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

This study has been completed.
Information provided by:
Sanofi Identifier:
First received: August 1, 2005
Last updated: February 4, 2014
Last verified: February 2014
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
Biological: Myozyme
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Resource links provided by NLM:

Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Long-term Safety and Efficacy [ Time Frame: 52 weeks ]

Enrollment: 16
Study Start Date: June 2005
Study Completion Date: December 2006
Primary Completion Date: June 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Biological: Myozyme
20 mg/kg qow or 40 mg/kg qow
Other Name: alglucosidase alfa


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00125879

United States, Alabama
University of Alabama
Birmingham, Alabama, United States, 35233
United States, Florida
Shands Hospital at the University of Florida
Gainesville, Florida, United States, 32610
Miami Children's Hospital
Miami, Florida, United States, 33155
United States, Georgia
Emory University Medical Genetics
Decatur, Georgia, United States, 30033
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Ohio
Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
CHU Amiens
Amiens, France, 80054
CHU Cote de Nacre
Caen, France, 14033
Universitats-Kinderklinik Mainz
Mainz, Germany, 55131
Rambam Medical Center
Haifa, Israel, 35254
San Gerardo Hospital
Monza, Italy, 20052
Erasmus MC University
Rotterdam, Netherlands, 3015 GJ
Tzu-Chi General Hospital
Hua-lien, Taiwan, 970
Chi-Mei Medical Center Dept of Pediatrics
Tainan, Taiwan, 710
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Medical Monitor, Genzyme Corporation Identifier: NCT00125879     History of Changes
Other Study ID Numbers: AGLU02403
Study First Received: August 1, 2005
Last Updated: February 4, 2014

Keywords provided by Sanofi:
Glycogen Storage Disease Type II
Pompe Disease
Pompe Disease (Late-onset)
Acid Maltase Deficiency Disease
Glycogenosis 2
Glycogen Storage Disease Type II (GSD-II)

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases processed this record on April 26, 2017