Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease



Status:Recruiting
Conditions:Diabetes
Therapuetic Areas:Endocrinology
Healthy:No
Age Range:3 - Any
Updated:3/28/2019
Start Date:October 19, 2016
End Date:September 24, 2024
Contact:Trial Transparency email recommended (Toll free number for US & Canada)
Email:Contact-Us@sanofi.com
Phone:800-633-1610

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A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatment-naïve Patients With Late-onset Pompe Disease

Primary Objective:

To determine the effect of neoGAA (GZ402666) 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 neoGAA treatment on functional endurance (6-minute walk
test (6MWT), inspiratory muscle strength (maximum inspiratory pressure (MIP)), expiratory
muscle strength (maximum expiratory pressure (MEP)), lower extremity muscle strength
(hand-held dynamometry (HHD)), motor function (Quick Motor Function Test (QMFT)), and
health-related quality of life (SF-12).

The duration of the study per patient will be up to 3 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, a 96-week open-label treatment period, and a 4-week post-treatment
observation period.

Inclusion criteria :

- The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2
confirmed GAA gene mutations.

- The patient 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 patients as defined by local regulation. If the patient is legally
minor, signed written consent shall be obtained from parent(s)/legal guardian and
assent obtained from patients, if applicable.

- The patient (and patient's legal guardian if patient is legally minor as defined by
local regulation) must have the ability to comply with the clinical protocol.

- The patient, if female and of childbearing potential, must have a negative pregnancy
test (beta-human chorionic gonadotropin) at baseline.

Exclusion criteria:

- The patient is <3 years of age.

- The patient has known Pompe specific cardiac hypertrophy.

- The patient is wheelchair dependent.

- The patient is not able to ambulate 40 meters (approximately 130 feet) without
stopping and without an assistive device.

- The patient requires invasive-ventilation (non-invasive ventilation is allowed).

- The patient is not able to successfully perform repeated forced vital capacity (FVC)
measurements in upright position of ≥30% predicted and ≤85% predicted.

- The patient has had previous treatment with alglucosidase alfa or any investigational
therapy for Pompe disease.

- The patient has prior or current use of immune tolerance induction therapy

The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial.
We found this trial at
25
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