This study examines the gene transfer therapy called SPK-3006 in adults with late-onset Pompe disease. It can enroll male and female patients aged 18 and older who have received enzyme replacement therapy (ERT) for at least 24 months and have clinically moderate late-onset Pompe disease. It is being conducted in Phase 1/Phase 2 and is currently not recruiting but still ongoing (active, not recruiting).
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Provide written informed consent; * Males and Females ≥18 years of age with late-onset Pompe disease; * Received ERT for at least the previous 24 months * Have clinically moderate, late-onset Pompe disease characteristics; * Agree to use reliable contraception. Exclusion Criteria: * Active hepatitis B and/or C; * Significant underlying liver disease; * Human immunodeficiency virus (HIV) infection; * Prior hypersensitivity to rhGAA; * Pre-existing anti-AAV neutralizing antibody titers; * High titer antibody responses to rhGAA; * Requires any invasive ventilation or requires noninvasive ventilation while awake and upright; * Received any prior vector or gene transfer agent; * Active malignancy (except non-melanoma skin cancer); * History of liver cancer; * Pregnant or nursing women; * Any evidence of active infection at the time of SPK-3006 infusion.