Study of rhASB in Patients With Mucopolysaccharidosis VI
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00104234|
Recruitment Status : Completed
First Posted : February 25, 2005
Results First Posted : March 6, 2009
Last Update Posted : February 2, 2010
|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|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
Drug: N-acetylgalactosamine 4-sulfatase
Other Name: 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.
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|