Study of rhASB in Patients With Mucopolysaccharidosis VI
A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI
1 other identifier
interventional
39
1 country
1
Brief Summary
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Feb 2004
Typical duration for phase_3
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2004
CompletedFirst Submitted
Initial submission to the registry
February 24, 2005
CompletedFirst Posted
Study publicly available on registry
February 25, 2005
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2006
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2006
CompletedResults Posted
Study results publicly available
March 6, 2009
CompletedFebruary 2, 2010
January 1, 2010
2.7 years
February 24, 2005
February 4, 2009
January 26, 2010
Conditions
Outcome Measures
Primary Outcomes (1)
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 Outcomes (2)
3-Minute Stair Climb
Baseline ASB-03-05 through week 96 of ASB-03-06.
Change in Urinary Glycosaminoglycans (GAG) Level
72 weeks
Study Arms (2)
rhASB/rhASB
OTHERN-acetylgalactosamine 4-sulfatase
Placebo/rhASB
OTHERInterventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
BioMarin Pharmaceutical Inc.
Novato, California, 94949, United States
Related Publications (1)
Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5.
PMID: 33874971DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Information Services
- Organization
- BioMarin Pharmaceutical Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
February 24, 2005
First Posted
February 25, 2005
Study Start
February 1, 2004
Primary Completion
October 1, 2006
Study Completion
October 1, 2006
Last Updated
February 2, 2010
Results First Posted
March 6, 2009
Record last verified: 2010-01