Fabry Disease Clinical Trials
A listing of Fabry Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 30 clinical trials
Rare disease registries for certain lysosomal storage disorders, including, but not limited to, Fabry, Gaucher, MPS I, and Pompe diseases (each, a Registry)
Rare disease registries for certain lysosomal storage disorders, including, but not limited to, Fabry, Gaucher, MPS I, and Pompe diseases (each, a Registry)
- 592 views
- 23 Nov, 2020
- 1 location
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (CARAT)
alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy.
- 2 views
- 24 Aug, 2023
- 22 locations
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease (PERIDOT)
disease in adult participants with Fabry disease who are treatment-naïve or untreated for at least 6 months. Study visits will take place approximately every 3 months. The double
- 0 views
- 28 Aug, 2023
A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease
ascending doses of ST-920. ST-920 aims to provide stable, long-term production of α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should
- 114 views
- 11 Oct, 2022
- 15 locations
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal
- 0 views
- 22 Aug, 2023
- 6 locations
T1 Mapping in Fabry Disease (MAP-FAB)
values in kidney in Fabry's disease The main Objective is to describe renal performance through multi-parametric MRI in Fabry nephropathy and the primary outcome will be the quantification of renal T1 in
- 0 views
- 17 Sep, 2023
- 1 location
A Study of Replagal in Treatment-naïve Adults With Fabry Disease
In this study, adults with Fabry Disease who have not had any treatment for this condition will be treated with Replagal. The main aim of the study is to check if Replagal improves kidney
- 0 views
- 04 Oct, 2022
- 2 locations
Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease (RaILRoAD)
Fabry disease (FD) is a genetic disorder that leads to progressive accumulation of fat or 'sphingolipid' within the tissues, including the heart muscle and conductive tissue. Improvements in the
- 7 views
- 16 Jun, 2022
- 3 locations
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males With Fabry Disease
is comprised of adult males with Fabry Disease.
- 18 views
- 24 Apr, 2022
- 5 locations
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease (RISE)
The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease. It is planned of a total of
- 0 views
- 21 Aug, 2023
- 1 location