title: A Phase I First Time in Human Open Label Study of GSK3745417 Administered With and Without Anticancer Agents in Participants With Advanced Solid Tumors

summary: This study aims to evaluate the safety, tolerability, and preliminary clinical activity and establish a recommended dose of GSK3745417 administered alone (Part 1A) or co-administered (Part 2A) with dostarlimab in participants with refractory/relapsed solid tumors. Both parts will consist of a dose escalation phase.

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

lead_sponsor: GlaxoSmithKline

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


type: EudraCT Number

title: A Multicentre, Open-Label Phase I/II Study to Evaluate the Safety, Tolerability, Biodistribution and Anti Tumour Activity of 177LU-OPS201 With Companion Imaging 68Ga OPS202 PET/CT in Previously Treated Subjects With Locally Advanced or Metastatic Cancers Expressing Somatostatin Receptor 2 (SSTR2)

summary: This study consists of two phases. The phase I study is designed to investigate the safety and tolerability of Satoreotide tetraxetan following fractionated i.v. administrations in pre-treated subjects with locally advanced or metastatic cancers expressing sstr2 as identified by Satoreotide trizoxetan Positron Emission Tomography (PET/CT) scans. This phase will encompass both radioactivity escalation and peptide mass dose evaluation. Phase II will assess the efficacy of Satoreotide tetraxetan in subjects in selected indications, in a basket design.

study_start_date_type: Actual

status: Terminated

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Other

enrollment_type: Actual

ipd_sharing: No

lead_sponsor: Ipsen

location_country:
Germany
United Kingdom
Switzerland
Austria
Belgium
United States
France

location_facility:
Universitaetsklinikum Essen
University Hospital Basel
Royal Marsden Hospital - Surrey
CHU de Marseille - Hôpital la Timone
MD Anderson Cancer Center
CHU de Bordeaux - Hôpital Haut Lévêque
Medical University of Innsbruck
University Hospital (UZ) Leuven

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Unknown


title: A Prospective Multicenter Clinical Study to Evaluate the Safety and Effectiveness of Subepithelial Implantation of the Transform Corneal Allograft (TCA) for Improving Near Vision in Presbyopic Subjects

summary: The objective of this clinical study is to evaluate the safety and effectiveness of subepithelial implantation of the Allotex TransForm corneal allograft (TCA) for improving near vision in presbyopic subjects.

study_start_date_type: Anticipated

status: Withdrawn

study_completion_date_type: Anticipated

primary_purpose: Treatment

lead_sponsor: Allotex, Inc.

location_country:
Ireland
United Kingdom
Switzerland
Austria
Belgium
France

location_facility: Institute Laser Vision Noemie de Rothschild, Fondation Ophthalmolique Adolphe de Rothschild

location_facility: Corneo Plastic Unit and Eye Bank Queen Victoria Hospital

intervention_type: Other


title: A Prospective Multicenter Clinical Study to Evaluate the Safety and Effectiveness of Intrastromal Implant of the Transform Corneal Allograft (TCA) for Providing Near Vision in Presbyopic Subjects

summary: The objective of this clinical study is to evaluate the safety and effectiveness of intrastromal implantation of the Allotex TransForm corneal allograft (TCA) for improving near vision in presbyopic subjects.

status: Unknown status


title: A Prospective Multi Center Clinical Study to Evaluate the Safety And Effectiveness of the Transform™ Corneal Allograft (TCA) for Treatment of Hyperopia

summary:
The objective of this clinical study is to evaluate the safety and effectiveness of intrastromal implantation of the Allotex TCA for improving distance vision in hyperopic subjects.

The overall objective with respect to visual outcome is to provide improved vision without the requirement of additional visual aids.


title: A Phase I/IIA, Multi-Centre, Open-Label, Dose-Escalation Study With Expansion Arms to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CB-103 Administered Orally in Adult Patients With Locally Advanced or Metastatic Solid Tumours and Haematological Malignancies Characterised by Alterations of the NOTCH Signalling Pathway

summary: This is a phase I/II, non randomized, open-label, dose escalation study to investigate the safety, tolerability and preliminary efficacy of CB-103.

lead_sponsor: Cellestia Biotech AG

location_country:
Germany
Switzerland
United States
Spain
Korea, Republic of
France

location_facility: Nationales Centrum für Tumorerkrankungen Heidelberg

location_facility: Oncology Institute of Southern Switzerland

location_facility: Severance Hospital - Yonsei Cancer Center

location_facility: Vall d'Hebron Institute of Oncology (VHIO)

location_facility: Charite- Universitaetsmedizin Berlin- Campus Benjamin Franklin


acronym: TETHYS

lead_sponsor: Fresenius Kabi

summary: The aim of the study is to investigate the safety of a 6% hydroxyethyl starch (HES) solution (Volulyte 6%) versus an electrolyte solution (Ionolyte) in trauma patients.

ReferenceType: derived

ReferenceCitation: Palma CD, Mamba M, Geldenhuys J, Fadahun O, Rossaint R, Zacharowski K, Brand M, Diaz-Cambronero O, Belda J, Westphal M, Brauer U, Dormann D, Dehnhardt T, Hernandez-Gonzalez M, Schmier S, de Korte D, Plani F, Buhre W. PragmaTic, prospEctive, randomized, controlled, double-blind, mulTi-centre, multinational study on the safety and efficacy of a 6% HydroxYethyl Starch (HES) solution versus an electrolyte solution in trauma patients: study protocol for the TETHYS study. Trials. 2022 Jun 2;23(1):456. doi: 10.1186/s13063-022-06390-x.

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

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

host_type: repository

repository_institution: Goethe University Frankfurt - Publication Server of Goethe University Frankfurt am Main

evidence: oa repository (via pmcid lookup)

license_normalized: no license

repository_normalized: PubMed Central

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

repository_institution: Research Square - Research Square

repository_normalized: Research Square

version: submittedVersion

repositories:
PubMed Central
Research Square
publikationen.ub.uni-frankfurt.de

unpaywall_oa_status: gold

publisher_dissemination: Springer-Nature

ReferenceCitation: Chappell D, Jacob M. Should hydroxyethyl starch be banned? Lancet. 2018 Jul 14;392(10142):118. doi: 10.1016/S0140-6736(18)31174-7. No abstract available.

oa_host_type: publisher

evidence: open (via free pdf)

repositories_concat: closed

unpaywall_oa_status: bronze

publisher_dissemination: Elsevier

sponsor_collaborators: European Society of Anaesthesiology and Intensive Care

location_facility: Academic Medical Center (AMC) Anesthesiology

location_facility: University Medical Center (UMC) Maastricht

location_facility: Gama Research Centre Emergency Department, Leratong Hospital

location_facility: Trident Clinical, Homestead Medical Centre

location_facility: University Hospital Schleswig-Holstein Campus Kiel

location_facility: Universitätsklinikum Carl Gustav Carus Dresden

location_facility: Hospital Universitari i Politecnic la Fe

status: Completed

title: Pragmatic, Prospective, Randomized, Controlled, Double-blind, Multi-centre, Multinational Study on the Safety and Efficacy of a 6% Hydroxyethyl Starch (HES) Solution Versus an Electrolyte Solution in Trauma Patients

type: Other Identifier

source: euctr

design_allocation: Randomized

location_country:
Germany
Netherlands
Belgium
Spain
South Africa
Czechia
France

status_simplified: Completed


title: A Multi-centre, Randomised Controlled Study, to Evaluate the Safety and Performance of The DIALIVE Liver Dialysis Device (LDD) in Patients With Acute on Chronic Liver Failure (ACLF) Versus Standard of Care (SOC)

acronym: DIALIVE _ACLF

summary: The First-In-Man study is a multi-centre, randomised, controlled, study to generate data for the evaluation of safety and performance of DIALIVE Liver Dialysis Device in 24 evaluable patients with Acute on Chronic Liver Failure (ACLF) versus standard of care (SOC).

primary_purpose: Device Feasibility

ReferenceType: background

ReferenceCitation: Lee KC, Baker LA, Stanzani G, Alibhai H, Chang YM, Jimenez Palacios C, Leckie PJ, Giordano P, Priestnall SL, Antoine DJ, Jenkins RE, Goldring CE, Park BK, Andreola F, Agarwal B, Mookerjee RP, Davies NA, Jalan R. Extracorporeal liver assist device to exchange albumin and remove endotoxin in acute liver failure: Results of a pivotal pre-clinical study. J Hepatol. 2015 Sep;63(3):634-42. doi: 10.1016/j.jhep.2015.04.020. Epub 2015 May 1.

ipd_sharing_description: Only anonymized clinical data can be shared out of central database according to the H2020 project plan.

lead_sponsor: Yaqrit Ltd

collaborators: European Foundation for Chronic Liver Failure

location_country:
Germany
United Kingdom
Denmark
Romania
Austria
Belgium
Spain
France

location_facility:
Hospital Clinic Barcelona
Royal Free Hospital London NHS
university Hospital Graz
University Hospital of Rostock
University Hospital Erasmus
Queens Medical Centre
Fundeni Clinical Institute
University Hospital Gregorio Maragnon
Hôpital Beaujon
university hospital BASILDON
Paul Brousse Hospital
University Hospital

intervention_type: Device


acronym: Mini-COMET

lead_sponsor: Genzyme, a Sanofi Company

summary:
Primary Objective:

To evaluate the safety profile of avalglucosidase alfa in participants with infantile-onset Pompe disease previously treated with alglucosidase alfa.

