Study of rhASB in Patients With Mucopolysaccharidosis VI

Phase 3

Completed

• Conditions: Mucopolysaccharidosis VI
• Interventions: Drug: N-acetylgalactosamine 4-sulfatase; Drug: Placebo/rhASB

• Registration Number: NCT00104234
• Lead Sponsor: BioMarin Pharmaceutical

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2004-02
• Study First Submitted: 2005-02-24
• Study First Submitted QC: 2005-02-24
• Study First Posted: 2005-02-25
• Primary Completion: 2006-10
• Study Completion: 2006-10
• Results First Submitted: 2009-02-04
• Results First Submitted QC: 2009-03-05
• Results First Posted: 2009-03-06
• Status Verified: 2010-01
• Last Update Submitted: 2010-01-26
• Last Update Posted: 2010-02-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 39

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
rhASB/rhASB	N-acetylgalactosamine 4-sulfatase	N-acetylgalactosamine 4-sulfatase
Placebo/rhASB	Placebo/rhASB	-

Primary Outcome Measures

Name	Time	Method
12-Minute Walk Test	Baseline of ASB-03-05 through week 96 of ASB-03-06	Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.

Secondary Outcome Measures

Name	Time	Method
3-Minute Stair Climb	Baseline ASB-03-05 through week 96 of ASB-03-06.	Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group.
Change in Urinary Glycosaminoglycans (GAG) Level	72 weeks	Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24.

Trial Locations

Locations (1)

BioMarin Pharmaceutical Inc.; 🇺🇸 Novato, California, United States