Late-Onset Treatment Study Extension Protocol

Genzyme

Status and phase: Completed; Phase 4

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

Treatments: Biological: alglucosidase alfa

Study type: Interventional

Funder types: Industry

Identifiers

NCT00455195

AGLU03206

2006-003644-31 (EudraCT Number)

Details and patient eligibility

About

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).

Enrollment: 81 patients

Sex: All

Ages: 8+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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81 participants in 2 patient groups

Alglucosidase Alfa/Alglucosidase Alfa

Experimental group

Description:

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.

Treatment:

Biological: alglucosidase alfa

Placebo/Alglucosidase Alfa

Experimental group

Description:

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.

Treatment:

Biological: alglucosidase alfa