A Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI - Aryplase Phase 3 Open-Label Extensio
- Conditions
- Mucopolysaccharidosis Type VI (MPS VIMaroteaux-Lamy Syndrome)MedDRA version: 6.0 Level: PT Classification code 10056892
- Registration Number
- EUCTR2004-000642-21-IE
- Lead Sponsor
- BioMarin Pharmaceutical Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 38
· The patient must sign an informed consent form before any protocol-related procedures can be performed. If the patient is under 18 years of age, a legally authorized guardian must sign the informed consent form. In addition, patients who are under 18 years of age and can understand the consent form may be required to indicate their willingness to participate by signing the minor assent portion of the informed consent, if stipulated by the site’s IRB.
· The patient must have successfully completed Study ASB-03-05, including having received at least 20 of the 24 scheduled weekly infusions and having missed no more than two consecutive infusions.
· A female patient of childbearing potential must have a negative pregnancy test (urine b–human chorionic gonadotropin) at entry (prior to the first infusion). Note: All female patients of childbearing potential and sexually mature male patients must be advised to use a medically accepted method of contraception throughout the study. Female patients of childbearing potential must be willing to undergo periodic pregnancy tests during the course of the study.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
· The patient is pregnant or lactating.
· The patient has received an investigational drug (other than rhASB in Study ASB-03-05) within 30 days prior to study enrollment.
· The patient is unwilling or unable to travel to the primary site for periodic assessments.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The objective of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with MPS VI.;Secondary Objective: ;Primary end point(s): The primary efficacy endpoint, change in the number of metres walked in the 12-minute walk test, will be compared between two treatment groups. Differences between the rhASB/rhASB and placebo/rhASB groups with respect to the change from baseline (beginning of Study ASB-03-05) to Week 48 of the extension study and the change from Week 24 (end of Study ASB-03 05) to Week 48 of the extension study will be tested using analysis of variance models with the number of metres walked at baseline as the only covariate. Similar analyses will be performed for secondary and tertiary efficacy endpoints.
- Secondary Outcome Measures
Name Time Method