Study of rhASB in Patients With Mucopolysaccharidosis VI

BioMarin Pharmaceutical39 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Mucopolysaccharidosis VI

Interventions

N-acetylgalactosamine 4-sulfataseDRUG

Placebo/rhASBDRUG

Eligibility

Sex: ALLMin age: 7 Years

Medical Language ↔ Plain English

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

Locations (1)

BioMarin Pharmaceutical Inc.

Novato, California, United States

Outcomes

Primary Outcomes

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.

Time frame: Baseline of ASB-03-05 through week 96 of ASB-03-06

Secondary Outcomes

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.

Time frame: 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.

Time frame: 72 weeks

Data from ClinicalTrials.gov

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