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Growth and Development Study of Myozyme
This study is currently recruiting participants.
Verified by Genzyme, July 2008
Sponsored by: Genzyme
Information provided by: Genzyme
ClinicalTrials.gov Identifier: NCT00486889
  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 objective of this study is to evaluate the long-term growth and development of patients with infantile-onset Pompe disease who are treated with Myozyme before 1 year of age over a 10-year period.


Condition Intervention Phase
Pompe Disease
Glycogen Storage Disease Type II (GSD-II)
Acid Maltase Deficiency Disease
Biological: Myozyme® (alglucosidase alfa)
Phase IV

Genetics Home Reference related topics: Pompe disease
Drug Information available for: Alglucosidase Alfa
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study
Official Title: A Long-Term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Myozyme® (Alglucosidase Alfa)

Further study details as provided by Genzyme:

Primary Outcome Measures:
  • Long-term growth and development [ Time Frame: 10 years ] [ Designated as safety issue: No ]
  • Collect long-term safety data on patients with infantile-onset Pompe disease. [ Time Frame: Up to 10 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 30
Study Start Date: December 2007
Estimated Study Completion Date: September 2020
Estimated Primary Completion Date: September 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
1: Experimental Biological: Myozyme® (alglucosidase alfa)
IV infusion: 20mg/kg qow

  Eligibility

Ages Eligible for Study:   up to 12 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures;
  • The patient must have a confirmed diagnosis of Pompe disease as determined by deficient endogenous GAA activity or GAA mutation analysis; and
  • The patient must be < 1 year of age prior to enrolling in this study.

Exclusion Criteria:

  • The patient is participating in another clinical study using Myozyme or any other clinical trial.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00486889

Contacts
Contact: Medical Information 800-745-4447 medinfo@genzyme.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com

Locations
United States, Georgia
Recruiting
Decatur, Georgia, United States
United States, Massachusetts
Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient. Recruiting
Cambridge, Massachusetts, United States
Sponsors and Collaborators
Genzyme
Investigators
Study Director: Medical Monitor Genzyme
  More Information

US FDA Approved Full Prescribing Information for Myozyme®  This link exits the ClinicalTrials.gov site

Responsible Party: Genzyme Corporation ( Medical Monitor )
Study ID Numbers: AGLU03606
Study First Received: June 13, 2007
Last Updated: November 3, 2008
ClinicalTrials.gov Identifier: NCT00486889  
Health Authority: United States: Food and Drug Administration

Keywords provided by Genzyme:
Glycogenesis 2

Study placed in the following topic categories:
Metabolic Diseases
Glycogen Storage Disease
Lysosomal Storage Diseases
Central Nervous System Diseases
Glycogen Storage Disease Type II
Brain Diseases
Glycogen storage disease type 2
Metabolism, Inborn Errors
Malnutrition
Genetic Diseases, Inborn
Nutrition Disorders
Brain Diseases, Metabolic, Inborn
Metabolic disorder
Deficiency Diseases
Brain Diseases, Metabolic

Additional relevant MeSH terms:
Lysosomal Storage Diseases, Nervous System
Nervous System Diseases
Carbohydrate Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on January 16, 2009