Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Fabry Disease: full-pipeline trial registry
9 of 33 indexed Fabry Disease trials are currently recruiting.
Corpus placement: #463 of 28,707 conditions by registered trial count.
33 US clinical trials · 9 currently recruiting · #463 of 28,707 conditions by registered trial count
Fabry Disease: 33 US clinical trials tracked, 9 recruiting.
Fabry Disease is the subject of 33 registered US clinical trials on ClinicalTrials.gov, 9 of them currently open to new participants. 11 are in Phase 3-4 (later-stage) and 10 in Phase 1-2 (earlier-stage). The most active sponsor is Amicus Therapeutics, running 8 of these trials.
Enrollment posture brief
Registry condition fabry-disease
OPEN 9 | RECR 27% | LATE 11 | EARLY 10 | RANK #463 | SPON 8
Volume-matched condition peers
Nearest open-share peer: Atherosclerotic Cardiovascular Disease (27% recruiting · 22 trials)
Fabry Disease lists 9 open slots (27% of 33 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Fabry Disease on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Fabry Disease is recruiting below its size-band peers.
9 of 33 indexed trials (27.3%) are open to enrollment, against a 39.7% average across 762 conditions with 20-99 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Fabry Disease research.
Amicus Therapeutics accounts for 8 of 33 trials (24.2%). 14 distinct sponsors appear on at least one study for this condition label.
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Most Fabry Disease trials plan to enrol fewer than 100 people.
Median target enrolment is 29 among the 33 trials that report a genuine target (sentinel values excluded). 24 of those (72.7%) plan for fewer than 100 participants.
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Fabry Disease splits between interventional and observational designs.
22 interventional and 11 observational studies make up this index (66.7% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
33 total, page 1 of 1
Fabry Disease Registry & Pregnancy Sub-registry
Genzyme, a Sanofi Company
NCT00196742
A Study of Patients With Fabry Disease (US Specific)
Amicus Therapeutics
NCT06906367
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Chiesi Farmaceutici S.p.A.
NCT06663358
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
University of Minnesota
NCT01581424
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.
NCT06328608
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Amicus Therapeutics
NCT04252066
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
UniQure Biopharma B.V.
NCT06270316
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Chiesi Farmaceutici S.p.A.
NCT06941025
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Amicus Therapeutics
NCT06904261
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
NCT05206773
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Idorsia Pharmaceuticals
NCT03737214
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Sanofi
NCT05280548
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Chiesi Farmaceutici S.p.A.
NCT03614234
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
4D Molecular Therapeutics
NCT04519749
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Amicus Therapeutics
NCT04020055
Fabry Outcome Survey (FOS)
Shire
NCT03289065
Fabry Screening Study
Baylor Research Institute
NCT01019629
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Massachusetts General Hospital
NCT02985710
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Protalix
NCT02795676
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Amicus Therapeutics
NCT01218659
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Amicus Therapeutics
NCT00925301
The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study
University of Alabama at Birmingham
NCT00446862
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
Shire
NCT01124643
Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function
Amicus Therapeutics
NCT01730469
Alternative Dosing and Regimen of Replagal to Treat Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS)
NCT00075244
An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS)
NCT00071877
Replagal Enzyme Replacement Therapy for Adults With Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS)
NCT00097890
PET Scans in Normal Volunteers and Patients With Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS)
NCT00005111
Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
Amicus Therapeutics
NCT01196871
Safety Study of Replagal® Therapy in Children With Fabry Disease
Shire
NCT01363492
Dosing Study of Replagal in Patients With Fabry Disease
Baylor Research Institute
NCT00068107
Antiproteinuric Agents and Fabry Disease
University of Alabama at Birmingham
NCT00343577
Alpha-Galactosidase A Replacement Therapy for Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS)
NCT00048906
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 2 | 7 |
| Phase 3 | 10 |
| Phase 4 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Fabry Disease records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Fabry Disease trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Fabry Disease page
33 registered trials, 9 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 9 Fabry Disease trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Amicus Therapeutics sponsors the most Fabry Disease trials on record, review their full trial history before evaluating a specific study. See Amicus Therapeutics's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Fabry Disease condition card totals 33 registered trials; most-linked intervention Replagal; top US site state Virginia. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.