Extension Study of Long-Term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

This study has been completed.

First Received: September 30, 2008 Last Updated: July 7, 2009 History of Changes

Sponsored by:	Genzyme
Information provided by:	Genzyme
ClinicalTrials.gov Identifier:	NCT00763932

Purpose

This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line

Condition	Intervention	Phase
Pompe Disease Infantile-Onset Glycogen Storage Disease Type II	Biological: Myozyme	Phase II

Study Type:	Interventional
Study Design:	Treatment, Open Label, Single Group Assignment, Safety/Efficacy Study
Official Title:	A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies

Resource links provided by NLM:

Genetics Home Reference related topics: Pompe disease succinic semialdehyde dehydrogenase deficiency

Drug Information available for: Alglucosidase Alfa

U.S. FDA Resources

Further study details as provided by Genzyme:

Primary Outcome Measures:

The objective of this extension study was to monitor the long-term safety and efficacy [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Enrollment:	7
Study Start Date:	April 2003
Study Completion Date:	July 2006
Primary Completion Date:	June 2006 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental	Biological: Myozyme 10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

The patient was enrolled in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502
The patient's legal guardian(s) provided written informed consent prior to any study related procedures being performed
The patient and his/her guardian(s) were able to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.

Exclusion Criteria:

Patients were excluded from this study if they did not meet the specific inclusion criteria, or if the patient experienced any unmanageable AE in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation), due to Synpac rhGAA therapy, that would preclude continuing therapy

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00763932

Locations

United States, California
Children's Hospital of Orange Country
Orange, California, United States
Children's Hospital of Oakland
Oakland, California, United States
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States
United States, New Jersey
Institute for Genetic Medicine, Saint Peter's University Hospital
New Brunswick, New Jersey, United States
United States, New York
New York University [NYU] School of Medicine
New York, New York, United States
France
Pediatrique Hospital Debrousse
Lyon, France
Hôpital Porte Madeleine
Orleans, France
South Africa
The Morningside Clinic
Johannesburg, South Africa

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Medical Monitor

Genzyme

More Information

Additional Information:

Myozyme prescribing information This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Genzyme Corporation ( Medical Monitor )
Study ID Numbers:	AGLU02003
Study First Received:	September 30, 2008
Last Updated:	July 7, 2009
ClinicalTrials.gov Identifier:	NCT00763932 History of Changes
Health Authority:	United States: Food and Drug Administration

Study placed in the following topic categories:

Metabolism, Inborn Errors
Glycogen Storage Disease Type 2
Metabolic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Glycogen Storage Disease

Central Nervous System Diseases
Glycogen Storage Disease Type II
Brain Diseases, Metabolic, Inborn
Brain Diseases
Metabolic Disorder
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Glycogen Storage Disease
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Glycogen Storage Disease Type II

Brain Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Carbohydrate Metabolism, Inborn Errors
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on September 11, 2009