Amicus Therapeutics clinical trials

Acquired by BioMarin Pharmaceutical, Apr 27, 2026 · source ↗

The registry lists 42 studies whose lead sponsor is Amicus Therapeutics. 8 are recruiting or about to start. Per ClinicalTrials.gov, last updated Oct 22, 2026.

Studies

Recruiting first, then most recently updated. Showing 42 of 42. All BioMarin Pharmaceutical studies →

• A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsRecruitingNCT06904261Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study of Patients With Fabry Disease (US Specific)RecruitingNCT06906367

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Global Prospective Observational Registry of Patients With Pompe DiseaseRecruitingNCT06121011

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18RecruitingNCT04808505Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Expanded Access for ATB200/AT2221 for the Treatment of Pompe DiseaseAvailableNCT03865836

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Expanded Access for ATB200/AT2221 for the Treatment of IOPDAvailableNCT04327973

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and BreastfeedingRecruitingNCT04252066

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry DiseaseAvailableNCT01476163

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDActive, not recruitingNCT03911505Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal DiseaseActive, not recruitingNCT04020055Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)CompletedNCT04138277Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry DiseaseCompletedNCT04049760Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.CompletedNCT04281537

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221CompletedNCT02675465Phase 1/2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe DiseaseCompletedNCT03729362Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe DiseaseTerminatedNCT00688597Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERTTerminatedNCT03347253

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Drug-drug Interaction StudyCompletedNCT01380743Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Understanding Fabry Disease Therapy Choices Through the Eyes of the PatientsCompletedNCT04804566

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)CompletedNCT03500094Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry DiseaseCompletedNCT02194985Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry DiseaseCompletedNCT01196871Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry DiseaseCompletedNCT01218659Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCompletedNCT00283959Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry DiseaseCompletedNCT00925301Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCompletedNCT00214500Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry DiseaseCompletedNCT00304512Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 StudyTerminatedNCT00526071Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Open-Label Phase 3 Long-Term Safety Study of MigalastatTerminatedNCT01458119Phase 3

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement TherapyCompletedNCT00433147Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCompletedNCT00283933Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher PatientsCompletedNCT00813865Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher DiseaseCompletedNCT00446550Phase 2

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis BullosaCompletedNCT03158662

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal FunctionCompletedNCT01730469Phase 1

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy VolunteersCompletedNCT02082327Phase 1

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)CompletedNCT01730482Phase 1

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Migalastat Food Effect StudyCompletedNCT01489995Phase 1

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese SubjectsCompletedNCT01853852Phase 1

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101TerminatedNCT00875160Phase 1

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• Study to Evaluate Blood Cell Lines From Patients With Gaucher DiseaseCompletedNCT00351156

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical

• A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe DiseaseCompletedNCT00515398

  Sponsor listed in the registry: Amicus Therapeutics · First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical