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Essai(s) clinique(s) national(aux)
ALLEMAGNE
Bayern
WÜRZBURG
MARVEL1: A Phase 1/2, Baseline-controlled, Non-randomised, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease -DE
Zentrum Innere Medizin (ZIM)
Medizinische Klinik und Poliklinik I - Fabry-Zentrum Würzburg
ALLEMAGNE
Bayern
WÜRZBURG
A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease (Phase 3) -DE-
Zentrum Innere Medizin (ZIM)
Medizinische Klinik und Poliklinik I - Nephrologie
ALLEMAGNE
Berlin
BERLIN
A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease (Phase 3) -DE-
Charité - Universitätsmedizin Berlin (CVK)
Medizinische Klinik mit Schwerpunkt Nephrologie und Internistische Intensivmedizin
AUTRICHE
WIEN
WIEN
A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease (Phase 3) - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Nephrologie und Dialyse
AUTRICHE
WIEN
WIEN
MODIFY: A Multicenter, dOuble-blind, ranDomized, Placebo-controlled, Parallel-group Study to Determine the effIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With FabrY Disease (Phase 3) - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Nephrologie und Dialyse
CANADA
Nouvelle-Écosse
HALIFAX
Enzyme Replacement Therapy for Fabry Disease: A Model for the Integration of Rare Disease Therapeutics Into the Canadian Health Care System (Phase IV)
Dalhousie University
Department of Medicine, Division of Nephrology
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease-CA
Institution: Information not provided - CA
ESPAGNE
Aragón
ZARAGOZA
Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease -ES
Hospital Quirónsalud Zaragoza
Servicio de Hematología
ESPAGNE
Madrid
ADDRESS: NOT PROVIDED - ES
A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease -ES
Institution: Information not provided - ES
ESPAGNE
Madrid
ADDRESS: NOT PROVIDED - ES
A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease -ES
Institution: Information not provided - ES
ESPAGNE
Madrid
ADDRESS: NOT PROVIDED - ES
A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy -ES
Institution: Information not provided - ES
FRANCE
NORMANDIE
ROUEN
MyFABT1 : Intérêt du suivi par IRM de patients atteints de la maladie de Fabry : impact du traitement par Agalsidase Alpha (Replagal®)
CHU de Rouen
Service d'Imagerie médicale Charles-Nicolle
IRLANDE
County Dublin
ADDRESS: NOT PROVIDED - IE
A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease-IE
Institution: Information not provided - IE
IRLANDE
County Dublin
ADDRESS: NOT PROVIDED - IE
A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.-IE
Institution: Information not provided - IE
NORVEGE
Vestlandet
BERGEN
FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - NO
Haukeland University Hospital
Paediatric Department
PAYS-BAS
Noord-Holland
AMSTERDAM
BALANCE: A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta (Phase III) - NL
Amsterdam UMC
Afdeling Erfelijke Stofwisselingsziekten
PAYS-BAS
Noord-Holland
AMSTERDAM
An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) (Phase III) - NL
Amsterdam UMC
Afdeling Erfelijke Stofwisselingsziekten
PAYS-BAS
Noord-Holland
AMSTERDAM
MODIFY: A Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel-group Study to Determine the EffIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (Phase III) - NL
Amsterdam UMC
Afdeling Erfelijke Stofwisselingsziekten
PAYS-BAS
Noord-Holland
AMSTERDAM
CARAT: A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy - NL
Amsterdam UMC
Afdeling Erfelijke Stofwisselingsziekten
PAYS-BAS
Noord-Holland
AMSTERDAM
Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease - NL
Amsterdam UMC
Afdeling Erfelijke Stofwisselingsziekten
PAYS-BAS
Noord-Holland
AMSTERDAM
FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - NL
Amsterdam UMC, locatie AMC
Polikliniek Kindermetabole Ziekten
POLOGNE
Warszawa
WARSAW
FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - PL
Instytut "Pomnik-Centrum Zdrowia Dziecka"
Klinika Pediatrii, Zywienia i Chorob Metabolicznych
ROYAUME-UNI
Greater London
LONDON
A Multicentre, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease - GB
Royal Free London Hospital
Royal Free London
ROYAUME-UNI
Greater Manchester
SALFORD
A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants (Phase IV)
Salford Royal NHS Foundation Trust
ROYAUME-UNI
South Glamorgan
CARDIFF
A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease-GB
University Hospital of Wales
Division of Psychological Medicine and Clinical Neurosciences
ROYAUME-UNI
West Midlands
BIRMINGHAM
A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease-GB
Queen Elizabeth Hospital
SUISSE
Suisse Alémanique
ZÜRICH
Effect of Cannabinoids on Pain in Fabry Disease Patients; a Prospective, Randomized, Double-blind, Placebo-controlled, Crossover, Multicenter Study
Universitätsspital Zürich
Klinik für Endokrinologie, Diabetologie und Klinische Ernährung
TCHEQUE, REPUBLIQUE
Capital City Prague
PRAHA
FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - CZ
Vseobecna fakultni nemocnice a 1. lekarska fakulta UK
General Teaching Hospital and 1st Faculty of Medicine
ROYAUME-UNI
Greater Manchester
MANCHESTER
The efficacy and mechanism of trientine in patients with hypertrophic cardiomyopathy
Wythenshawe Hospital
Cardiology
Essai(s) clinique(s) multinational(aux)
ETATS-UNIS
New Jersey
CRANBURY
A 24-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease (Phase II) (coordination)
Amicus Therapeutics Inc.
ETATS-UNIS
New Jersey
CRANBURY
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations (Phase III) (coordination)
Amicus Therapeutics Inc.
ETATS-UNIS
Washington
ADDRESS: NOT PROVIDED - US
An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease (phase III)
Institution: Information not provided - US
ETATS-UNIS
Washington
ADDRESS: NOT PROVIDED - US
ATTRACT: A randomized, open-label study to compare the efficacy and safety of AT1001 and Enzyme Replacement Therapy (ERT) in patients with Fabry disease and AT1001-responsive GLA mutations (Phase III)
Institution: Information not provided - US
ETATS-UNIS
Washington
ADDRESS: NOT PROVIDED - US
An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed With Fabry Disease
Institution: Information not provided - US
ISRAEL
ISRAEL
CARMIEL
ISRAEL
ISRAEL
CARMIEL
PAYS-BAS
Utrecht
ADDRESS: NOT PROVIDED - NL
A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Weeks to Adult Fabry Patients
Institution: Information not provided - NL
PAYS-BAS
Utrecht
ADDRESS: NOT PROVIDED - NL
An Extension of Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 38 Weeks to Adult Fabry Patients (Phase I/II)
Institution: Information not provided - NL
ROYAUME-UNI
Greater London
ADDRESS: NOT PROVIDED - GB
An Open-Label Extension Study to Evaluate the LongTerm Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease (Phase III) (Coordination)
Institution: Information not provided - GB
ROYAUME-UNI
Greater London
ADDRESS: NOT PROVIDED - GB
A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease
Institution: Information not provided - GB
SUISSE
Suisse Alémanique
ALLSCHWIL