A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease
- Conditions
- acid alfa glucosidase deficiencyPompe Disease10021605
- Registration Number
- NL-OMON46174
- Lead Sponsor
- Sanofi-aventis
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
-The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations
-The patient and/or their parent/legal guardian is willing and able to provide signed informedconsent, and the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
-The patient (and patient*s legal guardian if patient is <18 years of age) must have the ability to
comply with the clinical protocol.
-The patient, if female and of childbearing potential, must have a negative pregnancy test (betahuman
chorionic gonadotropin) at baseline.
-The patient is younger than 3 years of age.;-The patient has known Pompe specific cardiac hypertrophy.;-The patient is wheelchair dependent.;-The patient is not able to ambulate 40 meters (approximately 130 feet) without topping and without an assistive device.;-The patient requires invasive-ventilation (non-invasive ventilation is allowed).;-The patient is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of >=40% predicted and <=85% predicted.;-The patient has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease.;-The patient has prior or current use of immune tolerance induction therapy.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Change in FVC % predicted in the upright position from baseline to<br /><br>12 months.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Change in the following parameters from baseline to 12 months:<br /><br><br /><br>Efficacy:<br /><br>• MIP and MEP (% predicted)<br /><br>• 6MWT distance walked<br /><br>• Motor function (QMFT)<br /><br>• Muscle strength (HHD)<br /><br>• Health-related quality of life (SF-12)</p><br>