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

Phase 2

Completed

• Conditions: Glycogen Storage Disease Type II
• Interventions: Biological: Myozyme

• Registration Number: NCT00125879
• Lead Sponsor: Genzyme, a Sanofi Company

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2005-06
• Study First Submitted: 2005-08-01
• Study First Submitted QC: 2005-08-01
• Study First Posted: 2005-08-02
• Primary Completion: 2006-06
• Study Completion: 2006-12
• Status Verified: 2014-02
• Last Update Submitted: 2014-02-04
• Last Update Posted: 2014-02-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	Myozyme	-

Primary Outcome Measures

Name	Time	Method
Long-term Safety and Efficacy	52 weeks

Trial Locations

Locations (14)

Shands Hospital at the University of Florida; 🇺🇸 Gainesville, Florida, United States

CHU Amiens; 🇫🇷 Amiens, France

CHU Cote de Nacre; 🇫🇷 Caen, France

Universitats-Kinderklinik Mainz; 🇩🇪 Mainz, Germany

Rambam Medical Center; 🇮🇱 Haifa, Israel

San Gerardo Hospital; 🇮🇹 Monza, Italy

Erasmus MC University; 🇳🇱 Rotterdam, Netherlands

Tzu-Chi General Hospital; 🇨🇳 Hua-lien, Taiwan

Chi-Mei Medical Center Dept of Pediatrics; 🇨🇳 Tainan, Taiwan

Emory University Medical Genetics; 🇺🇸 Decatur, Georgia, United States

University of Alabama; 🇺🇸 Birmingham, Alabama, United States

Miami Children's Hospital; 🇺🇸 Miami, Florida, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States