Secondary Objective:

To characterize the pharmacokinetic profile of avalglucosidase alfa and to evaluate the preliminary efficacy of avalglucosidase alfa in comparison to alglucosidase alfa.

location_facility:
Investigational Site Number 1580001
Investigational Site Number 8400005
Investigational Site Number 8260001
Investigational Site Number 8400001
Investigational Site Number 3920002
Investigational Site Number 2500003
Investigational Site Number 8400002
Investigational Site Number 8260002
Investigational Site Number 2500001
Investigational Site Number 3920001

status: Active, not recruiting

title: An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response

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

location_country:
United Kingdom
Japan
United States
Taiwan
France

ipd_sharing: Yes

status_simplified: Ongoing


acronym: RSV F-021

summary: The purpose of this study is to rank different RSV vaccine dosages of antigen (or formulations) based on safety/reactogenicity and immune response data. The formulations eliciting strong immune responses while maintaining an acceptable safety profile will be considered for further evaluation, including in studies vaccinating pregnant women.

ReferenceCitation: Steff AM, Cadieux-Dion C, de Lannoy G, Prato MK, Czeszak X, Andre B, Ingels DC, Louckx M, Dewe W, Picciolato M, Maleux K, Fissette L, Dieussaert I. Hamster neogenin, a host-cell protein contained in a respiratory syncytial virus candidate vaccine, induces antibody responses in rabbits but not in clinical trial participants. Hum Vaccin Immunother. 2020 Jun 2;16(6):1327-1337. doi: 10.1080/21645515.2019.1693749. Epub 2020 Jan 17.

oa_colors: green hybrid

evidence: open (via crossref license)

repositories_concat: PubMed Central

unpaywall_oa_status: hybrid

publisher_dissemination: Informa

ReferenceCitation: Schwarz TF, McPhee RA, Launay O, Leroux-Roels G, Talli J, Picciolato M, Gao F, Cai R, Nguyen TL, Dieussaert I, Miller JM, Schmidt AC. Immunogenicity and Safety of 3 Formulations of a Respiratory Syncytial Virus Candidate Vaccine in Nonpregnant Women: A Phase 2, Randomized Trial. J Infect Dis. 2019 Oct 22;220(11):1816-1825. doi: 10.1093/infdis/jiz395.

repository_institution: PubMed Central - Europe PMC

publisher_dissemination: Oxford University Press

title: An Observer-blind Study to Rank Different Formulations of GSK Biologicals' Investigational RSV Vaccine (GSK3003891A) Administered to Healthy Women

primary_purpose: Prevention

location_country: Germany Estonia Belgium France


title: Assessing Clinical Outcomes Using the EDWARDS INTUITY Elite Valve System in Isolated AVR Using Minimally InvaSive Surgery In a EurOpean Multi-ceNter, Active, Post-market Registry

acronym: MISSION

summary: The purpose of this active, observational, open-label, non-randomized, post-market surveillance study is to confirm that EDWARDS INTUITY Elite reduces cross clamp time (XCT) in MIS setting when compared to published data with a conventional valve within the MIS setting. The published dataset will used as a control group. Then to describe short term (30 days) and long term (6 months) clinical safety, to assess and compare hemodynamic data with EDWARDS INTUITY Elite to a conventional valve at discharge and at 6 months post AVR, to assess Quality of Life at baseline, and at 6 months post AVR to assess NYHA functional class at baseline, discharge, 1 month and at 6 months post AVR to assess Fitness for hospital discharge.

study_type: Observational

time_perspective: Other

ReferenceCitation: Laufer G, Strauch JT, Terp KA, Salinas M, Arribas JM, Massetti M, Andreas M, Young CP. Real-world 6-month outcomes of minimally invasive aortic valve replacement with the EDWARDS INTUITY Elite valve system. Interact Cardiovasc Thorac Surg. 2022 Jul 9;35(2):ivac083. doi: 10.1093/icvts/ivac083.

repository_institution: Elsevier - Scopus

repository_normalized: Pure (Denmark)

repositories:
PubMed Central
Pure (Denmark)

repositories_concat: PubMed Central;Pure (Denmark)

lead_sponsor: Edwards Lifesciences

location_country:
Germany
Italy
United Kingdom
Denmark
Netherlands
Austria
Spain
Luxembourg
France

location_facility: Complexo Hospitalario Universitario A Coruna

location_facility: Hôpital Cardiologique CHU Bordeaux Haut Leveque

location_facility: Universitätsklinikum der Ruhr-Universität Bochum Klinik für Herz- und Thoraxchirurgie

location_facility: G. Pasquinucci Heart Hospital - "G.Monasterio" Foundation

location_facility: Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia"

location_facility: Università Cattolica del Sacro Cuore Policlinico

location_facility: Institut National de Chirurgie Cardiaque et Cardiologie Interventionnelle (INCCI)

location_facility: Klinisshe Abteiluing Für Herz-thoraxchirurgie

location_facility: Herz- und Gefäß-Klinik GmbH Bad Neustadt

location_facility: Universitätsklinik für Herzchirurgie Medizinische Universität Innsbruck

location_facility: Hospital Universitario Virgen de la Arrixaca


title: The SPACER Trial - Repair of Tricuspid Valve Regurgitation Using the Edwards TricuSPid TrAnsCatheter REpaiR System

acronym: SPACER

summary: The purpose of the study is to assess the safety and device performance of the Edwards Tricuspid Transcatheter Repair System in patients with clinically significant, symptomatic, tricuspid regurgitation who are at high surgical risk for standard tricuspid repair/replacement.

location_country: Germany Switzerland Greece Canada France

location_facility: Universitäts-Herzzentrum Freiburg - Bad Krozingen

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Quebec-Universite Laval

location_facility: St. Paul's Hospital, Providence Health Care Research Institute

location_facility: Munich University Clinic, Ludwig-Maximilian University


acronym: CALM

intervention_type: Biological

lead_sponsor: Institut de Recherches Internationales Servier

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.

ReferenceType: result

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.

oa_host_type: closed

ReferenceCitation: Benjamin R, Jain N, Maus MV, Boissel N, Graham C, Jozwik A, Yallop D, Konopleva M, Frigault MJ, Teshima T, Kato K, Boucaud F, Balandraud S, Gianella-Borradori A, Binlich F, Marchiq I, Dupouy S, Almena-Carrasco M, Pannaux M, Fouliard S, Brissot E, Mohty M; CALM Study Group. UCART19, a first-in-class allogeneic anti-CD19 chimeric antigen receptor T-cell therapy for adults with relapsed or refractory B-cell acute lymphoblastic leukaemia (CALM): a phase 1, dose-escalation trial. Lancet Haematol. 2022 Nov;9(11):e833-e843. doi: 10.1016/S2352-3026(22)00245-9. Epub 2022 Oct 10.

location_facility: University of Texas MD Anderson Cancer Center

location_facility: Hospital of the University of Pennsylvania

location_facility: King's College Hospital NHS Foundation Trust

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)

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.

location_country:
United States
United Kingdom
Japan
France


title: REDUCER-I: An Observational Study of the Neovasc Reducer™ System

summary: The purpose of this study is to collect long term data of the Neovasc Reducer System in subjects with refractory angina pectoris.

enrollment_type: Anticipated

ReferenceCitation: Banai S, Ben Muvhar S, Parikh KH, Medina A, Sievert H, Seth A, Tsehori J, Paz Y, Sheinfeld A, Keren G. Coronary sinus reducer stent for the treatment of chronic refractory angina pectoris: a prospective, open-label, multicenter, safety feasibility first-in-man study. J Am Coll Cardiol. 2007 May 1;49(17):1783-9. doi: 10.1016/j.jacc.2007.01.061.

ReferenceCitation: Banai S, Verheye S, Jolicoeur EM. A device to narrow the coronary sinus for angina. N Engl J Med. 2015 May 14;372(20):1967-8. doi: 10.1056/NEJMc1503672. No abstract available.

