Late-Onset Treatment Study Extension Protocol - Full Text View

Sponsored by:	Genzyme
Information provided by:	Genzyme
ClinicalTrials.gov Identifier:	NCT00455195

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 extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704.

Condition	Intervention	Phase
Pompe Disease (Late-Onset) Glycogen Storage Disease Type II (GSD-II) Glycogenesis 2 Acid Maltase Deficiency	Biological: Myozyme	Phase IV

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study
Official Title:	An Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704

Resource links provided by NLM:

Genetics Home Reference related topics: Pompe disease succinic semialdehyde dehydrogenase deficiency

Drug Information available for: Alglucosidase Alfa

U.S. FDA Resources

Further study details as provided by Genzyme:

Primary Outcome Measures:

Evaluate the safety of Myozyme [ Time Frame: Two years ] [ Designated as safety issue: No ]
Determine the effect of Myozyme on functional endurance as measured by the Six Minute Walk Test [ Time Frame: Two years ] [ Designated as safety issue: No ]
Determine the effect of Myozyme treatment on respiratory muscle weakness as measured by Forced Vital Capacity (FVC) [ Time Frame: Two years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Determine the effect of Myozyme treatment on proximal muscle weakness and health-related quality of life [ Time Frame: Two years ] [ Designated as safety issue: No ]
Determine the effect of Myozyme on health-related quality of life [ Time Frame: two years ] [ Designated as safety issue: No ]

Estimated Enrollment:	90
Study Start Date:	March 2007
Study Completion Date:	November 2008
Primary Completion Date:	October 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental	Biological: Myozyme IV infusion of 20 mg/kg; qow

Eligibility

Ages Eligible for Study:	8 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

patient must have completed protocol AGLU02704
patient must provide signed, informed consent prior to performing any study-related procedures
patient (and patient's legal guardian if patient is under 18 years of age) must have the ability to comply with the clinical protocol
a female patient of childbearing potential must have a negative pregnancy test at Baseline. (note: all female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.)

Exclusion Criteria:

the patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, would preclude treatment with Myozyme.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00455195

Show 29 Study Locations

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Medical Monitor

Genzyme Coorporation

More Information

Additional Information:

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

No publications provided

Responsible Party:	Genzyme ( Medical Monitor )
Study ID Numbers:	AGLU03206
Study First Received:	March 30, 2007
Last Updated:	November 25, 2008
ClinicalTrials.gov Identifier:	NCT00455195 History of Changes
Health Authority:	United States: Food and Drug Administration; Canada: Health Canada; Australia: Therapeutic Goods Administration

Study placed in the following topic categories:

Metabolism, Inborn Errors
Glycogen Storage Disease Type 2
Metabolic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Glycogen Storage Disease

Central Nervous System Diseases
Glycogen Storage Disease Type II
Brain Diseases, Metabolic, Inborn
Brain Diseases
Metabolic Disorder
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Glycogen Storage Disease
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Glycogen Storage Disease Type II

Brain Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Carbohydrate Metabolism, Inborn Errors
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on September 10, 2009