Clinical trial
A gene transfer study for late-onset Pompe disease (RESOLUTE)
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
ClinicalTrials.gov ID: NCT04093349
Sponsor: Spark Therapeutics, Inc.
Information provided by: Spark Therapeutics, Inc. (Responsible Party)
Last Update Posted: 2023-11-01
Brief 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.
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
Intervention / Treatment:
- Genetic: SPK-3006
| Category | Value |
|---|---|
| Study Start (Actual) |
2020-10-01
|
| Primary Completion (Estimated) |
2032-04-01
|
| Study Completion (Estimated) |
2032-04-01
|
| Enrollment (Actual) | 4 |
| Study Type | Interventional |
| Phase | Phase 1 Phase 2 |
| Other Study ID Numbers |
SPK-3006-101
2019-001283-30 (EudraCT Number) |