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A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

NCT00059280 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2014-02-05

No results posted yet for this study

Summary

Pompe disease (also known as glycogen storage disease type II, "GSD-II") is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied.

Conditions

  • Glycogen Storage Disease Type II

Interventions

BIOLOGICAL

Myozyme

20 mg/kg qow or 40mg/kg qow

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
FACTORIAL

Eligibility

Max Age
26 Weeks
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2003-04-30
Primary Completion
2005-06-30
Completion
2005-09-30

Countries

  • United States
  • France
  • Israel
  • Taiwan
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00059280 on ClinicalTrials.gov