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


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
John W. Day, MD, PhD

Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology


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.


drug: Avalglucosidase alfa (GZ402666)

drug: Alglucosidase alfa (GZ419829)

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305