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 (COMET)

Genzyme

Status and phase: Completed; Phase 3

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

Treatments: Drug: Alglucosidase alfa (GZ419829); Drug: Avalglucosidase alfa (GZ402666)

Study type: Interventional

Funder types: Industry

Identifiers

NCT02782741

U1111-1178-4806 (Other Identifier)

EFC14028

2016-000942-77 (EudraCT Number)

Details and patient eligibility

About

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).

Full description

The duration of the study per participant will be up to approximately 6 years that will consist of a 14-day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period (except for the subgroup of pediatric patients aged 3 to less than (<) 18 years enrolling directly in the open-label long-term follow-up phase), a 240-week open-label treatment period, and a 4-week post-treatment observation period.

Enrollment: 101 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

101 participants in 2 patient groups

avalglucosidase alfa (GZ402666)

Experimental group

Description:

Administered intravenously every 2 weeks

Treatment:

Drug: Avalglucosidase alfa (GZ402666)

alglucosidase alfa (GZ419829)

Active Comparator group

Description:

Administered intravenously every 2 weeks

Treatment:

Drug: Alglucosidase alfa (GZ419829)