An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704

• Conditions: Glycogen Storage Disease type II (Pompe's disease); MedDRA version: 9.1Level: LLTClassification code 10036143Term: Pompe's disease

• Registration Number: EUCTR2006-003644-31-DE
• Lead Sponsor: Genzyme Europe B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2007-07-11
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 82

Inclusion Criteria

1. The patient must have completed Protocol AGLU02704, A Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease”, OR for patients who reside in a region where Myozyme is not available through government reimbursement or charitable access mechanisms, the patient must have completed a minimum of 52 weeks in Protocol AGLU02704;
2. The patient must provide signed, informed consent prior to performing any study-related procedures. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years of age. If the patient is under 18 years of age and can understand the written informed consent, signature will be required from both the patient and the authorized guardian(s);
3. The patient (and patient’s legal guardian if patient is under 18 years of age) must have the ability to comply with the clinical protocol;
4. A female patient of childbearing potential must have a negative pregnancy test (urine beta-human chorionic gonadotropin [ß-hCG]) at Baseline. Note: All female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified