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Clinical Trial 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.


Clinical Trial Description

n/a


Study Design

N/A


Related Conditions & MeSH terms


NCT number NCT01858103
Study type Expanded Access
Source BioMarin Pharmaceutical
Contact
Status Approved for marketing
Phase N/A

See also
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Completed NCT01415427 - Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Phase 3
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Completed NCT01920828 - Gait Analysis in MPS IVA
Terminated NCT01697319 - Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation Phase 2
Terminated NCT01609062 - Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome Phase 2