AGLU02704: Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease

This study is ongoing, but not recruiting participants.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00158600

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 is to evaluate the safety, efficacy, and pharmacokinetics (PK) of Myozyme treatment in patients with late-onset Pompe disease as compared to placebo.

Condition	Intervention	Phase
Pompe Disease (Late-Onset) Glycogen Storage Disease Type II (GSD-II) Acid Maltase Deficiency Disease Glycogenosis 2	Drug: Myozyme (alglucosidase alfa)	Phase III

Study Type: Interventional

Study Design: Treatment, Randomized, Double-Blind, Placebo Control, Factorial Assignment, Safety/Efficacy Study

Official Title: Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.

Further Study Details:

Primary Outcomes:

Total Enrollment: 90

Study Start: 2005-09; Study Completion: 2007-09

Eligibility

Ages Eligible for Study: 8 Years and above, Genders Eligible for Study: Both

Criteria

Inclusion Criteria:

patient must provide signed, informed consent prior to performing any study-related procedures.

patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity in cultured skin fibroblasts of less than or equal to 40% of the normal mean of the testing laboratory and 2 confirmed GAA gene mutations;

patient must be greater than or equal to 8 years of age at the time of enrollment;

patient must be able to ambulate 40 meters (approximately 130 feet) in 6 minutes on each of 2 consecutive tests performed on the same day (use of assistive devices such as a walker, cane, or crutches, is permitted);

patient must have an FVC of greater than or equal to 30% and < 80% predicted in the upright position;

patient must have a postural drop in FVC (liters) of at least 10% from the upright to the supine position;

patient must have proximal muscle weakness in the lower limbs based on unilateral QMT of the knee extensors defined as < 80% of the predicted value based on age, gender and body size

patient must be able to tolerate pulmonary function testing (PFT) and muscle testing in the supine position;

patient must have testable muscle in bilateral knee flexors and knee extensors, and testable muscle in bilateral elbow flexors and elbow extensors;

patient must be able to provide reproducible muscle and pulmonary function test results;

patient (and patient’s legal guardian if patient is < 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 (urine) 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:

patient requires the use of invasive ventilatory support;

patient requires the use of noninvasive ventilatory support while awake and in an upright position;

patient has received enzyme replacement therapy with GAA from any source;

patient has used an investigational product within 30 days prior to study enrollment, or is currently enrolled in another study which involves clinical evaluations, unless prior approval is given by Genzyme;

patient has a major congenital anomaly, medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities;

Location Information

Please refer to this study by ClinicalTrials.gov identifier NCT00158600
Medical Information 800-745-4447 medinfo@genzyme.com
Medical Information 617-252-7832 medinfo@genzyme.com

California
Tower Hematology Oncology Medical Group, Beverly Hills, California, 90211, United States; Recruiting

District of Columbia
Children's National Medical Center, Washington, District of Columbia, 20010, United States; Recruiting

Missouri
Washington University Medical Center, St. Louis, Missouri, 63110, United States; Recruiting

New York
Mount Sinai School of Medicine, New York, New York, 10029, United States; Recruiting

Pennsylvania
University of Pittsburgh, Dept. of Neurology, Pittsburgh, Pennsylvania, 15213, United States; Recruiting

France
Groupe Hospitalier Pitie-Salpetriere, Paris, 75651, France; Recruiting

Netherlands
Erasmus Medical Centre Rotterdam, Rotterdam, 3015 GD, Netherlands; Recruiting
Sophia Children's Hospital, Erasmus MC, Rotterdam, 3000 CB, Netherlands; Recruiting

More Information

Myozyme® FDA-approved labeling information

Study ID Numbers: AGLU02704
ClinicalTrials.gov Identifier: NCT00158600
Health Authority: United States: Food and Drug Administration