Alglucosidase Alfa Temporary Access Program (ATAP)

February 4, 2014 updated by: Genzyme, a Sanofi Company
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 expanded access study is to provide patients with Pompe disease in the United States (US), access to alglucosidase alfa produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Sheffield, Alabama, United States
    • Alaska
      • Anchorage, Alaska, United States
    • Arizona
      • Phoenix, Arizona, United States
      • Prescott, Arizona, United States
      • Scottsdale, Arizona, United States
      • Tucson, Arizona, United States
    • California
      • Bakersfield, California, United States
      • Beverly Hills, California, United States
      • Loma Linda, California, United States
      • Los Angeles, California, United States
      • Sacramento, California, United States
      • San Diego, California, United States
      • Stanford, California, United States
    • Colorado
      • Aurora, Colorado, United States
    • District of Columbia
      • Washington, District of Columbia, United States
    • Florida
      • Bradenton, Florida, United States
      • Coral Springs, Florida, United States
      • Daytona Beach, Florida, United States
      • Gainsville, Florida, United States
      • St. Petersburg, Florida, United States
    • Georgia
      • Decatur, Georgia, United States
    • Idaho
      • Boise, Idaho, United States
    • Illinois
      • Chicago, Illinois, United States
    • Indiana
      • Evansville, Indiana, United States
    • Iowa
      • Iowa City, Iowa, United States
    • Kansas
      • Kansas City, Kansas, United States
    • Kentucky
      • Louisville, Kentucky, United States
    • Louisiana
      • New Orleans, Louisiana, United States
      • Shreveport, Louisiana, United States
    • Maine
      • Augusta, Maine, United States
    • Maryland
      • Baltimore, Maryland, United States
    • Massachusetts
      • Boston, Massachusetts, United States
      • Springfield, Massachusetts, United States
    • Michigan
      • Detroit, Michigan, United States
      • Grand Rapids, Michigan, United States
      • Lake Orion, Michigan, United States
      • Rochester Hills, Michigan, United States
      • St. Joseph, Michigan, United States
    • Minnesota
      • Minneapolis, Minnesota, United States
    • Montana
      • Missoula, Montana, United States
    • Nebraska
      • Omaha, Nebraska, United States
    • New Jersey
      • Midland Park, New Jersey, United States
    • New Mexico
      • Albuquerque, New Mexico, United States
    • New York
      • Albany, New York, United States
      • Corning, New York, United States
      • Manhasset, New York, United States
      • New York, New York, United States
    • North Carolina
      • Asheville, North Carolina, United States
      • Durham, North Carolina, United States
      • Jacksonville, North Carolina, United States
    • Ohio
      • Cincinnati, Ohio, United States
      • Cleveland, Ohio, United States
      • Greenville, Ohio, United States
      • Toledo, Ohio, United States
    • Oklahoma
      • Oklahoma City, Oklahoma, United States
      • Tulsa, Oklahoma, United States
    • Oregon
      • Portland, Oregon, United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States
      • Pittsburgh, Pennsylvania, United States
    • South Carolina
      • Greenville, South Carolina, United States
      • West Colombia, South Carolina, United States
    • Texas
      • Houston, Texas, United States
      • San Antonio, Texas, United States
      • Waco, Texas, United States
    • Utah
      • Salt Lake City, Utah, United States
    • Virginia
      • Charlottesville, Virginia, United States
      • Chesapeake, Virginia, United States
      • Newport News, Virginia, United States
      • Richmond, Virginia, United States
      • Winchester, Virginia, United States
    • Washington
      • Pullman, Washington, United States
      • Seattle, Washington, United States
    • West Virginia
      • Morgantown, West Virginia, United States
    • Wisconsin
      • Green Bay, Wisconsin, United States

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures.
  • The patient must reside in the US.
  • The patient must have a confirmed diagnosis of Pompe disease defined as documented acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or GAA gene mutations.
  • The patient must have/had documented clinical signs and symptoms of Pompe disease.
  • The patient must have/had prior treatment with alglucosidase alfa produced at commercial scale OR be naive to enzyme replacement therapy (ERT) for the treatment of Pompe disease and meet at least 1 of the following criteria: require a wheelchair OR require some respiratory assistance for any number of hours (including night time) through non-invasive ventilation.
  • The patient must be capable of complying with the required program schedule of assessments.

Exclusion Criteria:

  • Females who are pregnant or lactating
  • The patient has a clinical condition unrelated to Pompe disease that would interfere with program assessments.
  • The patient is currently enrolled in any clinical studies.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

August 21, 2007

First Submitted That Met QC Criteria

August 22, 2007

First Posted (Estimate)

August 23, 2007

Study Record Updates

Last Update Posted (Estimate)

February 6, 2014

Last Update Submitted That Met QC Criteria

February 4, 2014

Last Verified

February 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AGLU03907

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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