Back to resultsExtension 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
Primary Purpose
Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Myozyme
Sponsored by
About this trial
This is an interventional treatment trial for Pompe Disease Infantile-Onset
Eligibility 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
Sites / Locations
- Children's Hospital of Oakland
- Children's Hospital of Orange Country
- University of Nebraska Medical Center
- Institute for Genetic Medicine, Saint Peter's University Hospital
- New York University [NYU] School of Medicine
- Pediatrique Hospital Debrousse
- Hôpital Porte Madeleine
- The Morningside Clinic
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
1
Arm Description
Outcomes
Primary Outcome Measures
The objective of this extension study was to monitor the long-term safety and efficacy
Secondary Outcome Measures
Full Information
NCT ID
NCT00763932
First Posted
September 30, 2008
Last Updated
February 4, 2014
Sponsor
Genzyme, a Sanofi Company
1. Study Identification
Unique Protocol Identification Number
NCT00763932
Brief Title
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
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2014
Overall Recruitment Status
Completed
Study Start Date
April 2003 (undefined)
Primary Completion Date
June 2006 (Actual)
Study Completion Date
July 2006 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Genzyme, a Sanofi Company
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
7 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Myozyme
Other Intervention Name(s)
Alglucosidase alfa
Intervention Description
10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow
Primary Outcome Measure Information:
Title
The objective of this extension study was to monitor the long-term safety and efficacy
Time Frame
3 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital of Oakland
City
Oakland
State/Province
California
Country
United States
Facility Name
Children's Hospital of Orange Country
City
Orange
State/Province
California
Country
United States
Facility Name
University of Nebraska Medical Center
City
Omaha
State/Province
Nebraska
Country
United States
Facility Name
Institute for Genetic Medicine, Saint Peter's University Hospital
City
New Brunswick
State/Province
New Jersey
Country
United States
Facility Name
New York University [NYU] School of Medicine
City
New York
State/Province
New York
Country
United States
Facility Name
Pediatrique Hospital Debrousse
City
Lyon
Country
France
Facility Name
Hôpital Porte Madeleine
City
Orleans
Country
France
Facility Name
The Morningside Clinic
City
Johannesburg
Country
South Africa
12. IPD Sharing Statement
Learn more about this trial
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
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