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National clinical trial(s)

WIEN
ADDRESS: NOT PROVIDED - AT
An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease (Phase 3) - AT
Institution: Information not provided - AT

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

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

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

Capital City Prague
PRAHA
FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naďve, 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

NORMANDIE
ROUEN

MyFABT1 : Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®)
CHU de Rouen
Service d'Imagerie médicale Charles-Nicolle

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

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

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

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, locatie AMC
Afdeling Erfelijke Stofwisselingsziekten

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, locatie AMC
Afdeling Erfelijke Stofwisselingsziekten

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, locatie AMC
Afdeling Erfelijke Stofwisselingsziekten

Noord-Holland
AMSTERDAM
FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naďve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - NL
Amsterdam UMC, locatie AMC
Polikliniek Kindermetabole Ziekten

Vestlandet
BERGEN
FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naďve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - NO
Haukeland University Hospital
Paediatric Department

Warszawa
WARSAW

FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naďve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - PL
Instytut "Pomnik-Centrum Zdrowia Dziecka"
Klinika Pediatrii, Zywienia i Chorob Metabolicznych

Aragón
ZARAGOZA
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 - ES
Hospital Quirónsalud Zaragoza
Servicio de Hematología

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

Madrid
ADDRESS: NOT PROVIDED - ES
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 - ES
Institution: Information not provided - ES

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

Madrid
ADDRESS: NOT PROVIDED - ES


A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naďve Subjects with Fabry Disease - ES
Institution: Information not provided - ES

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

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

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
Royal Free London Hospital
Royal Free London

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

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

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

Greater Manchester
MANCHESTER

The efficacy and mechanism of trientine in patients with hypertrophic cardiomyopathy
Wythenshawe Hospital
Cardiology
Multinational clinical trial(s)

ISRAEL
CARMIEL

ISRAEL
CARMIEL

Suisse Alémanique
ALLSCHWIL
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
Idorsia Pharmaceuticals Ltd

New Jersey
CRANBURY
A 24-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease (Phase II) (coordination)
Amicus Therapeutics Inc.

New Jersey
CRANBURY
A 12-Week Safety and Pharmacodynamic Study of AT1001 in Female Patients With Fabry Disease (Phase II) (coordination)
Amicus Therapeutics Inc.

New Jersey
CRANBURY