drugset / Trial / NCT04093349
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
NaSequentialOpen-labelTreatment
Summary
The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.
Timeline
- Start
- 2020-10-01
- Primary completion
- 2032-04
- Completion
- 2032-04
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | VANGLUSAGENE ENSIPARVOVEC | Gene therapy (AAV / viral vector) | — | Intravenous |