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

Completed · Phase 2/Phase 3

Conditions studied: Glycogen Storage Disease Type II

In brief

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.

Key facts

Study ID

NCT00125879

Run by

Genzyme, a Sanofi Company

People needed

16

Starts

2005-06-01

Expected to finish

2006-12-01

Last updated by the study team

2014-02-06

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Patient has experienced any unmanageable adverse event (AE) in Protocol AGLU01602 due to Myozyme that would preclude continuing treatment with Myozyme

Where it is running

• University of Alabama — Birmingham, Alabama, United States
• Shands Hospital at the University of Florida — Gainesville, Florida, United States
• Miami Children's Hospital — Miami, Florida, United States
• Emory University Medical Genetics — Decatur, Georgia, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Children's Hospital Medical Center — Cincinnati, Ohio, 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 — Hualien City, Taiwan
• Chi-Mei Medical Center Dept of Pediatrics — Tainan, Taiwan

Full record on ClinicalTrials.gov

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