- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00104234
Study of rhASB in Patients With Mucopolysaccharidosis VI
January 26, 2010 updated by: BioMarin Pharmaceutical
A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
39
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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California
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Novato, California, United States, 94949
- BioMarin Pharmaceutical Inc.
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
7 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Other: rhASB/rhASB
N-acetylgalactosamine 4-sulfatase
|
Other Names:
|
Other: Placebo/rhASB
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
12-Minute Walk Test
Time Frame: 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.
|
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
Time Frame: 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.
|
Baseline ASB-03-05 through week 96 of ASB-03-06.
|
Change in Urinary Glycosaminoglycans (GAG) Level
Time Frame: 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.
|
72 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
February 1, 2004
Primary Completion (Actual)
October 1, 2006
Study Completion (Actual)
October 1, 2006
Study Registration Dates
First Submitted
February 24, 2005
First Submitted That Met QC Criteria
February 24, 2005
First Posted (Estimate)
February 25, 2005
Study Record Updates
Last Update Posted (Estimate)
February 2, 2010
Last Update Submitted That Met QC Criteria
January 26, 2010
Last Verified
January 1, 2010
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ASB-03-06
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Mucopolysaccharidosis VI
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, United Kingdom
-
University of ChicagoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedKrabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)United States
-
Fondazione TelethonActive, not recruitingMucopolysaccharidosis Type VIItaly, Turkey
-
University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VI | Mucopolysaccharidosis Type IV | Mucopolysaccharidosis Type VIIUnited States, Canada
-
Lundquist Institute for Biomedical Innovation at...CompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VIUnited States
-
Hospital de Clinicas de Porto AlegreThe Isaac FoundationActive, not recruitingMucopolysaccharidoses | Mucopolysaccharidosis VI | Morquio A Syndrome | Mucopolysaccharidosis IV A | MPS IV A | MPS VI | MPS - Mucopolysaccharidosis | Morquio Syndrome A | Morquio SyndromeBrazil
-
Children's Hospitals and Clinics of MinnesotaBioMarin Pharmaceutical; Gillette Children's Specialty Healthcare; Greenwood...CompletedMucopolysaccharidosis VI | Mucopolysaccharidosis IV AUnited States
-
Inventiva PharmaCompletedMucopolysaccharidosis VIUnited Kingdom, France, Germany, Portugal
-
BioMarin PharmaceuticalCompletedMucopolysaccharidosis VIUnited States
-
BioMarin PharmaceuticalCompletedMucopolysaccharidosis VIUnited States
Clinical Trials on N-acetylgalactosamine 4-sulfatase
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VIUnited States
-
BioMarin PharmaceuticalCompletedMucopolysaccharidosis VIUnited States
-
BioMarin PharmaceuticalCompletedMucopolysaccharidosis VIUnited States
-
BioMarin PharmaceuticalTerminatedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited Kingdom, United States, Germany
-
BioMarin PharmaceuticalCompletedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVATaiwan, United States, Italy, United Kingdom
-
BioMarin PharmaceuticalCompletedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV AUnited States, Canada, France, Taiwan, Argentina, Colombia, Spain, Turkey, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, United Kingdom, Germany, Norway, Portugal, Italy, Qatar
-
BioMarin PharmaceuticalCompletedMucopolysaccharidosis IVA (Morquio A Syndrome)Australia
-
BioMarin PharmaceuticalTerminatedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited States, United Kingdom, Canada, Germany
-
BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI | Maroteaux-Lamy SyndromeUnited States, Portugal, France
-
BioMarin PharmaceuticalCompletedMPS IV AUnited States, Canada, France, United Kingdom, Taiwan, Argentina, Colombia, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, Germany, Portugal, Italy, Qatar