Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study

The objective of this Phase 3 study was to confirm the efficacy and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI; Maroteaux-Lamy syndrome), a rare, fatal lysosomal storage disease with no effective treatment. Thirty-nine patients with MPS VI...

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Published in:The Journal of pediatrics 2006-04, Vol.148 (4), p.533-539.e6
Main Authors: Harmatz, Paul, Giugliani, Roberto, Schwartz, Ida, Guffon, Nathalie, Teles, Elisa Leão, Miranda, M. Clara Sá, Wraith, J. Edmond, Beck, Michael, Arash, Laila, Scarpa, Maurizio, Yu, Zi-Fan, Wittes, Janet, Berger, Kenneth I., Newman, Mary S., Lowe, Ann M., Kakkis, Emil, Swiedler, Stuart J.
Format: Article
Language:English
Subjects:
Adolescent
Adult
Analysis of Variance
Biological and medical sciences
Carbohydrates (enzymatic deficiencies). Glycogenosis
Child
Double-Blind Method
Errors of metabolism
Female
Follow-Up Studies
Forced Expiratory Volume
General aspects
Glycosaminoglycans - urine
Humans
Linear Models
Male
Medical sciences
Metabolic diseases
Mucopolysaccharidosis VI - drug therapy
N-Acetylgalactosamine-4-Sulfatase - therapeutic use
Recombinant Proteins
Safety
Walking
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Summary:The objective of this Phase 3 study was to confirm the efficacy and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI; Maroteaux-Lamy syndrome), a rare, fatal lysosomal storage disease with no effective treatment. Thirty-nine patients with MPS VI were evaluated in a randomized, double-blind, placebo-controlled, multicenter, multinational study for 24 weeks. The primary efficacy variable was the distance walked in a 12-minute walk test (12MWT), whereas the secondary efficacy variables were the number of stairs climbed in a 3-minute stair climb (3MSC) and the level of urinary glycosaminoglycan (GAG) excretion. All patients received drug in an open-label extension period for an additional 24 weeks. After 24 weeks, patients receiving rhASB walked on average 92 meters (m) more in the 12MWT ( p = .025) and 5.7 stairs per minute more 3MSC ( p = .053) than patients receiving placebo. Continued improvement was observed during the extension study. Urinary GAG declined by -227 ± 18 μg/mg more with rhASB than placebo ( p
ISSN:0022-3476
1097-6833
DOI:10.1016/j.jpeds.2005.12.014