Study of rhASB in Patients With Mucopolysaccharidosis VI

Phase 3 Completed 39 enrolled BioMarin Pharmaceutical

Non-randomizedSingle-groupOpen-labelTreatment

Summary

The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.

Timeline

Start: 2004-02
Primary completion: 2006-10
Completion: 2006-10

Outcome

Met primary endpoint

release “After 24 weeks of treatment, patients receiving Naglazyme demonstrated a statistically significant improvement (p=0.025) in endurance compared to patients receiving placebo.” biomarin.com ↗

release “Patients receiving Naglazyme improved by a mean of 183 m +/- 26 m from baseline to week 96 in the 12MWT (p<0.001).” biomarin.com ↗

Publications

Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Galsulfase	Protein / enzyme biologic	—	—