Phase 3
Completed N=39
Study of rhASB in Patients With Mucopolysaccharidosis VI
Mucopolysaccharidosis VI
Source: ClinicalTrials.gov NCT00104234 ↗
Enrolled (actual)
39
Serious AEs
36.8%
Results posted
Mar 2009
Primary outcomePrimary: 12-Minute Walk Test — 187; 118 meters
Summary
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
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 | — |
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. Informational only — not medical advice.