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 - 10/08/11
, Roberto Giugliani, MD, PhD, Ida Schwartz, MD, Nathalie Guffon, MD, Elisa Leão Teles, MD, M. Clara Sá Miranda, PhD, J. Edmond Wraith, MD, Michael Beck, MD, Laila Arash, MD, Maurizio Scarpa, MD, Zi-Fan Yu, ScD 1, Janet Wittes, PhD 1, Kenneth I. Berger, MD 1, Mary S. Newman, MS 2, Ann M. Lowe, MD 1, Emil Kakkis, MD, PhD 2, Stuart J. Swiedler, MD, PhD 2MPS VI Phase 3 Study Group
Reprint requests: Dr Paul Harmatz, Children’s Hospital & Research Center at Oakland, 747 52nd Street, Oakland, CA 94609.Resumen |
Objective |
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.
Study design |
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.
Results |
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 <.001). Infusions were generally safe and well tolerated. Patients exposed to drug experienced positive clinical benefit despite the presence of antibody to the protein.
Conclusion |
rhASB significantly improves endurance, reduces GAG, and has an acceptable safety profile.
El texto completo de este artículo está disponible en PDF.Abbreviations : AE, ASB, ERT, FVC, GAG, IAR, MPS, MVV, rhASB, ROM, 3MSC, 12MWT
Esquema
| This study was sponsored by BioMarin Pharmaceutical Inc., and it was supported, in part, with funds provided by the National Center for Research Resources, 5 M01 RR-01271 (Dr Harmatz). |
Vol 148 - N° 4
P. 533 - avril 2006 Regresar al número
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