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A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

NCT00158600 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 90

Last updated 2015-04-28

Study results available
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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, efficacy, and pharmacokinetics (PK) of alglucosidase alfa treatment in patients with late-onset Pompe disease as compared to placebo.

Conditions

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

Interventions

BIOLOGICAL

alglucosidase alfa

IV infusion of 20mg/kg; qow for 78 weeks.

DRUG

Placebo

Placebo Comparator; qow for 78 weeks.

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-09-30
Primary Completion
2007-09-30
Completion
2007-09-30

Countries

  • United States
  • France
  • Netherlands

Study Locations

More Related Trials

Entities

Drugs

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 NCT00158600 on ClinicalTrials.gov