How many clinical trials are tracked for Fabry disease?

40 trials are tracked, 8 with published results.

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Fabry disease

Part of Cerebral small vessel disease

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Fabry disease

40 trials tracked for Fabry disease: 20 in phase 3 or 4 and 8 with published results. The most-cited published study has 555 citations.

40Trials tracked

20Phase 3 & 4

0Recruiting

8With published results

Phase distribution

Phase 4 4 Phase 3 16 Phase 2 16 Phase 1 1 Other / NA 3

Phase 3 Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease Completed · 555 cited
Phase 3 Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease Completed · 385 cited
Phase 3 A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms Completed · 167 cited
Phase 3 Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function Completed · 42 cited
Phase 3 Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) Completed · 31 cited
Phase 3 Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease Completed · 13 cited

Show 34 more trials

Phase 3 A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease Completed · 4 cited
Phase 3 Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks Completed
Phase 4 A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta Completed
Phase 4 A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease Completed
Phase 4 A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease Completed
Phase 4 A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease Completed
Phase 3 Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) Completed
Phase 3 Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease Completed
Phase 3 A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease Completed
Phase 3 Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease Completed
Phase 3 Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease Completed
Phase 3 Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease Completed
Phase 3 Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease Completed
Phase 3 Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease Completed
Phase 2 A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Completed
Phase 2 A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease Completed
Phase 2 A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Completed
Phase 2 Safety Study of Replagal® Therapy in Children With Fabry Disease Completed
Phase 2 A Study of Fabrazyme in Pediatric Patients With Fabry Disease Completed
Phase 2 Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR) Completed
Phase 2 Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease Completed
Phase 2 Extension Study of PRX-102 for up to 60 Months Completed
Phase 2 This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes. Completed
Phase 2 Dosing Study of Replagal in Patients With Fabry Disease Completed
Phase 2 Replagal Enzyme Replacement Therapy for Children With Fabry Disease Completed
Phase 2 A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Completed
Phase 2 Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease Completed
Phase 2 Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease Completed
Phase 2 Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients Completed
Phase 2 A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease Completed
Phase 1 PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion Completed
N/A A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. Completed
N/A Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients Completed
N/A Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan Completed

Showing the 40 most-cited and recently-updated of 40 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.