Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults with Late-onset Pompe Disease
The purpose of this study is to evaluate the safety, tolerability, and efficacy of SPK-3006, which is a novel investigational gene therapy for the potential treatment of patients with clinically moderate, late-onset Pompe disease. Subjects must be on a stable dosing regimen of enzyme replacement therapy (ERT) for at least 24 months prior to enrollment.
SPK-3006 is administered via a single intravenous infusion during an overnight inpatient hospital stay. In addition to the study drug infusion, participants will complete follow-up visits to assess clinical and safety outcomes twice a week for the first 11 weeks, then twice a month for 2 months, then about once a month for the remainder of participation. The trial may last approximately 66 weeks from the time of screening (which can be up to 14 weeks), the dosing with SPK-3006 (one day), and a follow-up period of about 52 weeks.
Principal Investigator: Shafeeq Ladha, MD