BioMarin Pharmaceutical: Mucopolysaccharidoses trials

The registry lists 17 Mucopolysaccharidoses studies with BioMarin Pharmaceutical as lead sponsor. 0 are open to participants now and 10 have results posted.

Phase 1 3Phase 2 6Phase 3 5Phase 4 1No phase 4(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.

• Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited AmbulationTerminatedNCT01697319Phase 2Results posted
• Safety and Exercise Study of Two Doses of BMN 110 for Morquio A SyndromeTerminatedNCT01609062Phase 2Results posted
• Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01515956Phase 2Results posted
• Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01415427Phase 3Results posted
• A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01275066Phase 3Results posted
• A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)TerminatedNCT01242111Phase 1/2Results posted
• A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVACompletedNCT00884949Phase 1/2Results posted
• A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VICompletedNCT00299000Phase 4Results posted
• Study of rhASB in Patients With Mucopolysaccharidosis VICompletedNCT00104234Phase 3Results posted
• Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VICompletedNCT00067470Phase 3Results posted
• A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)CompletedNCT02294877
• BMN 110 Phase 3B in Australian PatientsCompletedNCT01966029Phase 3
• BMN 110 US Expanded Access ProgramApproved for marketingNCT01858103
• A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)TerminatedNCT00787995
• Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)CompletedNCT00214773
• Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VICompletedNCT00048711Phase 2
• Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VICompletedNCT00048620Phase 1