AGLU02403: Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

This study has been completed.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00125879

Purpose

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.

Condition	Intervention	Phase
Glycogen Storage Disease Type II	Drug: Myozyme (alglucosidase alfa)	Phase II, Phase III

Study Type: Interventional

Study Design: Treatment, Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Official Title: A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Further Study Details:

Expected Total Enrollment: 16

Study start: June 2005

Eligibility

Ages Eligible for Study: Both

Criteria

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

Location Information

Alabama
University of Alabama, Birmingham, Alabama, 35233, United States

Florida
Shands Hospital at the University of Florida, Gainesville, Florida, 32610, United States
Miami Children's Hospital, Miami, Florida, 33155, United States

Georgia
Emory University Medical Genetics, Decatur, Georgia, 30033, United States

North Carolina
Duke University Medical Center, Durham, North Carolina, 27710, United States

Ohio
Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States

France
CHU Amiens, Amiens, 80054, France
CHU Cote de Nacre, Caen, 14033, France

Germany
Universitats-Kinderklinik Mainz, Mainz, 55131, Germany

Israel
Rambam Medical Center, Haifa, 35254, Israel

Italy
San Gerardo Hospital, Monza, 20052, Italy

Netherlands
Erasmus MC University, Rotterdam, 3015 GJ, Netherlands

Taiwan
Chi-Mei Medical Center Dept of Pediatrics, Tainan, 710, Taiwan
Tzu-Chi General Hospital, Hua-lien, 970, Taiwan

More Information

Myozyme® FDA-approved labeling information

Study ID Numbers: AGLU02403
ClinicalTrials.gov Identifier: NCT00125879
Health Authority: United States: Food and Drug Administration