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

Phase 2

Completed

• Conditions: Glycogen Storage Disease Type II; Pompe Disease Infantile-Onset
• Interventions: Biological: Myozyme

• Registration Number: NCT00763932
• Lead Sponsor: Genzyme, a Sanofi Company

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2003-04
• Primary Completion: 2006-06
• Study Completion: 2006-07
• Study First Submitted: 2008-09-30
• Study First Submitted QC: 2008-09-30
• Study First Posted: 2008-10-01
• Status Verified: 2014-02
• Last Update Submitted: 2014-02-04
• Last Update Posted: 2014-02-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 7

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	Myozyme	-

Primary Outcome Measures

Name	Time	Method
The objective of this extension study was to monitor the long-term safety and efficacy	3 years

Trial Locations

Locations (8)

Institute for Genetic Medicine, Saint Peter's University Hospital; 🇺🇸 New Brunswick, New Jersey, United States

University of Nebraska Medical Center; 🇺🇸 Omaha, Nebraska, United States

Children's Hospital of Orange Country; 🇺🇸 Orange, California, United States

Children's Hospital of Oakland; 🇺🇸 Oakland, California, United States

Pediatrique Hospital Debrousse; 🇫🇷 Lyon, France

New York University [NYU] School of Medicine; 🇺🇸 New York, New York, United States

Hôpital Porte Madeleine; 🇫🇷 Orleans, France

The Morningside Clinic; 🇿🇦 Johannesburg, South Africa