Region:

Text Size

  • Smaller
  • Regular
  • Large

Open-label extension data

56 patients from 3 clinical studies were followed in an open-label extension period up to 144 weeks:

  • All patients received weekly infusions of NAGLAZYME® (galsulfase) at
    1 mg/kg of body weight7

12-minute walk test

NAGLAZYME 12-minute walk test

View Enlarged Image

  • By week 96, the group treated with NAGLAZYME showed a 183±26 m improvement from baseline
  • The crossover group showed a 117±25 m increase from the beginning of the open-label extension period

*Fitted values
† By week 96, patients in the drug group had been taking NAGLAZYME for 96 weeks; patients in the crossover group had only been taking NAGLAZYME for the 72-week open-label extension period.
‡ One patient in placebo group left the study for reasons unrelated to treatment. Patients receiving placebo were switched to NAGLAZYME in the trial extension period.
§One patient failed to complete the assessment.

3 Minute Stair Climb

NAGLAZYME 3-minute climb test

View Enlarged Image

  • By week 96, the group treated with NAGLAZYME showed a 13.1±2.0 stairs/min improvement from baseline stair-climbing rate
  • The crossover group showed an 11.0±1.7 stairs/min increase in stair-climbing rate from the beginning of the open-label extension period

*Fitted values
† By week 96, patients in the drug group had been taking NAGLAZYME for 96 weeks; patients in the placebo group had only been taking NAGLAZYME for the 72-week open-label extension period.
‡ One patient in placebo group left the study for reasons unrelated to treatment. Patients receiving placebo were switched to NAGLAZYME in the trial extension period.
§One patient failed to complete the assessment.

Urinary GAG levels

NAGLAZYME reduced urinary glycosaminoglycans

View Enlarged Image

  • The reduction in mean urinary GAG levels was maintained through
    week 96

* One patient in placebo group left the study for reasons unrelated to treatment. Patients receiving placebo were switched to NAGLAZYME in the trial extension period. Fitted values
† One patient failed to complete the assessment.
‡ Two patients failed to complete the assessment.

 Next: Dosing and administration

References

  1. Harmatz P, Giugliani R, Schwartz I, et al. 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. J Pediatr. 2006;148:533-539.
  1. Wraith JE. The mucopolysaccharidoses: a clinical review and guide to management. Arch Dis Childhood. 1995;72:263–267.
  1. Harmatz RP. Mucopolysaccharidosis type VI. Emedicine. December 18, 2006. Available at: http://www.Emedicine.com/PED/topic1373.htm Accessed: November 4, 2008.