Fabry Disease Clinical Trial Landscape
Data updated: May 20, 2026
61 clinical trials tracked since 2008
Fabry Disease is being studied across 61 clinical trials registered since 2008, with 15 programs currently active. The competitive pipeline includes 9 active Phase 3 trials, 7 active Phase 2 trials, and 4 active Phase 1 trials. Top industry sponsors include Chiesi Farmaceutici S.p.A., Amicus Therapeutics, Sanofi.
15 active
/
61 total (since 2008)
4
Phase 1 Active
22 total
7
Phase 2 Active
20 total
9
Phase 3 Active
28 total
1
Phase 4 Active
4 total
Top Industry Sponsors
Sorted by active Active Done Failed
Chiesi Farmaceutici S.p.A. 3 1 0
Amicus Therapeutics 2 11 1
Sanofi 2 0 1
4D Molecular Therapeutics 2 0 0
Takeda 1 6 1
+ 7 more sponsors — see full table below
Featured Phase 3 Trials
NCT06904261 RECRUITING
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Amicus Therapeutics n=8
NCT04020055 ACTIVE NOT RECRUITING
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Amicus Therapeutics n=14
NCT06328608 RECRUITING
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Chiesi Farmaceutici S.p.A. n=22
NCT05710692 RECRUITING
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Chiesi Farmaceutici S.p.A. n=16
NCT05280548 ACTIVE NOT RECRUITING
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Sanofi n=104
+ 23 more Phase 3 trials — see full database below
Fabry Disease — Trials Started by Year (last 10 years)
2016 3
2017 3
2018 6
2019 3
2020 3
2021 3
2022 8
2023 3
2024 1
2025 2
2026 1
TheraRadar.com
Full Trial Database
Complete sponsor table, every trial across all phases, plus competitive analytics. Sortable, filterable, exportable for Pro users.
Top Sponsors (all-time)
Chiesi Farmaceutici S.p.A. 4
Amicus Therapeutics 14
Sanofi 3
4D Molecular Therapeutics 2
Takeda 8
Idorsia Pharmaceuticals Ltd. 3
UniQure Biopharma B.V. 1
Guangzhou Jiayin Biotech Ltd 1
AceLink Therapeutics, Inc. 1
ISU Abxis Co., Ltd. 1
Active Trials by Phase
Phase 1 4
Phase 2 7
Phase 3 9
Phase 4 1
Enrollment Size Distribution
1-50 46
51-200 11
201-500 0
501-1k 0
1k+ 0
All Industry Sponsors
Sorted by active Active Done Failed
Chiesi Farmaceutici S.p.A. 3 1 0
Amicus Therapeutics 2 11 1
Sanofi 2 0 1
4D Molecular Therapeutics 2 0 0
Takeda 1 6 1
Idorsia Pharmaceuticals Ltd. 1 2 0
UniQure Biopharma B.V. 1 0 0
Guangzhou Jiayin Biotech Ltd 1 0 0
AceLink Therapeutics, Inc. 1 0 0
ISU Abxis Co., Ltd. 1 0 0
Genzyme, a Sanofi Company 0 5 1
Protalix 0 5 1
NCT06904261 RECRUITING
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Amicus Therapeutics n=8
NCT04020055 ACTIVE NOT RECRUITING
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Amicus Therapeutics n=14
NCT06328608 RECRUITING
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Chiesi Farmaceutici S.p.A. n=22
NCT05710692 RECRUITING
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Chiesi Farmaceutici S.p.A. n=16
NCT05280548 ACTIVE NOT RECRUITING
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Sanofi n=104
NCT05206773 ACTIVE NOT RECRUITING
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease
Sanofi n=122
NCT03614234 ACTIVE NOT RECRUITING
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Chiesi Farmaceutici S.p.A. n=29
NCT03737214 ACTIVE NOT RECRUITING
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Idorsia Pharmaceuticals Ltd. n=107
NCT06081062 RECRUITING
Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
ISU Abxis Co., Ltd. n=24
NCT03566017 COMPLETED
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
Chiesi Farmaceutici S.p.A. n=97
NCT05843916 COMPLETED
Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
Bio Sidus SA n=20
NCT04049760 COMPLETED
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Amicus Therapeutics n=16
NCT04974749 COMPLETED
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
Takeda n=20
NCT03425539 COMPLETED
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Idorsia Pharmaceuticals Ltd. n=118
NCT02795676 COMPLETED
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Protalix n=78
NCT03018730 COMPLETED
Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
Protalix n=22
NCT03180840 COMPLETED
Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
Protalix n=30
NCT03500094 COMPLETED
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Amicus Therapeutics n=22
NCT00864851 COMPLETED
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
Shire n=44
NCT01298141 COMPLETED
A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
Shire n=171
NCT01124643 COMPLETED
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
Shire n=35
NCT02194985 COMPLETED
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
Amicus Therapeutics n=84
NCT01218659 COMPLETED
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Amicus Therapeutics n=68
NCT00925301 COMPLETED
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Amicus Therapeutics n=67
NCT01458119 TERMINATED
Open-Label Phase 3 Long-Term Safety Study of Migalastat
Amicus Therapeutics n=85
NCT02921620 WITHDRAWN
Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease
Protalix
NCT00701415 COMPLETED
A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
Genzyme, a Sanofi Company n=31
Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only