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Naglazyme in Infants with MPS VI
NCT00299000Phase 4
Key facts
- Status
- Completed
- Start
- May 2006
- Primary completion
- Apr 2009
- Countries
- 3
- Sponsor
- BioMarin Pharmaceutical
- Modality
- Replacement Enzymes
- Drugs
- galsulfase
- Conditions
- mucopolysaccharidosis type VI
01
Overview
Phase 4, randomised, open-label trial of galsulfase in mucopolysaccharidosis type VI (2 arms), sponsored by BioMarin Pharmaceutical. Primary endpoints: Change in Height at 52 weeks, Change in Weight at 52 weeks and 2 more. Started May 2006; status: completed.
02
Design
2 arms- Study design
- RandomizedOpen-label
Naglazyme 1.0 mg/kg
Naglazyme 1.0 mg/kg weekly infusion
Naglazyme 2.0 mg/kg
Naglazyme 2.0 mg/kg weekly infusion
03
Who it enrols
Infants under 1 year of age with a confirmed diagnosis of MPS VI (enzyme activity <10%) and no evidence of skeletal dysplasia on physical exam. Excludes patients with prior Naglazyme treatment or HSCT.
04
Endpoints
Primary
Change in Height
Change in Weight
Change in Head Circumference
Progression of Skeletal Dysplasia
Secondary
Change in Urinary Glycosaminoglycan Levels
Gross and Fine Motor Function
Cardiac Function
Vision Assessment
Hearing Assessment
Use of Health Resources
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