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Alglucosidase Alfa Temporary Access Program | Clinical Trials | Clareo Health

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Approved For MarketingNCT00520143

Alglucosidase Alfa Temporary Access Program

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...

Lead sponsor: Genzyme, a Sanofi Company•74 U.S. locations•View source on ClinicalTrials.gov

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Sponsors

Lead Sponsor

Genzyme, a Sanofi Company

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

No

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.

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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: February 6, 2014Terms

Trial snapshot

StatusAPPROVED_FOR_MARKETING

Phase—

Enrollment—

Start date—

Last updatedFeb 6, 2014

Condition

Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-Onset)Acid Maltase Deficiency Disease Glycogenosis 2

Locations shown

Sheffield, Alabama

Anchorage, Alaska

Phoenix, Arizona

For caregivers

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 6, 2014Data synced to Clareo: April 28, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions