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COMPLETEDPhase 4NCT01288027

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

This is an open-label, multicenter study of participants with late-onset Pompe disease naive to treatment with enzyme replacement therapy (ERT). The primary objective of this study is to evaluate glyc...

Lead sponsor: Genzyme, a Sanofi Company•9 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 participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or confirmed GAA gene mutations and without known cardiac hypertrophy
•The participant is able to ambulate a distance without stopping and without an assistive device. Use of assistive device for community ambulation is appropriate
•The participant has a certain forced vital capacity (FVC) in upright position
•The participant, if female and of childbearing potential, must have a negative pregnancy test (urine beta-human chorionic gonadotropin \[beta-hCG\]) at baseline

Exclusion Criteria

•The participant has had previous treatment with ERT
•The participant is wheelchair dependent
•The participant requires invasive-ventilation (non-invasive ventilation is allowed)
•The participant is participating in another clinical study using investigational treatment
•The participant cannot submit to magnetic resonance imaging (MRI) examination because of a formal contraindication such as a pacemaker, implanted ferromagnetic metals, etc
•The participant, in the opinion of the Investigator, is unable to adhere to the requirements of the study

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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: December 19, 2014Terms

Trial snapshot

StatusCOMPLETED

PhasePHASE4

Enrollment16

Start dateJun 2011

Last updatedDec 19, 2014

Condition

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

Locations shown

Orange, California

Gainesville, Florida

Kansas City, Kansas

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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: December 19, 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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