Late-Onset Treatment Study Extension Protocol

NCT ID: NCT00455195

Last Updated: 2014-03-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 81 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-03-31
• Study Completion Date: 2008-11-30

Brief Summary

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).

Conditions

Pompe Disease (Late-Onset); Glycogen Storage Disease Type II (GSD-II); Glycogenesis Type II; Acid Maltase Deficiency (AMD)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Alglucosidase Alfa/Alglucosidase Alfa

Participants who received alglucosidase alfa during the double-blind study and, if they completed the double-blind study, continued that treatment during the extension study. Participants received an intravenous (IV) infusion of 20 mg/kg of alglucosidase alfa every other week (qow) until their participation in both the AGLU02704 (NCT00158600) and AGLU03206 studies combined equaled a minimum of 104 weeks.

Group Type: EXPERIMENTAL

alglucosidase alfa

Intervention Type: BIOLOGICAL

IV infusion of 20 mg/kg; every other week (qow)

Placebo/Alglucosidase Alfa

Participants given placebo during the double-blind study, completed the double-blind study (study AGLU02704, NCT00158600), and qualified to continue into the extension study on alglucosidase alfa. Participants received an intravenous (IV) infusion of 20 mg/kg of alglucosidase alfa every other week (qow) for up to 52 weeks. Only the alglucosidase alfa treatment experience is included in this extension study.

Group Type: EXPERIMENTAL

alglucosidase alfa

Intervention Type: BIOLOGICAL

IV infusion of 20 mg/kg; every other week (qow)

Interventions

alglucosidase alfa

IV infusion of 20 mg/kg; every other week (qow)

Intervention Type: BIOLOGICAL

Other Intervention Names

Myozyme; Lumizyme

Eligibility Criteria

Inclusion Criteria

• 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.)

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 alglucosidase alfa.

• Minimum Eligible Age: 8 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

Anchorage, Alaska, United States

Phoenix, Arizona, United States

Beverly Hills, California, United States

San Diego, California, United States

Washington D.C., District of Columbia, United States

Coral Springs, Florida, United States

Gainesville, Florida, United States

Sarasota, Florida, United States

Chicago, Illinois, United States

Kansas City, Kansas, United States

New Orleans, Louisiana, United States

Boston, Massachusetts, United States

Omaha, Nebraska, United States

New York, New York, United States

Durham, North Carolina, United States

Cincinnati, Ohio, United States

Toledo, Ohio, United States

Tulsa, Oklahoma, United States

Portland, Oregon, United States

Philadelphia, Pennsylvania, United States

Pittsburgh, Pennsylvania, United States

Greenville, South Carolina, United States

Dallas, Texas, United States

San Antonio, Texas, United States

Seattle, Washington, United States

Green Bay, Wisconsin, United States

Westmead, , Australia

Calgary, Alberta, Canada

Sherbrooke, Quebec, Canada

Paris, , France

Rotterdam, , Netherlands

Countries

United States; Australia; Canada; France; Netherlands

Other Identifiers

2006-003644-31

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

AGLU03206

Identifier Type: -

Identifier Source: org_study_id