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

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 21

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 [β-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.

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

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>1. Change from Treatment Baseline to End of Study time point in 6MWT - meters<br /><br>walked<br /><br>2. Change from Treatment Baseline to End of Study time point in % predicted FVC<br /><br>in the upright position</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>1. Change from Treatment Baseline to End of Study time point in QMT Leg Score<br /><br>for % predicted bilateral knee flexors and knee extensors<br /><br>2. Change from Treatment Baseline to End of Study time point in PCS score of<br /><br>the MOS SF-36 Health Survey</p><br>

Related Trials