ReferenceCitation: Verheye S, Jolicoeur EM, Behan MW, Pettersson T, Sainsbury P, Hill J, Vrolix M, Agostoni P, Engstrom T, Labinaz M, de Silva R, Schwartz M, Meyten N, Uren NG, Doucet S, Tanguay JF, Lindsay S, Henry TD, White CJ, Edelman ER, Banai S. Efficacy of a device to narrow the coronary sinus in refractory angina. N Engl J Med. 2015 Feb 5;372(6):519-27. doi: 10.1056/NEJMoa1402556.

ReferenceCitation: Konigstein M, Verheye S, Jolicoeur EM, Banai S. Narrowing of the Coronary Sinus: A Device-Based Therapy for Persistent Angina Pectoris. Cardiol Rev. 2016 Sep-Oct;24(5):238-43. doi: 10.1097/CRD.0000000000000101.

ReferenceCitation: Abawi M, Nijhoff F, Stella PR, Voskuil M, Benedetto D, Doevendans PA, Agostoni P. Safety and efficacy of a device to narrow the coronary sinus for the treatment of refractory angina: A single-centre real-world experience. Neth Heart J. 2016 Sep;24(9):544-51. doi: 10.1007/s12471-016-0862-2.

ReferenceCitation: Konigstein M, Meyten N, Verheye S, Schwartz M, Banai S. Transcatheter treatment for refractory angina with the Coronary Sinus Reducer. EuroIntervention. 2014 Feb;9(10):1158-64. doi: 10.4244/EIJV9I10A196.

ReferenceCitation: Verheye S, Agostoni P, Giannini F, Hill JM, Jensen C, Lindsay S, Stella PR, Redwood S, Banai S, Konigstein M. Coronary sinus narrowing for the treatment of refractory angina: a multicentre prospective open-label clinical study (the REDUCER-I study). EuroIntervention. 2021 Sep 20;17(7):561-568. doi: 10.4244/EIJ-D-20-00873.

lead_sponsor: Neovasc Inc.

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

location_facility: Contilia Heart and Vascular Centre, Elisabeth Krankenhaus Essen, Klinik fϋr Kardiologie und Angiologie

location_facility: University Heart Center Freiburg - Bad Krozingen


title: Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C

summary:
A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick disease type C (NPC).

The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the participant's current prescribed best routine clinical care; participant's routine clinical care may, or may not, include miglustat.

The CT-ORZY-NPC-002 study has been expanded to include an open label paediatric sub-study including participants aged 6 to <24 months at study enrolment.

ReferenceCitation: Patterson MC, Lloyd-Price L, Guldberg C, Doll H, Burbridge C, Chladek M, iDali C, Mengel E, Symonds T. Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale. Orphanet J Rare Dis. 2021 Feb 12;16(1):79. doi: 10.1186/s13023-021-01719-2.

ReferenceCitation: Mengel E, Patterson MC, Da Riol RM, Del Toro M, Deodato F, Gautschi M, Grunewald S, Gronborg S, Harmatz P, Heron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Andreasen AK, Geist MA, Havnsoe Torp Petersen N, Ingemann L, Hansen T, Blaettler T, Kirkegaard T, I Dali C. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021 Nov;44(6):1463-1480. doi: 10.1002/jimd.12428. Epub 2021 Sep 7.

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

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

repository_normalized: HAL

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

repository_institution: California Digital Library - eScholarship

publisher_dissemination: Wiley

lead_sponsor: ZevraDenmark

location_country:
Germany
Italy
Poland
United Kingdom
Denmark
Switzerland
United States
Spain
France

location_facility: Villa Metabolica, Universitätsmedizin Mainz

location_facility: Dr. von Haunersches Kinderspital der Universität München

location_facility: University Hospital Copenhagen (Rigshospitalet)

location_facility: Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" di Udin

location_facility: UCSF Benioff Children's Hospital Oakland


title: A Phase Ib Dose Escalation Study of BCL201 in Combination With Idelalisib in Patients With Follicular Lymphoma (FL) and Mantle Cell Lymphoma (MCL)

summary:
This is a phase Ib multi-center, open-label study: escalation part followed by expansion part. The primary purpose of the Phase Ib CBCL201X2102C study is to characterize the safety and tolerability of BCL201 combined with idelalisib in patients with FL and MCL.

Approximately 65 patients are to be enrolled.

The primary endpoint for the Phase Ib is frequency, severity and seriousness of AEs, lab abnormalities and other safety parameters such as ECG changes. An adaptive Bayesian logistic regression model (BLRM) will guide the dose escalation to determine the MTD/RDE in phase Ib. In addition Bayesian regression models will be used to estimate the dose-exposure relationships for both BCL201 and idelalisib in order to guide the escalation steps. A Bayesian method for the expansion part will be used for the primary activity objective.

The study data will be analyzed and reported based on all patients' data of the escalation and expansion part.

design_allocation: Non-Randomized

lead_sponsor: Novartis Pharmaceuticals

location_country:
United States
Germany
Austria
France


title: A Prospective, Non-comparative, Multi-center Clinical Trial to Evaluate the Safety and Efficacy of the STARflo Glaucoma Implant in Patients With Open Angle Glaucoma

summary: Prospective, Non-comparative, Multi-center clinical trial to evaluate the safety and efficacy of the STARflo Glaucoma Implant in patients with refractory open angle glaucoma.

lead_sponsor: iSTAR Medical

location_country: Germany Switzerland Belgium Bulgaria France

location_facility: Universitatsklinik fur Augenheilkunde Inselspital


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.

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. doi: 10.1056/NEJM200108023450501.

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. doi: 10.1111/j.1528-1167.2007.01421.x. 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.

ReferenceCitation: Blumcke 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.

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, Trankle C, Eyupoglu IY, Wirth B, Blumcke 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.

ReferenceCitation: Gottlicher M, Minucci S, Zhu P, Kramer 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. doi: 10.1093/emboj/20.24.6969.

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.

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, Muhlebner A, El-Osta A, Blumcke 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

sponsor_collaborators:
University College, London
University of Liverpool

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

location_facility: Great Ormond Street Hospital for Children NHS Trust

intervention_type: Dietary Supplement

lead_sponsor_type: academique


type: Other Grant/Funding Number

title: Swiss Trial of Decompressive Craniectomy Versus Best Medical Treatment of Spontaneous Supratentorial Intracerebral Hemorrhage (SWITCH): a Randomized Controlled Trial

acronym: SWITCH

summary:
Spontaneous intracerebral hemorrhage (ICH) remains a devastating disease with mortality rates up to 52% at 30 days. It is a major public health problem with an annual incidence of 10-30 per 100'000 population, accounting for 2 million (10-15%) of about 15 million strokes worldwide each year. The strategy of decompressive craniectomy (DC) is beneficial in patients with malignant middle cerebral artery (MCA) infarction. Based on the common pathophysiological mechanisms of these two conditions, this procedure is also frequently performed in patients with ICH, but is has not yet been investigated in a randomized trial.

The primary objective of this randomized controlled trial is to determine whether decompressive surgery and best medical treatment in patients with spontaneous ICH will improve outcome compared to best medical treatment only.

Secondary objectives are to analyze mortality, dependency and quality of life. Safety endpoints are to determine cause of any mortality and the rate of medical and surgical complications after DC compared with best medical treatment alone.

lead_sponsor: Insel Gruppe AG, University Hospital Bern

location_country: Finland Germany Netherlands Austria Belgium Switzerland Spain France

location_facility: Klinik für Neurochirurgie, Universitätsmedizin Göttingen

location_facility: Servicios de Neurología, Neurocirugía y Cuidados Intensivos del Hospital Virgen del Rocío

location_facility: Servicio de Neurología, Hospital Universitario La Paz

location_facility: Dep. of Neurology / Dep. of Neurosurgery, Bern University Hospital

location_facility: Dep. Neurosurgery, Ospedale Regionale di Lugano

location_facility: Neurochirurgische Klinik, Universitätsklinikum Gießen und Marburg UKGM

location_facility: Neurochirurgische Klinik, Universitätsmedizin Mainz

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Freiburg

location_facility: Centre Hospitalier Universitaire de Caen

location_facility: Academic Medical Center Amsterdam, Department of Neurology

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Düsseldorf

location_facility: Klinik für Neurochirurgie, Klinikum Kassel

location_facility: Dep. of Neurosurgery, University Hospital Zürich

location_facility: Universitätsklinik für Neurochirurgie, Kepler Universitätsklinikum Linz

location_facility: Dep. of Neurosurgery, Klinikum rechts der Isar der Technischen Universität München

location_facility: University Medical Center Utrecht, Department of Neurology, Department of Neurosurgery

location_facility: Klinik und Poliklinik für Neurochirurgie, Universitätsklinikum Bonn

location_facility: Zentrum der Neurologie und Neurochirurgie, Universitätsklinikum Frankfurt

location_facility: Neurochirurgische Klinik, Universitätsklinikum Mannheim

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Würzburg

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Schleswig Holstein

location_facility: Dep. of Clinical Neuroscience, Service of Neurosurgery

location_facility: Department of Neurology, Helsinki University Central Hospital

location_facility: Klinik für Allgemeine Neurologie, Universitätsklinikum Münster

location_facility: Klinik für Neurochirurgie Uniklinik RWTH Aachen

location_facility: Department of Neurosurgery, Charité - Universitätsmedizin Berlin

