Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa in Patients With Late-onset Pompe Disease
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To determine the effect of neoGAA (GZ402666) treatment on respiratory muscle strength measured by percent predicted forced vital capacity (% FVC) in the upright position, as compared to alglucosidase alfa.
To determine the safety and effect of neoGAA treatment on functional endurance (6-minute walk test (6MWT), inspiratory muscle strength (maximum inspiratory pressure (MIP)), expiratory muscle strength (maximum expiratory pressure (MEP)), lower extremity muscle strength (hand-held dynamometry (HHD)), motor function (Quick Motor Function Test (QMFT)), and health-related quality of life (SF-12).
The duration of the study per patient will be up to 3 years that will consist of a 14- day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period, a 96-week open-label treatment period, and a 4-week post-treatment observation period.
Are You a Candidate?
- The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations
- 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 legally minor patients as defined by local regulation. If the patient is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from patients, if applicable
- The patient (and patient’s legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol
- The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline
- The patient is less than three 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 stopping 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 ≥30% 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
The above information is not intended to contain all considerations relevant to a patient’s potential participation in a clinical trial.