Amicus Therapeutics
Amicus Therapeutics sponsors 34 registered US clinical trials on ClinicalTrials.gov, 5 of them currently recruiting, and 22 completed. 12 of these trials are in Phase 3-4 (later-stage) and 13 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Fabry Disease, with 8 trials.
Trial Pipeline
34 total, page 1 of 1
A Study of Patients With Fabry Disease (US Specific)
NCT06906367
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
NCT06904261
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
NCT04252066
A Global Prospective Observational Registry of Patients With Pompe Disease
NCT06121011
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
NCT04808505
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
NCT04020055
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
NCT03911505
Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
NCT04804566
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
NCT04281537
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
NCT04138277
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
NCT04049760
A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
NCT03729362
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
NCT03500094
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
NCT03347253
Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa
NCT03158662
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
NCT02675465
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
NCT02194985
Drug-drug Interaction Study
NCT01380743
Open-Label Phase 3 Long-Term Safety Study of Migalastat
NCT01458119
Migalastat Food Effect Study
NCT01489995
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
NCT01218659
Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function
NCT01730469
Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
NCT01196871
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
NCT00925301
A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
NCT00813865
A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
NCT00875160
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
NCT00688597
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
NCT00446550
Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
NCT00526071
A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
NCT00515398
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
NCT00433147
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
NCT00304512
Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
NCT00351156
A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
NCT00214500
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 4 |
| Phase 2 | 9 |
| Phase 3 | 12 |
What the Pipeline for Amicus Therapeutics Shows
According to the ClinicalTrials.gov registry, Amicus Therapeutics is linked to 34 US clinical trials across every stage of research activity. Of those, 5 studies are currently recruiting, about 15% of the sponsor's indexed portfolio, and 22 are already marked complete, representing roughly 65% of the total.
The phase mix for Amicus Therapeutics reports 12 late-stage studies (Phase 3 and Phase 4 combined) and 13 earlier-phase studies (Phase 1 and Phase 2).
The top therapeutic focus area indexed for Amicus Therapeutics is Fabry Disease with 8 linked trials, and 6 other condition areas appear in the top list above.
How to read these pipeline numbers
Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.
A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work, safety, dosing, and early signal detection, and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.
That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice, verify current details directly with ClinicalTrials.gov before relying on any figure here.
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.