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
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.
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.
Genzyme, a Sanofi Company
- Drug: Avalglucosidase alfa (GZ402666)
- Drug: Alglucosidase alfa (GZ419829)
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.
- 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