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Clinical Trial Summary

The primary objective of this study was to evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 196 weeks. Secondary objectives were to investigate the effect of the two doses on exercise capacity for up to 196 weeks. In addition, the pharmacokinetic (PK) parameters of both doses of BMN 110 was assessed.


Clinical Trial Description

n/a


Study Design

Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT01609062
Study type Interventional
Source BioMarin Pharmaceutical
Contact
Status Terminated
Phase Phase 2
Start date April 2012
Completion date November 2014

See also
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Terminated NCT01697319 - Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation Phase 2
Approved for marketing NCT01858103 - BMN 110 US Expanded Access Program N/A