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  • Lysosomal Storage Disorders (LSD) Registry Program

    The Lysosomal Storage Disorders (LSD) Registry Program is a multicenter, international, observational program for people with certain rare diseases. It is designed to track the natural history and outcomes of patients. Currently, patients diagnosed with Pompe disease may participate in this registry program.

    No experimental intervention is given as part of this program. As such, participants will be assessed clinically and receive standard-of-care treatment as determined by their physician. Physicians will determine the actual frequency of necessary assessments according to a patient’s individualized need for medical care and routine follow up.

    Principal Investigator: Shafeeq Ladha, MD

    Enrollment Contact
    Jessie Duncan
    (602) 406-1466
    [email protected]

    Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa in 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.

    Are You a Candidate?

    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 less than three 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.

    About Barrow Neurological Institute
    Since our doors opened as a regional specialty center in 1962, we have grown into one of the premier destinations in the world for neurology and neurosurgery. Our experienced, highly skilled, and comprehensive team of neurological specialists can provide you with a complete spectrum of care–from diagnosis through outpatient neurorehabilitation–under one roof. Barrow Neurological Institute: Discover. Educate. Heal.