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Late-Onset Treatment Study Extension Protocol

NCT00455195 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 81

Last updated 2014-03-07

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 objective of this extension study is to assess the long-term safety and efficacy of alglucosidase alfa treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704 (NCT00158600).

Conditions

  • Pompe Disease (Late-Onset)
  • Glycogen Storage Disease Type II (GSD-II)
  • Glycogenesis Type II
  • Acid Maltase Deficiency (AMD)

Interventions

BIOLOGICAL

alglucosidase alfa

IV infusion of 20 mg/kg; every other week (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
PARALLEL

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-03-31
Primary Completion
2008-10-31
Completion
2008-11-30

Countries

  • United States
  • Australia
  • Canada
  • France
  • 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 NCT00455195 on ClinicalTrials.gov