AGLU03206: Late-Onset Treatment Study Extension Protocol

This study is ongoing, but not recruiting participants.

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	Drug: Myozyme (alglucosidase alfa)	Phase 4

Study Type: Interventional

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

Number of arms in study: 1

Official Title: An Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704

Further Study Details:

Primary Outcome Measures:

Evaluate the safety of Myozyme [Time Frame: Two years]

Determine the effect of Myozyme on functional endurance as measured by the Six Minute Walk Test [Time Frame: Two years]

Determine the effect of Myozyme treatment on respiratory muscle weakness as measured by Forced Vital Capacity (FVC) [Time Frame: Two years]

Secondary Outcome Measures:

Determine the effect of Myozyme treatment on proximal muscle weakness and health-related quality of life [Time Frame: Two years]

Total Enrollment: 90
Study Start: 2007-03; Study Completion: 2009-03

Eligibility

Ages Eligible for Study: 8 Years and above

Genders Eligible for Study: Both

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.

Location and Contact Information

Arizona
Phoenix, Arizona, United States

California
San Diego, California, United States
Beverly Hills, California, United States

District of Columbia
Washington, District of Columbia, United States

Florida
Gainesville, Florida, United States

Illinois
Chicago, Illinois, United State

Kansas
Kansas City, Kansas, United States

Louisiana
New Orleans, Louisiana, United States

Massachusetts
Boston, Massachusetts, United States

Nebraska
Omaha, Nebraska, United States

New York
New York, New York, United States

Ohio
Cincinnati, Ohio, United States
Toledo, Ohio, United States

Oklahoma
Tulsa, Oklahoma, United States

Oregon
Portland, Oregon, United States

Pennsylvania
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States

South Carolina
Greenville, South Carolina, United States

Texas
Dallas, Texas, United States

Washington
Seattle, Washington, United States

Wisconsin
Green Bay, Wisconsin, United States

Australia
Westmead, Australia

Canada, Alberta
Calgary, Alberta, Canada

Canada, Quebec
Sherbrooke, Quebec, Canada

More Information

Myozyme® FDA-approved labeling information

Study ID Numbers: AGLU03206
ClinicalTrials.gov Identifier: NCT00455195
Health Authority: United States: Food and Drug Administration