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

Completed · Phase 3

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

In brief

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).

Key facts

Study ID

NCT02782741

Run by

Genzyme, a Sanofi Company

People needed

101

Starts

2016-11-02

Expected to finish

2023-05-31

Last updated by the study team

2024-04-04

Who can join

Age: 3 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Investigational Site Number 8400020 — Los Angeles, California, United States
• Investigational Site Number 8400011 — Orange, California, United States
• Investigational Site Number 8400017 — Stanford, California, United States
• Investigational Site Number 8400016 — Gainesville, Florida, United States
• Investigational Site Number 8400007 — Decatur, Georgia, United States
• Investigational Site Number 8400023 — Chicago, Illinois, United States
• Investigational Site Number 8400002 — Iowa City, Iowa, United States
• Investigational Site Number 8400012 — Kansas City, Kansas, United States
• Investigational Site Number 8400010 — Boston, Massachusetts, United States
• Investigational Site Number 8400001 — Detroit, Michigan, United States
• Investigational Site Number 8400019 — Minneapolis, Minnesota, United States
• Investigational Site Number 8400026 — Great Neck, New York, United States
• Investigational Site Number 8400008 — Valhalla, New York, United States
• Investigational Site Number 8400006 — Durham, North Carolina, United States
• Investigational Site Number 8400009 — Cincinnati, Ohio, United States
• Investigational Site Number 8400014 — Portland, Oregon, United States
• Investigational Site Number 8400025 — Pittsburgh, Pennsylvania, United States
• Investigational Site Number 8400018 — Salt Lake City, Utah, United States
• Investigational Site Number 8400005 — Fairfax, Virginia, United States
• Investigational Site Number 8400024 — Morgantown, West Virginia, United States
• Investigational Site Number 0320001 — Caba, Argentina
• Investigational Site Number 0360001 — Auchenflower, Australia
• Investigational Site Number 0400001 — Vienna, Austria
• Investigational Site Number 0560003 — Brussels, Belgium
• Investigational Site Number 8400015 — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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