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Essen (AöR)

location_facility: Neurologische Klinik, Universitätsklinikum Erlangen

location_facility: Klinik für Neurochirugie, Helios Klinikum Erfurt

location_facility: Servicio de Neurocirurgía Bellvitge Hospital

intervention_type: Procedure


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: Meridian Bioscience, Inc.

location_country:
United States
Spain
Switzerland
France

location_facility: (University of Barcelona) Hospital Clinic


title: Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)

acronym: EFACTS

summary:
This is a multi-centre, multi-national, prospective, observational study of Friedreich's Ataxia (FRDA) with a control group to:

obtain natural history data on individuals affected by FRDA
relate clinical assessments and results from proteomic analyses
expedite identification and recruitment of participants for clinical trials
develop and validate sensitive and reliable outcome measures for detecting onset and change over the natural course of FRDA which may also be potential outcome measures for use in future clinical trials and clinical care
plan for future research studies

status: Recruiting

time_perspective: Prospective

ReferenceCitation: Reetz K, Dogan I, Costa AS, Dafotakis M, Fedosov K, Giunti P, Parkinson MH, Sweeney MG, Mariotti C, Panzeri M, Nanetti L, Arpa J, Sanz-Gallego I, Durr A, Charles P, Boesch S, Nachbauer W, Klopstock T, Karin I, Depondt C, vom Hagen JM, Schols L, Giordano IA, Klockgether T, Burk K, Pandolfo M, Schulz JB. Biological and clinical characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) cohort: a cross-sectional analysis of baseline data. Lancet Neurol. 2015 Feb;14(2):174-82. doi: 10.1016/S1474-4422(14)70321-7. Epub 2015 Jan 5.

ReferenceCitation: Reetz K, Dogan I, Hilgers RD, Giunti P, Parkinson MH, Mariotti C, Nanetti L, Durr A, Ewenczyk C, Boesch S, Nachbauer W, Klopstock T, Stendel C, Rodriguez de Rivera Garrido FJ, Rummey C, Schols L, Hayer SN, Klockgether T, Giordano I, Didszun C, Rai M, Pandolfo M, Schulz JB; EFACTS study group. Progression characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS): a 4-year cohort study. Lancet Neurol. 2021 May;20(5):362-372. doi: 10.1016/S1474-4422(21)00027-2. Epub 2021 Mar 23.

oa_host_type: repository

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

repositories_concat: UCL Discovery

unpaywall_oa_status: green

ReferenceCitation: Indelicato E, Nachbauer W, Eigentler A, Amprosi M, Matteucci Gothe R, Giunti P, Mariotti C, Arpa J, Durr A, Klopstock T, Schols L, Giordano I, Burk K, Pandolfo M, Didszdun C, Schulz JB, Boesch S; EFACTS (European Friedreich's Ataxia Consortium for Translational Studies). Onset features and time to diagnosis in Friedreich's Ataxia. Orphanet J Rare Dis. 2020 Aug 3;15(1):198. doi: 10.1186/s13023-020-01475-9.

repositories:
HAL
PubMed Central
Research Square
UCL Discovery

repositories_concat: HAL;PubMed Central;Research Square;UCL Discovery

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

ReferenceCitation: Bonnechere B, Jansen B, Haack I, Omelina L, Feipel V, Van Sint Jan S, Pandolfo M. Automated functional upper limb evaluation of patients with Friedreich ataxia using serious games rehabilitation exercises. J Neuroeng Rehabil. 2018 Oct 4;15(1):87. doi: 10.1186/s12984-018-0430-7.

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

ReferenceCitation: Reetz K, Dogan I, Hohenfeld C, Didszun C, Giunti P, Mariotti C, Durr A, Boesch S, Klopstock T, Rodriguez de Rivera Garrido FJ, Schols L, Giordano I, Burk K, Pandolfo M, Schulz JB; EFACTS Study Group. Nonataxia symptoms in Friedreich Ataxia: Report from the Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS). Neurology. 2018 Sep 4;91(10):e917-e930. doi: 10.1212/WNL.0000000000006121. Epub 2018 Aug 10.

repository_institution: Ludwid Maxmilian's Universitat Munchen - Open access LMU

ReferenceCitation: Tanguy Melac A, Mariotti C, Filipovic Pierucci A, Giunti P, Arpa J, Boesch S, Klopstock T, Muller Vom Hagen J, Klockgether T, Burk K, Schulz JB, Reetz K, Pandolfo M, Durr A, Tezenas du Montcel S; EFACTS group. Friedreich and dominant ataxias: quantitative differences in cerebellar dysfunction measurements. J Neurol Neurosurg Psychiatry. 2018 Jun;89(6):559-565. doi: 10.1136/jnnp-2017-316964. Epub 2017 Dec 26.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université de Nantes

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

ReferenceCitation: Lad M, Parkinson MH, Rai M, Pandolfo M, Bogdanova-Mihaylova P, Walsh RA, Murphy S, Emmanuel A, Panicker J, Giunti P. Urinary, bowel and sexual symptoms in a cohort of patients with Friedreich's ataxia. Orphanet J Rare Dis. 2017 Sep 26;12(1):158. doi: 10.1186/s13023-017-0709-y.

repository_institution: CERN European Organization for Nuclear Research - Zenodo

ReferenceCitation: Reetz K, Dogan I, Hilgers RD, Giunti P, Mariotti C, Durr A, Boesch S, Klopstock T, de Rivera FJR, Schols L, Klockgether T, Burk K, Rai M, Pandolfo M, Schulz JB; EFACTS Study Group. Progression characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS): a 2 year cohort study. Lancet Neurol. 2016 Dec;15(13):1346-1354. doi: 10.1016/S1474-4422(16)30287-3. Erratum In: Lancet Neurol. 2017 Dec;16(12 ):954.

location_country:
Germany
Ireland
Italy
United Kingdom
Austria
Belgium
Spain
Greece
Czechia
France

location_facility: University Hospital Aachen, Dept. of Neurology

location_facility: Hospital Sant Joan de Déu, Servicio de Neurología

location_facility: Medical University Innsbruck, Department of Neurology

location_facility: University College of London, Ataxia Centre, National Hospital for Neurology and Neurosurgery

location_facility: Tallaght University Hospital, Department of Neurology

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta

location_facility: National and Kapodistrian University of Athens, Neurogenetics Unit

location_facility: University Hospital Bonn, Dept. of Neurology

location_facility: University of Tübingen, Dept. of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research

location_facility: Bambino Gesù Children's Hospital, Department of Neurosciences

location_facility: Hôpital de Hautepierre, Service de Neurologie

location_facility: Motol University Hospital, Centre for Hereditary Ataxias

location_facility: Hospital Universitario La Paz, Servicio de Neurologia

location_facility: Université Libre de Bruxelles, Hôpital Erasme, Dpt of Neurology

location_facility: University of Munich, Dept. of Neurology, Friedrich-Baur-Institut


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.

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. doi: 10.1042/BJ20040156.

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. doi: 10.1152/ajpheart.00307.2003. 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, Ekstrom 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. doi: 10.1529/biophysj.107.114314. 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. doi: 10.1182/blood-2006-02-005272. 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. doi: 10.1016/s0301-4622(97)00079-3.

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.

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. doi: 10.1172/JCI26857. 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. doi: 10.1007/s00109-007-0276-0. Epub 2007 Nov 22.

