BioMarin Pharmaceutical: Fabry Disease trials

The registry lists 26 Fabry Disease studies with BioMarin Pharmaceutical as lead sponsor. 4 are open to participants now and 13 have results posted.

Phase 1 5Phase 2 6Phase 3 8No phase 7(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• A Study of Patients With Fabry Disease (US Specific)RecruitingNCT06906367
• A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsRecruitingNCT06904261Phase 3
• A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and BreastfeedingRecruitingNCT04252066
• Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry DiseaseAvailableNCT01476163
• A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.CompletedNCT04281537Results posted
• Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry DiseaseCompletedNCT04049760Phase 3Results posted
• Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)CompletedNCT03500094Phase 3Results posted
• Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry DiseaseCompletedNCT02194985Phase 3Results posted
• Open-Label Phase 3 Long-Term Safety Study of MigalastatTerminatedNCT01458119Phase 3Results posted
• Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry DiseaseCompletedNCT01218659Phase 3Results posted
• Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry DiseaseCompletedNCT01196871Phase 2Results posted
• Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry DiseaseCompletedNCT00925301Phase 3Results posted
• Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 StudyTerminatedNCT00526071Phase 2Results posted
• A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry DiseaseCompletedNCT00304512Phase 2Results posted
• A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCompletedNCT00283959Phase 2Results posted
• A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCompletedNCT00283933Phase 2Results posted
• A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCompletedNCT00214500Phase 2Results posted
• Understanding Fabry Disease Therapy Choices Through the Eyes of the PatientsCompletedNCT04804566
• French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With MigalastatCompletedNCT04602364
• Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and PreferencesCompletedNCT04043273
• A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal DiseaseActive, not recruitingNCT04020055Phase 3
• A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy VolunteersCompletedNCT02082327Phase 1
• 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
• A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)CompletedNCT01730482Phase 1
• Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal FunctionCompletedNCT01730469Phase 1
• Migalastat Food Effect StudyCompletedNCT01489995Phase 1