Phase 3
N=39
Study of rhASB in Patients With Mucopolysaccharidosis VI
Mucopolysaccharidosis VI
Bottom Line
View on ClinicalTrials.gov: NCT00104234 ↗Enrolled (actual)
39
Serious AEs
36.8%
Results posted
Mar 2009
Primary outcome: Primary: 12-Minute Walk Test — 187; 118 meters
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- N-acetylgalactosamine 4-sulfatase (Drug); Placebo/rhASB (Drug)
- Age
- Pediatric, Adult, Older Adult · 7+ yrs
- Sex
- All
- Sponsor
- BioMarin Pharmaceutical
- Primary completion
- Oct 2006
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 12-Minute Walk Test |
187; 118 | — |
| SECONDARY 3-Minute Stair Climb |
13.1; 11.1 | — |
| SECONDARY Change in Urinary Glycosaminoglycans (GAG) Level |
-231 | — |
Summary
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
Eligibility Criteria
Inclusion Criteria
- Patient consent
- Patient must have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions
- If female of childbearing potential, patient must have a negative pregnancy test
Exclusion Criteria
- Pregnant or lactating patient
- Patient has received an investigational drug within 30 days prior to study enrollment
- Patient is unwilling or unable to travel to the primary site for periodic assessments
Data sourced from ClinicalTrials.gov (NCT00104234). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.