BioMarin Pharmaceutical: Mucopolysaccharidosis IV trials

The registry lists 11 Mucopolysaccharidosis IV studies with BioMarin Pharmaceutical as lead sponsor. 0 are open to participants now and 7 have results posted.

Phase 1 2Phase 2 5Phase 3 3No phase 3(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 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