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:
United Kingdom
Brazil
Netherlands
Lebanon
Bahrain
Belgium
Qatar
Turkey
France

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.

repositories_concat: HAL

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

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

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université de Nantes

lead_sponsor: University Hospital, Geneva

sponsor_collaborators:
University Hospital, Geneva
University Hospital, Clermont-Ferrand
Erasme University Hospital
Federal University of Pelotas
Clinical Hospital Centre Zagreb
University Hospital, Rouen
Rabin Medical Center
Vilnius University
Stanley Dudrick's Memorial Hospital
Clinica USP Palmaplanas

location_country: France Lithuania Poland Brazil Switzerland Belgium Spain Croatia Israel

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

lead_sponsor: Prof. Laszlo Czirjak

collaborators: University of Campania "Luigi Vanvitelli"

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

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

location_facility: Department of Internal Medicine Hôpital Claude Huriez

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

location_facility: Royal Free Hospital, University College London

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

location_facility: Institute of Rheumatology, Russian Academy of Medical Science

location_facility: University of Ghent, Department of Rheumatology

location_facility: University of Zurich, Department of Rheumatology

location_facility: Clinic of Nephrology, Internal and Occupational Diseases

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

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

location_facility: Centre for Pediatric Rheumatology, Klinikum Eilbek

location_facility: University of Marmara, Department of Rheumatology

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

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

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

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

location_facility: Hospital Universitario Madrid Norte Sanchinarro

location_facility: Pecsi Tudomanyegyetem - University of Pecs

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

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

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


title: Phase IIb Study to Assess the Efficacy, Safety and Tolerability of Two Dosing Regimens of AllerT in Comparison to Placebo in Adult Subjects Allergic to Birch Pollen

summary: The main objective of the trial is to demonstrate the efficacy of a two months pre-seasonal treatment with AllerT 100 µg maintenance dose in reducing symptoms of allergic rhinoconjunctivitis during the following birch pollen season

lead_sponsor: Anergis

location_country: Lithuania Poland Denmark Sweden Switzerland Latvia France

location_facility: Antakalnio affiliation of the Vilnius City Allergy Center

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Center of examination and treatment of allergic diseases

location_facility: Paul Stradins Clinical University Hospital - Pulmonology Allergology

location_facility: Viktorija Vevere private practice of Allergology

location_facility: Silesian Piasts University of Medicine in Wrocław

location_facility: SPZOZ Uniwersytecki Szpital Kliniczny - Allergology


title: An Open-label Extension of Study AC-066A301 Investigating the Safety and Tolerability of ACT-385781A in Patients With Pulmonary Arterial Hypertension (PAH)

summary: This is an open-label, nonrandomized extension to study AC-066A301. The study will assess safety and tolerability of ACT-385781A while providing a means for continuing treatment after ending participation in study AC-066A301.

lead_sponsor: Actelion

collaborators:
Chiltern International Ltd.
Effi-Stat

sponsor_collaborators:
Actelion
Chiltern International Ltd.
Effi-Stat

location_country: Italy Netherlands Belgium Spain Canada France

location_facility: Sir Mortimer B Davis Jewish General Hospital


summary: This study is investigating the effect of switching from Flolan® to Epoprostenol for Injection (EFI/ACT-385781A) in pulmonary arterial hypertension patients currently treated with Flolan®. For this purpose patients being treated for at least 12 months with Flolan® will be switched from Flolan® to EFI/ACT-385781A and followed-up for 90 days. During these 90 days safety and tolerability of EFI/ACT-385781A will closely be monitored in all treated patients. This 90 follow-up will provide clinical evidence on the safety, tolerability, efficacy and treatment satisfaction of switching from Flolan® to EFI/ACT-385781A in patients with pulmonary arterial hypertension.

location_facility: University Health Network, Toronto TGH - 10 EN - 220

title: A Multicenter, Single-arm, Open-label, Phase 3b Study to Assess the Effects of Switching From Flolan® to EFI/ACT-385781A in Patients With Pulmonary Arterial Hypertension


title: An Open-label, Phase I/IIa, Dose Escalating Study of 2B3-101 in Patients With Solid Tumors and Brain Metastases or Recurrent Malignant Glioma.

summary: The purpose of this study is to determine the safety, tolerability, and pharmacokinetics (PK) of 2B3-101 both as single agent and in combination with trastuzumab. Furthermore, the study will explore the preliminary antitumor activity of 2B3-101 as single agent in patients with with solid tumors and brain metastases or recurrent malignant glioma as well as in patients with various forms of breast cancer with and in combination with trastuzumab in HER2+ breast cancer patients with brain metastases.

lead_sponsor: BBB-Therapeutics B.V.

location_country:
United States
Netherlands
Belgium
France

location_facility: UNC Lineberger Comprehensive Cancer Center

location_facility: Vrije Universiteit medisch centrum (Vumc)

location_facility: Leids Universitair Medisch Centrum (LUMC)


title: REVOLUTION (WFCC-133): Clinical Workflow Study for the Evaluation of the Multi-Electrode Pulmonary Vein Isolation System for the Treatment of Paroxysmal Atrial Fibrillation (PAF)

acronym: REVOLUTION

summary: The purpose of this study is to assess the safety and effectiveness of the Circular and Crescent Mapping and Ablation catheters and the workflow of the Multi-Electrode Irrigated Pulmonary Vein Isolation System when used for the treatment of drug refractory symptomatic paroxysmal atrial fibrillation (PAF).

lead_sponsor: Biosense Webster, Inc.

location_country: Germany Italy Denmark Belgium Czechia France

location_facility: Institute for Clinical and Experimental Medicine (IKEM)


acronym: EARLY

summary: This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.

ReferenceCitation: Ratjen F, Moeller A, McKinney ML, Asherova I, Alon N, Maykut R, Angyalosi G; EARLY study group. Eradication of early P. aeruginosa infection in children <7 years of age with cystic fibrosis: The early study. J Cyst Fibros. 2019 Jan;18(1):78-85. doi: 10.1016/j.jcf.2018.04.002. Epub 2018 Apr 21.

license: publisher-specific-oa

license_normalized: publisher-specific

title: A Randomized, Double-Blind, Placebo-Controlled, Crossover Multi-Center Study to Assess the Efficacy and Safety of Inhaled Tobramycin Nebuliser Solution (TOBI®) for the Treatment of Early Infections of P. Aeruginosa in Cystic Fibrosis Subjects Aged From 3 Months to Less Than 7 Years

location_country:
Germany
Italy
Russian Federation
Hungary
Romania
Switzerland
Greece
Canada
Egypt
France


title: International, Multicenter, Prospective, Randomized, Parallel Group, Open Label Protocol to Evaluate Safety and Efficacy of Isolated Pancreas Transplantation Compared to Intensive Insulin Therapy in Type 1 Diabetic Patients With Overt Diabetic Nephropathy and Mildly Reduced Renal Function

acronym: PANCREAS

summary: Current medical therapies are not able to prevent progression of established macroproteinuira (i.e. diabetic nephropathy) to end-stage renal failure in type 1 (insulin dependent) diabetic patients. In this setting, proteinuria is a major risk factor for mortality. Pancreas transplantation, on the contrary, can revert diabetic nephropathy and thereby prevent end-stage chronic renal failure, with theoretically lower risk of death as compared to current medical therapies.The main objective of this study is to assess superiority of isolated pancreas transplantation versus intensive exogenous insulin therapy in type 1 diabetic patients with overt diabetic nephropathy and mildly reduced renal function. The primary endpoint is a composite efficacy/failure end-point including: patient mortality and renal function impairment during 5 years in patients with badly controlled diabetes and nephropathy resisting to up-to-date nephroprotective therapies.Main secondary objectives are safety and efficacy of both regimens, including proteinuria and renal histology evaluation, metabolic control and quality of life, acute and chronic extrarenal complications of diabetes, pancreas survival and all risks related to the transplant procedure (anaesthesia, surgery and immunosuppression side-effects) and to the intensive insulin therapy management.

lead_sponsor: Nantes University Hospital

location_country:
Italy
Brazil
United States
Czech Republic
France

location_facility: Diabetes Center - Institute for Clinical and Experimental Medicine

location_facility: Hôpital Edouard-Herriot - Hospices Civils de Lyon

location_facility: Centre Hospitalier et Universitaire de Nantes

location_facility: Istituto Scientifico Ospedale San Raffaele


title: The Cardiovascular Morbidity in Children With Chronic Renal Failure Study

summary:
Children and adolescents with chronic kidney disease (CKD) are at high risk for cardiovascular (CV) morbidity and mortality. Recent studies suggest that pediatric patients with even moderately impaired kidney function may be afflicted with significant early cardiac and vascular abnormalities.

