drugset / Trial / NCT01858103

BMN 110 US Expanded Access Program

NCT01858103 ↗

Expanded access Approved for marketing BioMarin Pharmaceutical

Summary

The Expanded Access Program (EAP) is an open-label, multicenter program to: 1. Provide patients who have been diagnosed with Mucopolysaccharidosis IVA (MPS IVA) access to BMN 110 until commercial product is available 2. Collect additional information on the safety and tolerability of BMN 110 administration in patients with MPS IVA Patients enrolled in the EAP will receive 2.0 mg/kg intravenous infusions of BMN 110 every week during the program.

Timeline

Start
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Primary completion
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Completion
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Drugs

EvaluationDrugModalityDoseRoute
Subject elosulfase alfa Protein / enzyme biologic 2 mg/kg Intravenous