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Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

NCT00125879 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2014-02-06

No results posted yet for this study

Summary

Pompe disease (also known as glycogen storage disease type 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. The overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.

Conditions

  • Glycogen Storage Disease Type II

Interventions

BIOLOGICAL

Myozyme

20 mg/kg qow or 40 mg/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
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-06-30
Primary Completion
2006-06-30
Completion
2006-12-31

Countries

  • United States
  • France
  • Germany
  • Israel
  • Italy
  • Netherlands
  • Taiwan

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 NCT00125879 on ClinicalTrials.gov