Glycogen Storage Disease type II (Pompe's disease)
Conditions
Interventions
Trade Name: Myozyme
Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: Alglucosidase alfa
CAS Number: n.a.
Cur
Sponsors
Genzyme Europe BV
Eligibility
Sex/Gender
All
Inclusion criteria
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? no 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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objective of this extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704. The primary objectives of the study are: 1) to evaluate the safety profile of Myozyme; 2) to determine the effect of Myozyme treatment on functional endurance as measured by the Six Minute Walk Test (6MWT); and 3) to determine the effect of Myozyme treatment on respiratory muscle weakness as measured by Forced Vital Capacity (FVC) in the upright position. ;Secondary Objective: Secondary objectives are: 1) to determine the effect of Myozyme treatment on proximal muscle weakness in the lower limbs as measured by Quantitative Muscle Testing (QMT) in bilateral knee flexors (hamstrings) and knee extensors (quadriceps) and 2) to determine the effect of Myozyme treatment on health-related quality of life as measured by the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) SF-36 Health Survey.;Primary end point(s): See objective section | — |
Countries
France, Germany, Netherlands
Outcome results
None listed