Late-Onset Treatment Study Extension Protocol

Last updated: February 4, 2014
Sponsor: Genzyme, a Sanofi Company
Overall Status: Completed

Phase

4

Condition

Liver Disorders

Pompe Disease

Treatment

N/A

Clinical Study ID

NCT00455195
AGLU03206
2006-003644-31
  • Ages > 8
  • All Genders

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 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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patient must have completed protocol AGLU02704 (NCT00158600)

  • Patient must provide signed, informed consent prior to performing any study-relatedprocedures

  • Patient (and patient's legal guardian if patient is under 18 years of age) must havethe ability to comply with the clinical protocol

  • A female patient of childbearing potential must have a negative pregnancy test atBaseline. (note: all female patients of childbearing potential and sexually maturemales must use a medically accepted method of contraception throughout the study.)

Exclusion

Exclusion Criteria:

  • The patient has a medical condition, serious intercurrent illness, or otherextenuating circumstance that, in the opinion of the Investigator, would precludetreatment with alglucosidase alfa.

Study Design

Total Participants: 81
Study Start date:
March 01, 2007
Estimated Completion Date:
November 30, 2008

Connect with a study center

  • Westmead,
    Australia

    Site Not Available

  • Calgary, Alberta
    Canada

    Site Not Available

  • Sherbrooke, Quebec
    Canada

    Site Not Available

  • Paris,
    France

    Site Not Available

  • Rotterdam,
    Netherlands

    Site Not Available

  • Anchorage, Alaska
    United States

    Site Not Available

  • Phoenix, Arizona
    United States

    Site Not Available

  • Beverly Hills, California
    United States

    Site Not Available

  • San Diego, California
    United States

    Site Not Available

  • Washington, District of Columbia
    United States

    Site Not Available

  • Coral Springs, Florida
    United States

    Site Not Available

  • Gainesville, Florida
    United States

    Site Not Available

  • Sarasota, Florida
    United States

    Site Not Available

  • Chicago, Illinois
    United States

    Site Not Available

  • Kansas City, Kansas
    United States

    Site Not Available

  • New Orleans, Louisiana
    United States

    Site Not Available

  • Boston, Massachusetts
    United States

    Site Not Available

  • Omaha, Nebraska
    United States

    Site Not Available

  • New York, New York
    United States

    Site Not Available

  • Durham, North Carolina
    United States

    Site Not Available

  • Cincinnati, Ohio
    United States

    Site Not Available

  • Toledo, Ohio
    United States

    Site Not Available

  • Tulsa, Oklahoma
    United States

    Site Not Available

  • Portland, Oregon
    United States

    Site Not Available

  • Philadelphia, Pennsylvania
    United States

    Site Not Available

  • Pittsburgh, Pennsylvania
    United States

    Site Not Available

  • Greenville, South Carolina
    United States

    Site Not Available

  • Dallas, Texas
    United States

    Site Not Available

  • San Antonio, Texas
    United States

    Site Not Available

  • Seattle, Washington
    United States

    Site Not Available

  • Green Bay, Wisconsin
    United States

    Site Not Available

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