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Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
Not Recruiting
Trial ID: NCT02782741
Purpose
Primary Objective:
To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as compared to alglucosidase alfa.
Secondary Objective:
To determine the safety and effect of avalglucosidase alfa treatment on functional endurance (6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held dynamometry), motor function (Quick Motor Function Test), and health-related quality of life (Short Form-12).
Official Title
A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease
Stanford Investigator(s)
John W. Day, MD, PhD
Professor of Neurology and Neurological Sciences (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology
Eligibility
Inclusion criteria :
* The participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations.
* The participant 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 participant as defined by local regulation. If the participant is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from participants, if applicable.
Exclusion criteria:
* The participant is \<3 years of age.
* The participant has known Pompe specific cardiac hypertrophy.
* The participant is wheelchair dependent.
* The participant is not able to ambulate 40 meters (approximately 130 feet) without stopping and without an assistive device.
* The participant requires invasive-ventilation (non-invasive ventilation is allowed).
* The participant is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of greater than or equal to 30% predicted and less than or equal to 85% predicted.
* The participant (and participant's legal guardian if participant is legally minor as defined by local regulation) is (are) not able to comply with the clinical protocol.
* The participant has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease.
* The participant has prior or current use of immune tolerance induction therapy.
* The participant, if female and of childbearing potential, has a positive pregnancy test (beta-human chorionic gonadotropin) at baseline.
The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Intervention(s):
drug: Avalglucosidase alfa (GZ402666)
drug: Alglucosidase alfa (GZ419829)
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305