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A Study of rhGAA in Patients With Late-Onset Pompe Disease

NCT00250939 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

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 is to evaluate the safety, pharmacokinetics (PK) and efficacy of Myozyme treatment.

Conditions

  • Pompe Disease (Late-onset)
  • Glycogen Storage Disease Type II (GSD-II)
  • Acid Maltase Deficiency Disease
  • Glycogenosis 2

Interventions

BIOLOGICAL

Myozyme

20 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
5 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-02-28
Primary Completion
2006-07-31
Completion
2006-11-30

Countries

  • Netherlands

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 NCT00250939 on ClinicalTrials.gov