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

Glycogen Storage Disease Type II Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00763932
• Other study ID #: AGLU02003
• Status: Completed
• Phase: Phase 2
• First received: September 30, 2008
• Last updated: February 4, 2014
• Start date: April 2003
• Est. completion date: July 2006

Study information

• Verified date: February 2014
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 7
• Est. completion date: July 2006
• Est. primary completion date: June 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Glycogen Storage Disease
• Glycogen Storage Disease Type II
• Pompe Disease Infantile-Onset

Intervention

Biological:
• Myozyme
10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  France,  South Africa, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The objective of this extension study was to monitor the long-term safety and efficacy		3 years	No

External Links

•   Myozyme prescribing information