Trial Search Results

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

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).

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Genzyme, a Sanofi Company

Stanford Investigator(s):


  • Drug: Avalglucosidase alfa (GZ402666)
  • Drug: Alglucosidase alfa (GZ419829)


Phase 3


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.

Ages Eligible for Study

3 Years - N/A

Genders Eligible for Study


Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Not Recruiting