Late-Onset Treatment Study Extension Protocol

Completed · Phase 4 · Has a placebo group

Conditions studied: Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)

In brief

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 alglucosidase alfa treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704 (NCT00158600).

Key facts

Study ID

NCT00455195

Run by

Genzyme, a Sanofi Company

People needed

81

Starts

2007-03-01

Expected to finish

2008-11-01

Last updated by the study team

2014-03-07

Who can join

Age: 8 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patient must have completed protocol AGLU02704 (NCT00158600)
• 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.)

You may not qualify if…

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

Where it is running

• Study site — Anchorage, Alaska, United States
• Study site — Phoenix, Arizona, United States
• Study site — Beverly Hills, California, United States
• Study site — San Diego, California, United States
• Study site — Washington D.C., District of Columbia, United States
• Study site — Coral Springs, Florida, United States
• Study site — Gainesville, Florida, United States
• Study site — Sarasota, Florida, United States
• Study site — Chicago, Illinois, United States
• Study site — Kansas City, Kansas, United States
• Study site — New Orleans, Louisiana, United States
• Study site — Boston, Massachusetts, United States
• Study site — Omaha, Nebraska, United States
• Study site — New York, New York, United States
• Study site — Durham, North Carolina, United States
• Study site — Cincinnati, Ohio, United States
• Study site — Toledo, Ohio, United States
• Study site — Tulsa, Oklahoma, United States
• Study site — Portland, Oregon, United States
• Study site — Philadelphia, Pennsylvania, United States
• Study site — Pittsburgh, Pennsylvania, United States
• Study site — Greenville, South Carolina, United States
• Study site — Dallas, Texas, United States
• Study site — San Antonio, Texas, United States
• Study site — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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