Organization
Amicus Therapeutics
15 clinical trials
Clinical trial
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-Stage Renal Disease Treated With HemodialysisStatus: Recruiting, Estimated PCD: 2024-12-31
Clinical trial
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and BreastfeedingStatus: Recruiting, Estimated PCD: 2029-08-01
Clinical trial
A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe DiseaseStatus: Active (not recruiting), Estimated PCD: 2024-12-01
Clinical trial
A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA VariantsStatus: Active (not recruiting), Estimated PCD: 2025-12-01
Clinical trial
An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe DiseaseStatus: Active (not recruiting), Estimated PCD: 2024-06-01
Clinical trial
A Global Prospective Observational Registry of Patients With Pompe DiseaseStatus: Recruiting, Estimated PCD: 2034-12-20
Clinical trial
A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/PlaceboStatus: Completed, Estimated PCD: 2020-12-15
Clinical trial
Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9Status: Active (not recruiting), Estimated PCD: 2024-09-30
Clinical trial
A Multi-country Time and Motion Study to Describe the Experience of Clinicians, Patients and Their Caregivers During the Treatment of Fabry Disease With Enzyme Replacement Therapy With Agalsidase Alfa and Agalsidase BetaStatus: Completed, Estimated PCD: 2022-05-18
Clinical trial
Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe DiseaseStatus:
Clinical trial
Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9Status: Completed, Estimated PCD: 2021-10-27
Clinical trial
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe DiseaseStatus: Recruiting, Estimated PCD: 2026-06-01
Clinical trial
Long-Term Follow-Up of AT-GTX-501 scAAV9 Gene Transfer in Subjects With CLN6 Batten DiseaseStatus: Active (not recruiting), Estimated PCD: 2024-10-01
Clinical trial
Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe DiseaseStatus:
Clinical trial
An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 YearsStatus: Recruiting, Estimated PCD: 2027-04-01