom

location_facility: Istituto Nazionale per la Ricerca sul Cancro

location_facility: Istituto Nazionale per lo Studio e la Cura dei Tumori

location_facility: Istanbul University-Institute of Oncology

intervention_type: Radiation


status: Not yet recruiting

study_start_date_type: Anticipated

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

lead_sponsor: EMD Serono Research & Development Institute, Inc.

location_country:
United States
France
Korea, Republic of
Germany
Canada

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Institut Curie - Centre de Lutte Contre le Cancer (CLCC) de Paris

title: Single-arm, Multicenter Phase I/Ib Study of Avelumab + Lenvatinib in Children With Primary CNS Tumors

summary: This study consists of 2 parts: Dose Escalation Part 1 and Dose Expansion Part 2. The Dose Escalation Part 1 will evaluate the safety and tolerability of Avelumab in combination with Lenvatinib and determine the recommended Avelumab and Lenvatinib dose for expansion. Dose Expansion Part 2 will assess the efficacy of Avelumab in combination with Lenvatinib by Progression-free Survival in participants with pre-defined primary central nervous system (CNS) tumors.


title: A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients With GSD III

summary: The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
Italy
France
Spain
United States
United Kingdom
Canada

intervention_type: Other


title: A 2-year Randomized, 3-arm, Double-blind, Non-inferiority Study Comparing the Efficacy and Safety of Ofatumumab and Siponimod Versus Fingolimod in Pediatric Patients With Multiple Sclerosis Followed by an Open-label Extension

acronym: NEOS

summary: Efficacy and safety of ofatumumab and siponimod compared to fingolimod in pediatric patients with multiple sclerosis

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

lead_sponsor: Novartis Pharmaceuticals

location_country:
Austria
France
Poland
Australia
Belgium
United States


title: A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

acronym: SMART

summary: To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.

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 https://www.clinicalstudydatarequest.com/.

location_country: France Belgium Australia Portugal Canada

intervention_type: Genetic


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

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

location_country:
Italy
France
Czechia
Japan
Malaysia
Singapore
China
Germany
United Kingdom
Korea, Republic of

acronym: APPOINT-PNH

title: A Multicenter, Single-arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

summary: The purpose of this Phase 3 study is to determine whether iptacopan is efficacious and safe for the treatment of PNH patients who are naive to complement inhibitor therapy


title: An Open-Label, Multi-Centre Phase I/IIa Study Evaluating the Safety and Clinical Activity of Neoantigen Reactive T Cells in Patients With Advanced Non-Small Cell Lung Cancer

summary: This is a first-in-human, open-label, multi-centre, phase I/IIa study to characterise the safety and clinical activity autologous clonal neoantigen reactive T cells (cNeT) administered intravenously in adults with advanced non-small cell lung cancer (NSCLC).

lead_sponsor: Achilles Therapeutics UK Limited

location_country:
United States
France
Spain
Germany
United Kingdom

location_facility: Guys and St Thomas' NHS Foundation Trust

location_facility: University Hospital Southampton NHS Foundation Trust

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: University College London Hospital (UCLH)

location_facility: Cambridge University Hospitals NHS Foundation Trust

location_facility: Universitätsklinikum Carl Gustav Carus an der TU Dresden Anstalt des öffentlichen Rechts des Freistaates Sachsen

location_facility: H Lee Moffitt Cancer Center and Research Unit

location_facility: Hospital Universitario Fundación Jiménez Díaz


title: Stereotactic Ablative Body Radiotherapy (SABR) Combined With Immunotherapy (L19-IL2) in Stage IV NSCLC Patients, ImmunoSABR: a Multicentre, Randomised Controlled Open-label Phase II Trial

summary:
This will be a phase II trial testing if the combination of stereotactic ablative body radiotherapy (SABR) and L19-IL2 improve the progression-free survival in patients with limited metastatic non-small cell lung cancer (NSCLC). The trial consists of one cohort with two arms; C-arm and an E-arm.

Patients with oligometastatic disease will receive SABR to minimal 1 and max all metastatic sites (max 5 sites irradiated) and patients with diffuse metastatic lesions (6 to max 10) will receive radiotherapy to max 5 sites. In the experimental arm, immunotherapy will be given after irradiation.

ReferenceCitation: Olivo Pimentel V, Marcus D, van der Wiel AM, Lieuwes NG, Biemans R, Lieverse RI, Neri D, Theys J, Yaromina A, Dubois LJ, Lambin P. Releasing the brakes of tumor immunity with anti-PD-L1 and pushing its accelerator with L19-IL2 cures poorly immunogenic tumors when combined with radiotherapy. J Immunother Cancer. 2021 Mar;9(3). pii: e001764. doi: 10.1136/jitc-2020-001764.

family: Olivo Pimentel

full_name: Veronica Olivo Pimentel

given: Veronica

family: Marcus

full_name: Damiënne Marcus

given: Damiënne

family: van der Wiel

full_name: Alexander MA van der Wiel

given: Alexander MA

family: Lieuwes

full_name: Natasja G Lieuwes

given: Natasja G

family: Biemans

full_name: Rianne Biemans

given: Rianne

family: Lieverse

full_name: Relinde IY Lieverse

given: Relinde IY

family: Neri

full_name: Dario Neri

given: Dario

family: Theys

full_name: Jan Theys

given: Jan

family: Yaromina

full_name: Ala Yaromina

given: Ala

family: Dubois

full_name: Ludwig J Dubois

given: Ludwig J

family: Lambin

full_name: Philippe Lambin

given: Philippe

currency_apc_doaj: USD

journal_name: Journal for ImmunoTherapy of Cancer

repository_institution: ETH Zurich - Repository for Publications and Research Data

