Amicus Therapeutics
Open enrollment across a mixed-phase pipeline
According to ClinicalTrials.gov-derived indexes, Amicus Therapeutics sits in the top decile of US sponsors by registered trial count (#484 of 15,742 sponsors by registered trial count).
Amicus Therapeutics: 34 sponsored US clinical trials, 5 recruiting.
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.
Top-decile sponsor footprint
Amicus Therapeutics ranks in the top decile of sponsors by registered trial count (#484 of 15,742 sponsors by registered trial count). Volume at this scale usually reflects a long institutional pipeline, read phase tilt and therapeutic areas below before treating it as current-year activity.
Trial completion reliability score
22 of 27 decided trials (Completed vs. Terminated on ClinicalTrials.gov) reached their planned completion (81.5%), vs. national p10 68.4% / p90 100% among 1,234 sponsors with at least 10 decided trials. Still-open trials (Recruiting, Active-not-recruiting, Not-yet-recruiting) are excluded since they have not reached an outcome yet.
Open and recent trials
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 is open in the Amicus Therapeutics pipeline
5 recruiting of 34 indexed (15% open); 22 completed (65%).
Phase mix: 12 late (III/IV) vs 13 early (I/II) (balanced across stages).
Top condition: Fabry Disease (8 trials), 6 others listed.
How open-pipeline share is computed
Recruiting share is recruiting_count divided by trial_count for this sponsor in the public registry export. Status can change between pulls; treat the figure as a research-attention snapshot, not product availability.
Amicus Therapeutics: open-pipeline peers nationwide
Peers matched on 34 registered trials and 15% open enrollment, not the therapeutic-area sidebar.
Amicus Therapeutics sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together.
Sponsors near 34 trials (± recruiting share)
- Allegheny Singer Research Institute (also known as Allegheny Health Network Research Institute) · 34 trials · 38% recruiting
- American Regent · 34 trials · 0% recruiting
- Cochlear · 34 trials · 12% recruiting
- Emergent BioSolutions · 34 trials · 0% recruiting
Sponsors near 15% open enrollment (± trial volume)
- Brown University · 15% recruiting · 231 trials
- Loma Linda University · 15% recruiting · 237 trials
- United States Naval Medical Center, San Diego · 15% recruiting · 41 trials
- University of Rochester · 15% recruiting · 636 trials
Related
What to do with this Amicus Therapeutics page
34 registered trials is a research-attention snapshot, not an endorsement of Amicus Therapeutics or any single study.
- 5 of Amicus Therapeutics's trials are currently recruiting, check each one's own eligibility criteria before contacting a site. Browse recruiting trials
- Fabry Disease is the condition Amicus Therapeutics studies most, compare against other sponsors active in the same condition before evaluating a specific study. See Fabry Disease 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.
Amicus Therapeutics pipeline card: 34 registered trials from official public datasets.Report a data error.