Study of rhASB in Patients With Mucopolysaccharidosis VI
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ClinicalTrials.gov Identifier: NCT00104234 |
Recruitment Status :
Completed
First Posted : February 25, 2005
Results First Posted : March 6, 2009
Last Update Posted : February 2, 2010
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Mucopolysaccharidosis VI | Drug: N-acetylgalactosamine 4-sulfatase Drug: Placebo/rhASB | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 39 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI |
Study Start Date : | February 2004 |
Actual Primary Completion Date : | October 2006 |
Actual Study Completion Date : | October 2006 |

Arm | Intervention/treatment |
---|---|
rhASB/rhASB
N-acetylgalactosamine 4-sulfatase
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Drug: N-acetylgalactosamine 4-sulfatase
Other Name: rhASB |
Placebo/rhASB |
Drug: Placebo/rhASB |
- 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.
- 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.
- 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.

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Ages Eligible for Study: | 7 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00104234
United States, California | |
BioMarin Pharmaceutical Inc. | |
Novato, California, United States, 94949 |
ClinicalTrials.gov Identifier: | NCT00104234 |
Other Study ID Numbers: |
ASB-03-06 |
First Posted: | February 25, 2005 Key Record Dates |
Results First Posted: | March 6, 2009 |
Last Update Posted: | February 2, 2010 |
Last Verified: | January 2010 |
Mucopolysaccharidoses Mucopolysaccharidosis VI Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |
Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |