Amicus Therapeutics

34 total trials 5 currently recruiting 22 completed

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

RECRUITING

A Study of Patients With Fabry Disease (US Specific)

NCT06906367

RECRUITING Phase 3

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261

RECRUITING

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

NCT04252066

RECRUITING

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011

RECRUITING Phase 3

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

NCT04808505

ACTIVE NOT RECRUITING Phase 3

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055

ACTIVE NOT RECRUITING Phase 3

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505

COMPLETED

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT04804566

COMPLETED

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

NCT04281537

COMPLETED Phase 3

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

NCT04138277

COMPLETED Phase 3

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

NCT04049760

COMPLETED Phase 3

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

NCT03729362

COMPLETED Phase 3

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

NCT03500094

TERMINATED

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

NCT03347253

COMPLETED

Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa

NCT03158662

COMPLETED Phase 1

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT02675465

COMPLETED Phase 3

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

NCT02194985

COMPLETED Phase 2

Drug-drug Interaction Study

NCT01380743

TERMINATED Phase 3

Open-Label Phase 3 Long-Term Safety Study of Migalastat

NCT01458119

COMPLETED Phase 1

Migalastat Food Effect Study

NCT01489995

COMPLETED Phase 3

Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

NCT01218659

COMPLETED Phase 1

Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function

NCT01730469

COMPLETED Phase 2

Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

NCT01196871

COMPLETED Phase 3

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

NCT00925301

COMPLETED Phase 2

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865

TERMINATED Phase 1

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

NCT00875160

TERMINATED Phase 2

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

NCT00688597

COMPLETED Phase 2

A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

NCT00446550

TERMINATED Phase 2

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

NCT00526071

COMPLETED

A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

NCT00515398

COMPLETED Phase 2

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

NCT00433147

COMPLETED Phase 2

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

NCT00304512

COMPLETED

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

NCT00351156

COMPLETED Phase 2

A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

NCT00214500

Phase Distribution

PhaseTrial 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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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.