NCT00884949
MPS IVA, MPS IV A
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed with Mucopolysaccharidosis IVA (MPS IVA).
All
5 Years to 18 Years
No
BMN 110
Phase 1/Phase 2
Interventional
20
2009-04
2014-06-30
Birmingham, , United Kingdom
London, , United Kingdom
Manchester, , United Kingdom
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