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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
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

NCT02782741

CompletedPhase 3

Sponsor: Genzyme, a Sanofi Company

Conditions: Glycogen Storage Disease Type II;Pompe's Disease

Interventions: Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829)

Countries: United States, Argentina, Australia, Austria, Belgium, Brazil, Canada, Czechia

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).

Eligibility overview

Sex: ALL

Age: 3 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
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.
Locations (69)
  • Phoenix, Arizona, United States
  • Los Angeles, California, United States
  • Orange, California, United States
  • Stanford, California, United States
  • Gainesville, Florida, United States
  • Decatur, Georgia, United States
  • Chicago, Illinois, United States
  • Iowa City, Iowa, United States
  • Kansas City, Kansas, United States
  • Boston, Massachusetts, United States
  • Detroit, Michigan, United States
  • Minneapolis, Minnesota, United States
  • Great Neck, New York, United States
  • Valhalla, New York, United States
  • Durham, North Carolina, United States
  • Cincinnati, Ohio, United States
  • Portland, Oregon, United States
  • Pittsburgh, Pennsylvania, United States
  • Salt Lake City, Utah, United States
  • Fairfax, Virginia, United States
  • Morgantown, West Virginia, United States
  • Caba, Argentina
  • Auchenflower, Australia
  • Vienna, Austria
  • Brussels, Belgium
  • Leuven, Belgium
  • Brasília, Brazil
  • São Paulo, Brazil
  • Hamilton, Canada
  • Montreal, Canada
  • + 39 more on CT.gov