AGLU03907: Myozyme Temporary Access Program (MTAP)

Expanded access is currently available for this treatment.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00268944

Purpose

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 US, access to Myozyme 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	Intervention
Glycogen Storage Disease Type II Pompe Disease Acid Maltase Deficeincy Disease Glycogenosis 2	Drug: Myozyme® (recombinant human acid alpha-glucosidase (rhGAA))

Study Type: Expanded Access

Official Title: Myozyme Temporary Access Program

Further Study Details:

Participating US Locations:

Phoenix, Arizona

Prescott, Arizona;

Scottsdale, Arizona

Bakersfield, California

Beverly Hills, California

Decatur, Georgia

Chicago, Illinois

Evansville, Indiana

Shreveport, Louisiana

Plymouth, Massachusetts

Grand Rapids, Michigan

Lake Orion, Michigan

Midland Park, New Jersey

Albany, New York

Corning, New York

New York, New York

Durham, North Carolina

Portland, Oregon

Philadelphia, Pennsylvania

Salt Lake City, Utah

Charlottesville, Virginia

Chesapeake, Virginia

Newport News, Virginia

Greenbay, Wisconsin

Eligibility

Ages Eligible for Study: 18 Years and above; Genders Eligible for Study: Both

Criteria

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 Myozyme produced at commercial scal OR be naive to 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.

Location and Contact Information

Medical Information 800-745-4447 medinfo@genzyme.com
Medical Information 617-252-7832 medinfo@genzyme.com

More Information

Myozyme® FDA-approved labeling information

Study ID Numbers AGLU03907
ClinicalTrials.gov Identifier NCT00520143
Health Authority: United States: Food and Drug Administration