BioMarin Pharmaceutical: Glycogen Storage Disease Type II trials
The registry lists 14 Glycogen Storage Disease Type II studies with BioMarin Pharmaceutical as lead sponsor. 2 are open to participants now and 9 have results posted.
Phase 1 2Phase 2 5Phase 3 4No phase 5(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 Global Prospective Observational Registry of Patients With Pompe DiseaseRecruitingNCT06121011
- Expanded Access for ATB200/AT2221 for the Treatment of Pompe DiseaseAvailableNCT03865836
- A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)CompletedNCT04138277Phase 3Results posted
- A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe DiseaseCompletedNCT03729362Phase 3Results posted
- First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221CompletedNCT02675465Phase 1/2Results posted
- A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)TerminatedNCT02191917Results posted
- BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)TerminatedNCT01924845Phase 3Results posted
- Extension Study for Patients Who Have Participated in a BMN 701 StudyTerminatedNCT01435772Phase 2Results posted
- Drug-drug Interaction StudyCompletedNCT01380743Phase 2Results posted
- Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe DiseaseCompletedNCT01230801Phase 1/2Results posted
- Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe DiseaseTerminatedNCT00688597Phase 2Results posted
- 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
- A Prospective, Noninterventional, Observational Study of Late-Onset Pompe DiseaseTerminatedNCT02221362
- A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe DiseaseCompletedNCT00515398