A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
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.
Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 30 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date: October 1, 2020
Estimated Primary Completion Date: October 2023
Estimated Study Completion Date: October 2027
Arm:
- Experimental: SPK-3006
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Estimated enrolment | 30 |
| Actual Study start date | 01 October 2020 |
| Estimated study completion date | 01 October 2027 |