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

title: Releasing the brakes of tumor immunity with anti-PD-L1 and pushing its accelerator with L19–IL2 cures poorly immunogenic tumors when combined with radiotherapy

ReferenceCitation: Lieverse RIY, Van Limbergen EJ, Oberije CJG, Troost EGC, Hadrup SR, Dingemans AC, Hendriks LEL, Eckert F, Hiley C, Dooms C, Lievens Y, de Jong MC, Bussink J, Geets X, Valentini V, Elia G, Neri D, Billiet C, Abdollahi A, Pasquier D, Boisselier P, Yaromina A, De Ruysscher D, Dubois LJ, Lambin P. Stereotactic ablative body radiotherapy (SABR) combined with immunotherapy (L19-IL2) versus standard of care in stage IV NSCLC patients, ImmunoSABR: a multicentre, randomised controlled open-label phase II trial. BMC Cancer. 2020 Jun 15;20(1):557. doi: 10.1186/s12885-020-07055-1.

full_name: Relinde I. Y. Lieverse

given: Relinde I. Y.

family: Van Limbergen

full_name: Evert J. Van Limbergen

given: Evert J.

family: Oberije

full_name: Cary J. G. Oberije

given: Cary J. G.

family: Troost

full_name: Esther G. C. Troost

given: Esther G. C.

family: Hadrup

full_name: Sine R. Hadrup

given: Sine R.

family: Dingemans

repository_institution: Technical University of Denmark, DTU - Technical University of Denmark, DTU Orbit

repository_institution: Ghent University - Ghent University Academic Bibliography

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

repositories_institution: University College London - UCL Discovery

location_country:
Italy
Netherlands
France
Belgium
Germany
United Kingdom

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS Università Cattolica del Sacro Cuore

location_facility: INSTITUT régional du CANCER MONTPELLIER - ICM - VAL d'AURELLE

location_facility: Academisch Ziekenhuis Maastricht (Leading Centre)


type: Other Identifier

title: International, Non-interventional, Post-Authorization Long-Term Safety Study of Lutathera, in Patients With Unresectable or Metastatic, Well-Differentiated, Somatostatin Receptor Positive, Gastroenteropancreatic Neuroendocrine Tumours

acronym: SALUS

summary: Study to assess the long-term safety of LUTATHERA for the labeled indication (SmPC/USPI).

time_perspective: Other

lead_sponsor: Advanced Accelerator Applications

location_country:
United States
United Kingdom
France
Portugal

location_facility: Guys Hospital Nuclear Medicine Department

location_facility: The Ohio State University Wexner Medical Center

location_facility: Oregon Health & Sciences University Hospital

location_facility: Centro Hospitalar e Universitario de Coimbra, E.P.E.


ReferenceCitation: Garrelfs SF, Frishberg Y, Hulton SA, Koren MJ, O'Riordan WD, Cochat P, Deschênes G, Shasha-Lavsky H, Saland JM, Van't Hoff WG, Fuster DG, Magen D, Moochhala SH, Schalk G, Simkova E, Groothoff JW, Sas DJ, Meliambro KA, Lu J, Sweetser MT, Garg PP, Vaishnaw AK, Gansner JM, McGregor TL, Lieske JC; ILLUMINATE-A Collaborators. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021 Apr 1;384(13):1216-1226. doi: 10.1056/NEJMoa2021712.

name: From the Department of Pediatric Nephrology, Emma Children’s Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women’s and Children’s Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London — both in the United Kingdom;...

family: Garrelfs

full_name: Sander F. Garrelfs

given: Sander F.

family: Frishberg

full_name: Yaacov Frishberg

given: Yaacov

family: Hulton

full_name: Sally A. Hulton

given: Sally A.

family: Koren

full_name: Michael J. Koren

given: Michael J.

family: O’Riordan

full_name: William D. O’Riordan

given: William D.

family: Cochat

full_name: Pierre Cochat

given: Pierre

family: Deschênes

full_name: Georges Deschênes

given: Georges

family: Shasha-Lavsky

full_name: Hadas Shasha-Lavsky

given: Hadas

family: Saland

full_name: Jeffrey M. Saland

given: Jeffrey M.

family: van’t Hoff

full_name: William G. van’t Hoff

given: William G.

family: Fuster

full_name: Daniel G. Fuster

given: Daniel G.

family: Magen

full_name: Daniella Magen

given: Daniella

family: Moochhala

full_name: Shabbir H. Moochhala

given: Shabbir H.

family: Schalk

full_name: Gesa Schalk

given: Gesa

family: Simkova

full_name: Eva Simkova

given: Eva

family: Groothoff

full_name: Jaap W. Groothoff

given: Jaap W.

family: Sas

full_name: David J. Sas

given: David J.

family: Meliambro

full_name: Kristin A. Meliambro

given: Kristin A.

family: Lu

full_name: Jiandong Lu

given: Jiandong

family: Sweetser

full_name: Marianne T. Sweetser

given: Marianne T.

family: Garg

full_name: Pushkal P. Garg

given: Pushkal P.

family: Vaishnaw

full_name: Akshay K. Vaishnaw

given: Akshay K.

family: Gansner

full_name: John M. Gansner

given: John M.

family: McGregor

full_name: Tracy L. McGregor

given: Tracy L.

family: Lieske

full_name: John C. Lieske

given: John C.

location_country:
United States
United Arab Emirates
Switzerland
Netherlands
France
Israel
Germany
United Kingdom

title: ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

summary: The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).