Study of rhASB in Patients With Mucopolysaccharidosis VI

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

Official Title
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A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI

Conditions
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Mucopolysaccharidosis VI

Intervention / Treatment
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* Drug: N-acetylgalactosamine 4-sulfatase
* Drug: Placebo/rhASB


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 7 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: rhASB/rhASB<br>N-acetylgalactosamine 4-sulfatase | Drug: N-acetylgalactosamine 4-sulfatase<br> <br> * Other names: rhASB;|
| Other: Placebo/rhASB<br> | Drug: Placebo/rhASB<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 12-Minute Walk Test | 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. | Baseline of ASB-03-05 through week 96 of ASB-03-06 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 3-Minute Stair Climb | 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. | Baseline ASB-03-05 through week 96 of ASB-03-06. | 
| Change in Urinary Glycosaminoglycans (GAG) Level | 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. | 72 weeks |