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ATAP: Alglucosidase Alfa Temporary Access Program

Sponsor
Genzyme, a Sanofi Company (Industry)
Overall Status
Approved for marketing
CT.gov ID
NCT00520143
Collaborator
(none)
74
Locations

Study Details

Study Description

Brief 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 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.

Condition or Disease Intervention/Treatment Phase
  • Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])

Study Design

Study Type:
Expanded Access
Official Title:
Alglucosidase Alfa Temporary Access Program

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Sheffield Alabama United States
    2 Anchorage Alaska United States
    3 Phoenix Arizona United States
    4 Prescott Arizona United States
    5 Scottsdale Arizona United States
    6 Tucson Arizona United States
    7 Bakersfield California United States
    8 Beverly Hills California United States
    9 Loma Linda California United States
    10 Los Angeles California United States
    11 Sacramento California United States
    12 San Diego California United States
    13 Stanford California United States
    14 Aurora Colorado United States
    15 Washington District of Columbia United States
    16 Bradenton Florida United States
    17 Coral Springs Florida United States
    18 Daytona Beach Florida United States
    19 Gainsville Florida United States
    20 St. Petersburg Florida United States
    21 Decatur Georgia United States
    22 Boise Idaho United States
    23 Chicago Illinois United States
    24 Evansville Indiana United States
    25 Iowa City Iowa United States
    26 Kansas City Kansas United States
    27 Louisville Kentucky United States
    28 New Orleans Louisiana United States
    29 Shreveport Louisiana United States
    30 Augusta Maine United States
    31 Baltimore Maryland United States
    32 Boston Massachusetts United States
    33 Springfield Massachusetts United States
    34 Detroit Michigan United States
    35 Grand Rapids Michigan United States
    36 Lake Orion Michigan United States
    37 Rochester Hills Michigan United States
    38 St. Joseph Michigan United States
    39 Minneapolis Minnesota United States
    40 Missoula Montana United States
    41 Omaha Nebraska United States
    42 Midland Park New Jersey United States
    43 Albuquerque New Mexico United States
    44 Albany New York United States
    45 Corning New York United States
    46 Manhasset New York United States
    47 New York New York United States
    48 Asheville North Carolina United States
    49 Durham North Carolina United States
    50 Jacksonville North Carolina United States
    51 Cincinnati Ohio United States
    52 Cleveland Ohio United States
    53 Greenville Ohio United States
    54 Toledo Ohio United States
    55 Oklahoma City Oklahoma United States
    56 Tulsa Oklahoma United States
    57 Portland Oregon United States
    58 Philadelphia Pennsylvania United States
    59 Pittsburgh Pennsylvania United States
    60 Greenville South Carolina United States
    61 West Colombia South Carolina United States
    62 Houston Texas United States
    63 San Antonio Texas United States
    64 Waco Texas United States
    65 Salt Lake City Utah United States
    66 Charlottesville Virginia United States
    67 Chesapeake Virginia United States
    68 Newport News Virginia United States
    69 Richmond Virginia United States
    70 Winchester Virginia United States
    71 Pullman Washington United States
    72 Seattle Washington United States
    73 Morgantown West Virginia United States
    74 Green Bay Wisconsin United States

    Sponsors and Collaborators

    • Genzyme, a Sanofi Company

    Investigators

    • Study Director: Medical Monitor, Genzyme, a Sanofi Company

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Genzyme, a Sanofi Company
    ClinicalTrials.gov Identifier:
    NCT00520143
    Other Study ID Numbers:
    • AGLU03907
    First Posted:
    Aug 23, 2007
    Last Update Posted:
    Feb 6, 2014
    Last Verified:
    Feb 1, 2014
    Keywords provided by Genzyme, a Sanofi Company
    GSD-II, Pompe Disease, Glycogen Storage Disease II
    Additional relevant MeSH terms:
    Glycogen Storage Disease Type II
    Glycogen Storage Disease
    Deficiency Diseases

    Study Results

    No Results Posted as of Feb 6, 2014