The pathogenesis and the natural course of CV comorbidity in pediatric CKD patients is still elusive. In this multicenter, prospective, observational study the prevalence, degree and progression of CV comorbidity in children will be characterized and related to CKD progression. The morphology and function of the heart and vessels will be monitored by sensitive, non-invasive methods and will be compared with aged matched healthy controls. Multiple potential clinical, anthropometric, biochemical, and pharmacological risk factors will be monitored prospectively and will be related to CV status. Genotyping might identify predisposing genetic factors for progression of CV comorbidity and underlying nephropathies.

lead_sponsor: Heidelberg University

collaborators: German Federal Ministry of Education and Research

location_country:
Germany
Italy
Lithuania
Poland
Portugal
Hungary
United Kingdom
Sweden
Switzerland
Austria
Belgium
Turkey
Serbia
Czech Republic
France

location_facility: Haseki Educational and Research Hospital

location_facility: Center for Pediatrics and Adolescent Medicine

location_facility: Goztepe Educational and Research Hospital


title: A 2-Part Trial: a Randomized 6-day Repeat-dose, Parallel-group Study in Subjects With T2DM to Assess the Safety and Tolerability of GSK1614235 Compared to Placebo and Sitagliptin; and a Randomized Single-dose, Food Effect Study in Healthy Volunteers to Assess Safety and Tolerability of GSK1614235

summary:
The purpose of Part A of this study is to test whether repeated doses of the study drug (GSK1614235) are safe and well tolerated (i.e. do not produce unacceptable side effects) and whether we can obtain some preliminary information as to whether it works in lowering blood glucose levels. We will do this by comparing the effect of the study drug with placebo (no drug present) and against a drug (sitagliptin) known to control blood glucose in the treatment of diabetes.

The purpose of Part B of this study is to determine the how the timing of dosing, relative to meals, affects the response to study drug.

ReferenceCitation: Dobbins RL, Greenway FL, Chen L, Liu Y, Breed SL, Andrews SM, Wald JA, Walker A, Smith CD. Selective sodium-dependent glucose transporter 1 inhibitors block glucose absorption and impair glucose-dependent insulinotropic peptide release. Am J Physiol Gastrointest Liver Physiol. 2015 Jun 1;308(11):G946-54. doi: 10.1152/ajpgi.00286.2014. Epub 2015 Mar 12.

publisher_dissemination: American Physiological Society

lead_sponsor: GlaxoSmithKline

location_country:
United States
United Kingdom
Germany
France


summary: This is a phase I/II open-label study to delineate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of TKI258. The eligible subject population consists of subjects who have been diagnosed with advanced or metastatic renal cell cancer that is refractory to standard therapy or for which no curative standard therapy exists.

title: A Phase I/II Multi-center, Open Label Study of TKI258 Administered Orally on an Intermittent Schedule in Adult Patients With Advanced or Metastatic Renal Cell Cancer (RCC)

location_country:
Germany
Netherlands
United States
Spain
Taiwan
France


title: A Randomised, Controlled and Double-blind Clinical Investigation on the Effectiveness and Safety of a Foam Dressing Biatain Ibu Non-adhesive vs. Biatain Non-adhesive, in Painful Chronic Venous Leg Ulcers

summary: The objective of this investigation is to demonstrate the effectiveness and safety of Biatain Ibu non-adhesive foam dressing compared to Biatain non-adhesive foam dressing.

lead_sponsor: Coloplast A/S

location_country: Germany Denmark Spain France

location_facility: Fachartzpraxis für Dermatologie, Allergologie, Lasermedizin

location_facility: C.S. SERGAS Rosalía de Castro - Enfermero

location_facility: Department for Dermatology, University School of Medicine


title: Therapeutic Impact of Albumin Dialysis With the Molecular Adsorbents Recirculating System (MARS®) in Severely Decompensated Chronic Liver Disease

acronym: RELIEF

summary: The objective of this trial is to evaluate the impact of elimination of albumin bound substances during albumin dialysis (MARS®) on mortality and the clinical time course in patients with a recent severe clinical deterioration of chronic liver disease caused by a precipitating (trigger) event within 4 weeks manifested by jaundice, encephalopathy and/or renal failure.

ReferenceCitation: Stange J, Mitzner S, Ramlow W, Gliesche T, Hickstein H, Schmidt R. A new procedure for the removal of protein bound drugs and toxins. ASAIO J. 1993 Jul-Sep;39(3):M621-5.

ReferenceCitation: Stange J, Ramlow W, Mitzner S, Schmidt R, Klinkmann H. Dialysis against a recycled albumin solution enables the removal of albumin-bound toxins. Artif Organs. 1993 Sep;17(9):809-13. doi: 10.1111/j.1525-1594.1993.tb00635.x.

lead_sponsor: Baxter Healthcare Corporation

sponsor_collaborators:
Baxter Healthcare Corporation
2ConduCT
G.E.M. mbh Meerbusch
DatInf
Gambro Lundia AB

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

location_facility: Martin Luther Universität Halle-Wittenberg


title: A Phase I/II Open Label Dose Escalation Study to Investigate the Safety and Tolerability of Acadesine in Patients With B-cell Chronic Lymphocytic Leukemia

summary: The main aim of this study is to test the safety of acadesine in patients with B-CLL and see what effects it has on patients and their leukaemia. The study also aims to examine the way acadesine is processed by the body. The study will look at the effects of acadesine in the body and the concentration of the drug and its main by-product (ZMP) in the blood to determine the dose and the frequency of dosing that is likely to be the most effective.

lead_sponsor: Advancell - Advanced In Vitro Cell Technologies, S.A.

location_country: Spain Belgium France

location_facility: Service des maladies du sang Hôpital HURIEZ, CHRU

location_facility: Hematologia Clinica, Institut Catala d'Oncologia

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Cliniques universitaires Saint-Luc Haematology Dept


title: A Multicenter, Phase III, Randomised Study of Photodynamic Therapy With Metvix Cream 160 mg/g in Comparison With Cryotherapy in Patients With Primary Superficial Basal Cell Carcinoma

summary:
The purpose of this study is to compare the efficacy of Photodynamic Therapy (PDT) with methyl aminolevulinate (MAL) cream to cryotherapy, in treatment of patients with primary superficial basal cell carcinoma.

Secondary objectives are to compare cosmetic outcome and tolerability (adverse events) in these patients, 3 months after treatment. In addition the recurrence rates in the two treatment groups will be compared up to five years after treatment.

lead_sponsor: Galderma R&D

location_country:
Finland
Italy
United Kingdom
Sweden
Austria
Belgium
France

location_facility: Service de Dermatologie, C.H.U Saint Louis


title: An Open-labeled Trial With a Dose-escalation Part and a Parallel Group Design(1) Investigating Zalutumumab, an Anti-EGF Receptor Antibody, in Combination With Chemo-Radiation as First Line Treatment of Patients With Cancer of the Head and Neck (1) The Parallel Group Part Was Cancelled

summary: The purpose of this study is to investigate the safety of zalutumumab in combination with chemotherapy and radiotherapy as treatment of patients with head and neck cancer

lead_sponsor: Genmab

location_country:
France
Netherlands
Belgium
United States
Sweden

location_facility: Hopital Bretonneau Clinique d'Oncologie et Radiothérapie

location_facility: Centre Georges-Francois Leclerc Hospital


title: Profile of Depressive Symptoms in Parkinson's Disease (PRODEST-PD)

summary: To explore or establish the relationship between cognitive, mood and motor symptoms in PD to scores on depression rating scales in a naturalistic setting.

lead_sponsor: Boehringer Ingelheim

location_country:
Germany
Italy
United Kingdom
Netherlands
Austria
Switzerland
Spain
France

location_facility: Universitatsklinikum Carl Gustav Carus Dresden

location_facility: Boehringer Ingelheim Investigational Site


title: Enzymatic Debridement in Burns Patients (Children & Adults): A Comparison to Standard of Care (Protocol MW 2004-11-02)

summary:
Burns represent one of the most severe and dreaded traumas. Burned and traumatized tissue is known as eschar. The dead eschar, if not removed, often becomes heavily contaminated and is the source of local and/or systemic infection or sepsis. The local inflammation and infection destroy healthy surrounding tissues and extends the original damage. In order to prevent these complications, and in order to minimize the risk of infection, it is imperative to evaluate the burn and remove all of the offending eschar at the earliest possible opportunity. This removal of dead tissue is termed "debridement".

The most direct debridement method for eschar removal is surgery. Traditional, conservative non-surgical debridement is a lengthy process which often involves many complications.

The objective of this study is to evaluate the safety and enzymatic debriding efficacy of Debrase Gel Dressing (DGD) in hospitalized patients with deep partial thickness and/or full thickness thermal burns and to compare DGD to standard of care (SOC).

ReferenceCitation: Rosenberg L, Lapid O, Bogdanov-Berezovsky A, Glesinger R, Krieger Y, Silberstein E, Sagi A, Judkins K, Singer AJ. Safety and efficacy of a proteolytic enzyme for enzymatic burn debridement: a preliminary report. Burns. 2004 Dec;30(8):843-50. doi: 10.1016/j.burns.2004.04.010.

