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rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

NCT00053573 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2014-02-05

No results posted yet for this study

Summary

Glycogen Storage Disease Type II ("GSD-II"; also known as Pompe disease) 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 GSD-II, 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 GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.

Conditions

  • Glycogen Storage Disease Type II
  • Pompe Disease
  • Acid Maltase Deficiency Disease
  • Glycogenosis 2

Interventions

BIOLOGICAL

Myozyme

20 mg/kg to 40 mg/kg qow

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
36 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2003-02-28
Primary Completion
2006-07-31
Completion
2006-11-30

Countries

  • United States
  • France
  • Israel
  • 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 NCT00053573 on ClinicalTrials.gov