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Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

NCT ID: NCT00125879

Last Updated: 2014-02-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-06-30

Study Completion Date

2006-12-31

Brief Summary

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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 overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.

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Detailed Description

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Conditions

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Glycogen Storage Disease Type II

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

Group Type EXPERIMENTAL

Myozyme

Intervention Type BIOLOGICAL

20 mg/kg qow or 40 mg/kg qow

Interventions

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Myozyme

20 mg/kg qow or 40 mg/kg qow

Intervention Type BIOLOGICAL

Other Intervention Names

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alglucosidase alfa

Eligibility Criteria

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Inclusion Criteria

* The patient's legal guardian(s) must provide written informed consent prior to any study-related procedures being performed
* The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol
* The patient must have completed Protocol AGLU01602.

Exclusion Criteria

* Patient has experienced any unmanageable adverse event (AE) in Protocol AGLU01602 due to Myozyme that would preclude continuing treatment with Myozyme
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role lead

Responsible Party

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Genzyme Corporation

Principal Investigators

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Medical Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

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University of Alabama

Birmingham, Alabama, United States

Site Status

Shands Hospital at the University of Florida

Gainesville, Florida, United States

Site Status

Miami Children's Hospital

Miami, Florida, United States

Site Status

Emory University Medical Genetics

Decatur, Georgia, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

CHU Amiens

Amiens, , France

Site Status

CHU Cote de Nacre

Caen, , France

Site Status

Universitats-Kinderklinik Mainz

Mainz, , Germany

Site Status

Rambam Medical Center

Haifa, , Israel

Site Status

San Gerardo Hospital

Monza, , Italy

Site Status

Erasmus MC University

Rotterdam, , Netherlands

Site Status

Tzu-Chi General Hospital

Hualien City, , Taiwan

Site Status

Chi-Mei Medical Center Dept of Pediatrics

Tainan City, , Taiwan

Site Status

Countries

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United States France Germany Israel Italy Netherlands Taiwan

References

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Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, Bali D, Smith SA, Li JS, Mandel H, Koeberl D, Rosenberg A, Chen YT. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab. 2010 Jan;99(1):26-33. doi: 10.1016/j.ymgme.2009.08.003.

Reference Type DERIVED
PMID: 19775921 (View on PubMed)

Other Identifiers

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AGLU02403

Identifier Type: -

Identifier Source: org_study_id

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