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COMPLETEDPhase 2NCT00051935

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

GSD-II (also known as Pompe disease) 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...

Lead sponsor: Genzyme, a Sanofi Company•1 U.S. locations•View source on ClinicalTrials.gov

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Sponsors

Lead Sponsor

Genzyme, a Sanofi Company

Age

All ages

Sex

ALL

Healthy Volunteers

No

Inclusion Criteria

•Written informed consent must be obtained from the parent or guardian prior to performing any study related procedures;
•Patient must have a clinical diagnosis of GSD-II confirmed by endogenous GAA activity below normal in at least one tissue;
•Patient must have a sibling with a clinical diagnosis of GSD-II confirmed by an endogenous GAA activity below normal in at least one tissue, who is eligible for participation in this study;
•Patient must have a sibling with identical GAA mutations who is eligible for participation in this study;
•Patient must have a sibling with evidence of different progression of GSD-II who is eligible for participation in this study;
•The patient or his/her guardian(s) must have the ability to comply with the clinical protocol.

Exclusion Criteria

•Patient has significant organic disease (with the exception of symptoms relating to GSD-II), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, would preclude participation in the trial;
•Patient is participating in another investigational study.

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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: February 5, 2014Terms

Trial snapshot

StatusCOMPLETED

PhasePHASE2

Enrollment2

Start dateJan 2003

Last updatedFeb 5, 2014

Condition

Glycogen Storage Disease Type II Pompe Disease Acid Maltase Deficiency Disease Glycogenosis 2

Locations shown

Saint Peter's University Hospital

New Brunswick, New Jersey

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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 5, 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.

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