ReferenceCitation: Rosenberg L, Krieger Y, Bogdanov-Berezovski A, Silberstein E, Shoham Y, Singer AJ. A novel rapid and selective enzymatic debridement agent for burn wound management: a multi-center RCT. Burns. 2014 May;40(3):466-74. doi: 10.1016/j.burns.2013.08.013. Epub 2013 Sep 26.

lead_sponsor: MediWound Ltd

location_country:
Germany
France
Italy
Poland
Slovakia
United Kingdom
Romania
Australia
Brazil
Israel

location_facility: Centre Hospitalier Regional et Universitaire de Marseille, Service de Chirurgie Plastique Reparatrice et Esthetique

location_facility: Center for Burns & Reconstructive Surgery, University Hopsital Bratislava

location_facility: Direttore U.O. Chirurgia Plastica e Centro Ustioni Ospedale Civico

location_facility: Hospital do Servidor Publico do Estado de Sao Paulo

location_facility: Pronto Socorro para Queimaduras de Goiania

location_facility: Clinic of Burns and Reconstructive Surgery Hospital Kosice

location_facility: Emergency Clinic Hospital "Bagdazar-Arsenie"


type: Registry Identifier

title: A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Efficacy and Safety of TAK-475 or Placebo When Co-administered With Current Lipid-lowering Therapy in Subjects With Homozygous Familial Hypercholesterolemia.

summary: The purpose of the study is to determine the efficacy of lapaquistat acetate, once daily (QD), to lower cholesterol in subjects with homozygous familial hypercholesterolemia undergoing lipid-lowering treatment.

ReferenceCitation: Stein EA, Bays H, O'Brien D, Pedicano J, Piper E, Spezzi A. Lapaquistat acetate: development of a squalene synthase inhibitor for the treatment of hypercholesterolemia. Circulation. 2011 May 10;123(18):1974-85. doi: 10.1161/CIRCULATIONAHA.110.975284. Epub 2011 Apr 25.

lead_sponsor: Takeda

location_country:
France
Poland
United Kingdom
United States
Canada
Israel


title: A Comparison of the Inflammatory Control of Asthma Provided by One Inhalation of Symbicort® Turbuhaler® 160/4.5 µg/Inhalation b.i.d. Plus As-needed Versus One Inhalation of Symbicort® Turbuhaler® 320/9 µg/Inhalation b.i.d. + One Inhalation of Pulmicort® Turbuhaler® 400 µg/Dose b.i.d. Plus Terbutaline Turbuhaler® 0.4 mg/Inhalation As-needed. A 12-month, Randomised, Double-blind, Parallel-group, Active Controlled, Multinational, Phase IIIB Study in Adult Patients With Asthma, EOS

summary: The purpose of this study is to determine if a flexible dose of Symbicort has a similar effect on the airways of patients with asthma as a higher fixed-dose of Symbicort with additional glucocorticosteroids added.

lead_sponsor: AstraZeneca

location_country:
Germany
France
United Kingdom
Denmark
Spain
Sweden


title: A Phase IIIb, Randomized, Open Label Study to Compare the Safety, Efficacy and Tolerability of Anagrelide Hydrochloride Versus Hydroxyurea in High-Risk Essential Thrombocythaemia Patients.

summary: Essential thrombocythaemia is a disorder of bone marrow, which causes too many platelets to be produced. Platelets are small cells carried around in the blood, which help form blood clots. When patients have too many platelets, there is a risk of blood clots forming unnecessarily and excessive bleeding. The aim of this study is to gain additional information on the safety profile of Anagrelide (Xagrid(r)) and Hydroxyurea (also known as hydroxycarbamide).

ReferenceCitation: Birgegard G, Folkvaljon F, Garmo H, Holmberg L, Besses C, Griesshammer M, Gugliotta L, Wu J, Achenbach H, Kiladjian JJ, Harrison CN. Leukemic transformation and second cancers in 3649 patients with high-risk essential thrombocythemia in the EXELS study. Leuk Res. 2018 Nov;74:105-109. doi: 10.1016/j.leukres.2018.10.006. Epub 2018 Oct 11.

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repository_institution: Universitat Pompeu Fabra - Repositori digital de la UPF

version: acceptedVersion

repository_institution: King's College London - Research Portal

ReferenceCitation: Gotic M, Egyed M, Gercheva L, Warzocha K, Kvasnicka HM, Achenbach H, Wu J. Cardiovascular Safety of Anagrelide Hydrochloride versus Hydroxyurea in Essential Thrombocythaemia. Cardiovasc Toxicol. 2021 Mar;21(3):236-247. doi: 10.1007/s12012-020-09615-0. Epub 2020 Oct 29.

lead_sponsor: Shire

location_country: Poland Portugal Hungary Bulgaria Serbia France

location_facility: Uniwersyteckie Centrum Kliniczne Katedra i Klinika Hematologii i Transplantologii

location_facility: Institute for Haematology of Clinical Centre of Serbia

location_facility: Hopital Saint Louis - Centre d'Investigation Clinique

location_facility: CHU Angers Services des Maladies du Sang

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 1

location_facility: University of Debrecen Medical and Health Science Centre

location_facility: Klinika Hematologii Instytut Hematologii i Transfuzjologi

location_facility: University Multiprofile Hospital for Active Treatment ''Dr Georgi Stranski'' - Pleven

location_facility: University Multiprofile Hospital for active Treatment ''Alexandrovska'' Clinic of Haematology

location_facility: Katedra i Klinika Onkologii i Chorob Wewnetrznych Akademii Medycznej

location_facility: University Multiprofile Hospital for Active Treament ''Sv. Marina'' - Varna Haematology Clinic


title: Non-Randomized, Prospective, Multi-center Evaluation of the ABSOLUTE™ .035 Peripheral Self-Expanding Stent System for Occluded or Stenotic Superficial Femoral or Proximal Popliteal Arteries

summary: The purpose of this study is to investigate the performance of the ABSOLUTE™ .035 peripheral self-expanding stent system in preventing restenosis of occluded or stenotic superficial femoral or proximal popliteal arteries.

lead_sponsor: Abbott Medical Devices

location_country: Germany Italy Austria Belgium Spain Greece France

location_facility: Allgemeines Krankenhaus der Stadt Wien (AKH Wien)

location_facility: Universitäres Herz & Gefässzentrum Hamburg


title: An EORTC Randomized, Double Blind, Placebo-Controlled, Phase II Multi-Center Trial Of Anastrozole (Arimidex) In Combination With ZD 1839 (Iressa) Or Placebo In Patients With Advanced Breast Cancer

summary:
RATIONALE: Estrogen can stimulate the growth of breast cancer cells. Hormone therapy using anastrozole may fight breast cancer by reducing the production of estrogen. Gefitinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Combining anastrozole with gefitinib may kill more tumor cells.

PURPOSE: Randomized phase II trial to compare the effectiveness of anastrozole with or without gefitinib in treating postmenopausal women who have metastatic or locally recurrent breast cancer.

ReferenceCitation: Mauriac L, Cameron D, Dirix L: Results of randomized phase II trial combining Iressa (gefitinib) and arimidex in women with advanced breast cancer (ABC): EORTC protocol 10021. [Abstract] 31st Annual San Antonio Breast Cancer Symposium, December 10-14, 2008, San Antonio, Texas. A-6133, 2008.

ReferenceCitation: Tryfonidis K, Basaran G, Bogaerts J, Debled M, Dirix L, Thery JC, Tjan-Heijnen VC, Van den Weyngaert D, Cufer T, Piccart M, Cameron D; EORTC-Breast Cancer Group. A European Organisation for Research and Treatment of Cancer randomized, double-blind, placebo-controlled, multicentre phase II trial of anastrozole in combination with gefitinib or placebo in hormone receptor-positive advanced breast cancer (NCT00066378). Eur J Cancer. 2016 Jan;53:144-54. doi: 10.1016/j.ejca.2015.10.012. Epub 2015 Dec 24.

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

location_country:
Slovenia
United Kingdom
Netherlands
Belgium
France

location_facility: Netherlands Cancer Institute - Antoni van Leeuwenhoek Hospital

location_facility: Edinburgh Cancer Centre at Western General Hospital

location_facility: Universitair Medisch Centrum St. Radboud - Nijmegen


title: Oral Empirical Therapy of Fever in Low-Risk Neutropenic Cancer Patients: A Prospective, Double-Blind, Randomized, Multicenter Trial Comparing Monotherapy (Single Daily Dose Moxifloxacin) With Combination Therapy (Ciprofloxacin Plus Amoxicillin/Clavulanic Acid)

summary:
RATIONALE: Antibiotics such as amoxicillin, ciprofloxacin, and moxifloxacin may be effective in preventing or controlling fever and neutropenia in patients with cancer. It is not yet known whether moxifloxacin alone is more effective than amoxicillin combined with ciprofloxacin in treating neutropenia and fever.

PURPOSE: This randomized clinical trial is studying how well moxifloxacin works and compares it to ciprofloxacin together with amoxicillin in treating neutropenia and fever in patients with cancer.

primary_purpose: Supportive Care

ReferenceCitation: Kern WV, Marchetti O, Drgona L, Akan H, Aoun M, Akova M, de Bock R, Paesmans M, Viscoli C, Calandra T. Oral antibiotics for fever in low-risk neutropenic patients with cancer: a double-blind, randomized, multicenter trial comparing single daily moxifloxacin with twice daily ciprofloxacin plus amoxicillin/clavulanic acid combination therapy--EORTC infectious diseases group trial XV. J Clin Oncol. 2013 Mar 20;31(9):1149-56. doi: 10.1200/JCO.2012.45.8109. Epub 2013 Jan 28.

publisher_dissemination: American Society of Clinical Oncology (ASCO)

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

location_country: Germany Italy France Slovakia Switzerland Belgium Turkey Israel

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Hacettepe University - Faculty of Medicine

location_facility: Medizinische Universitaetsklinik I at the University of Cologne

location_facility: Charite - Universitaetsmedizin Berlin - Campus Benjamin Franklin

location_facility: Istituto Nazionale per la Ricerca sul Cancro

location_facility: Frauenklinik - Universitaetsklinikum Rostock am Klinikum Sudstadt

location_facility: Klinikum der Albert - Ludwigs - Universitaet Freiburg

location_facility: Ruprecht - Karls - Universitaet Heidelberg


title: A Study of Two Different Doses of ABT-378/Ritonavir in HIV-Infected Patients Who Have Taken Protease Inhibitors and Non-Nucleoside Reverse Transcriptase Inhibitors

summary: The purpose of this study is to determine the safety and effectiveness of a high dose of ABT-378/ritonavir

lead_sponsor: Abbott

location_country:
United States
Spain
France

location_facility: The University of North Carolina at Chapel Hill


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, Kohne CH, Hohenberger P, Labianca R, Schmoll HJ, Pahlman 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. doi: 10.1093/jnci/djm024. Epub 2007 Jun 27.

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.

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.

ReferenceCitation: Roth AD, Tejpar S, Yan P, et al.: Correlation of molecular markers in colon cancer with stage-specific prognosis: results of the translational study on the PETACC 3 - EORTC 40993-SAKK 60-00 trial. [Abstract] American Society of Clinical Oncology 2009 Gastrointestinal Cancers Symposium, 15-17 January 2009, San Francisco, CA. A-288, 2009.

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, Andre 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.

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, Andre 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.

repository_institution: Université de Lausanne - SERVAL

lead_sponsor: Pfizer

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

location_facility: Instituto Portugues de Oncologia do Porto

location_facility: Hopital Cantonal Universitaire de Geneva

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


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.

location_country:
Italy
United Kingdom
Belgium
Spain
Turkey
France

location_facility: Istanbul University-Institute of Oncology

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


title: A Modular Phase I/II, Open-label, Multicentre Study to Evaluate the Safety, Tolerability, and Efficacy of AZD3470, a PRMT5 Inhibitor, as Monotherapy and in Combination With Anticancer Agent(s) in Participants With Relapsed/Refractory Haematologic Malignancies

acronym: PRIMAVERA

summary: This study is designed to evaluate the safety, tolerability, PK and preliminary efficacy following oral administration of AZD3470 as a monotherapy, and in combination with other anticancer agents in participants with haematologic malignancies.

status: Not yet recruiting

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

https://astrazenecagrouptrials.pharmacm.com /ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

location_country:
Italy
Australia
United States
Korea, Republic of
France


title: A Global, Open-label Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Hemophilia B With Inhibitors

summary: The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational program.

lead_sponsor: ApcinteX Ltd

sponsor_collaborators:
ApcinteX Ltd
Centessa Pharmaceuticals plc

location_country:
Australia
United States
Spain
Armenia
Taiwan
France

location_facility: Centre of Haematology named after prof. R. O. Yeolian/ Hemophilia center

location_facility: University of Colorado School of Medicine


title: A Randomized, Open-label, Multicenter, Multi-arm, Phase 1b/2 Platform Study to Evaluate Safety and Efficacy of Investigational Immunotherapies in Participants With Previously Treated Unresectable or Metastatic Melanoma

summary: This is a platform study evaluating the safety and efficacy of multiple novel investigational products (IPs) that target unresectable or metastatic cutaneous melanoma in participants who have failed standard treatment.

lead_sponsor: Innovent Biologics (Suzhou) Co. Ltd.

sponsor_collaborators:
Innovent Biologics (Suzhou) Co. Ltd.
Innovent Biologics (USA), Inc.

location_country:
Germany
United Kingdom
Australia
Switzerland
United States
Spain
France

location_facility: Cambridge University Hospitals NHS Foundation Trust (Oxford)

location_facility: University of California San Francisco Medical Center

location_facility: Hospices Civil De Lyon Nord - Centre Hospitalier Lyon Sud - Dermatologie

location_facility: Hospital Universitario Reina Sofia, Cordoba

location_facility: Institut Catala d'Oncologia Hospital Universitari Germans Trials I Pujol, Barcelona

intervention_type: Combination Product


title: A Randomized, Open-Label, Phase 2 Study Evaluating Abemaciclib in Combination With Irinotecan and Temozolomide in Participants With Relapsed or Refractory Ewing's Sarcoma

summary: The purpose of this study is to measure the benefit of adding abemaciclib to chemotherapy (irinotecan and temozolamide) for Ewing's sarcoma that has come back or did not respond to treatment. This trial is part of the CAMPFIRE master protocol, which is a platform to speed development of new treatments for children and young adults with cancer. Your participation in this trial could last 11 months or longer, depending on how you and your tumor respond.

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

lead_sponsor: Eli Lilly and Company

location_country:
Australia
Japan
United States
Spain
France

location_facility: Riley Hospital for Children at Indiana University Health

location_facility: Institut Bergonié - Centre Régional de Lutte Contre Le Cancer de Bordeaux et Sud Ouest

location_facility: Hyogo Prefectural Kobe Children's Hospital


title: KontRASt-03: A Phase Ib/II, Multicenter, Open-label Platform Study of JDQ443 With Select Combinations in Patients With Advanced Solid Tumors Harboring the KRAS G12C Mutation

summary: This is Phase Ib/II, multicenter, open-label adaptive platform study of JDQ443 with select therapies in patients with advanced solid tumors harboring the KRAS G12C mutation.

ipd_sharing: Undecided

lead_sponsor: Novartis Pharmaceuticals

location_country:
Germany
Italy
Singapore
Belgium
United States
Korea, Republic of
Spain
France

location_facility: Dana Farber Cancer Institute Dept.of DFCI


title: Skeletal Muscle-derived Cell Implantation for the Treatment of Fecal Incontinence: a Phase III, Randomized, Controlled, Double Blind, Two Armed Clinical Study

acronym: Fidelia

summary: The objective of this study is the final assessment of clinical safety and efficacy of autologous autologous skeletal muscle derived cells for patients with urge fecal incontinence due to external anal sphincter dysfunction caused by its disruption and/or weakness.

lead_sponsor: Innovacell AG

location_country:
Germany
United Kingdom
Japan
Sweden
Austria
Spain
Czechia
Bulgaria
France

location_facility: The Institute for the Care of Mother and Child

location_facility: Hosp. General Universitario Morales Meseguer

location_facility: Heidelberg University, Medical Faculty Mannheim, Department of surgery


title: Phase 1b, Multi-center, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, Tolerability, PD and PK of VK0214, in Subjects With the Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

summary: Subjects will be administered multiple doses of VK0214 in a cohort dose escalation process. A total of up to 36 subjects will be enrolled in the study, into one of three dose cohorts. For each cohort, up to 12 subjects will be randomized to receive VK0214 or placebo in a 3:1 ratio so that there will be a total of up to 9 subjects for each of the active doses and up to 3 subjects dosed with placebo in each cohort.

lead_sponsor: Viking Therapeutics, Inc.

location_country:
Germany
Italy
United Kingdom
United States
France

location_facility:
Viking Clinical Site 201
Viking Clinical Site 207
Viking Clinical Site 203
Viking Clinical Site 209
Viking Clinical Site 212
Viking Clinical Site 205
Viking Clinical Site 210
Viking Clinical Site 215
Viking Clinical Site 214