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

Status

Indicates the current recruitment status or the expanded access status.
Approved for marketing

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 21, 2007

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 22, 2007

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 23, 2007

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 6, 2014

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 4, 2014

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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