Trials / Active Not Recruiting
Active Not RecruitingNCT04093349
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
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
- Status
- Active Not Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 4 (actual)
- Sponsor
- Spark Therapeutics, Inc. · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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.
Conditions
- Pompe Disease
- Pompe Disease (Late-onset)
- Glycogen Storage Disease Type 2
- Glycogen Storage Disease Type II
- LOPD
- Lysosomal Storage Diseases
- Acid Maltase Deficiency
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | SPK-3006 | adeno-associated viral (AAV) vector |
Timeline
- Start date
- 2020-10-01
- Primary completion
- 2032-04-01
- Completion
- 2032-04-01
- First posted
- 2019-09-18
- Last updated
- 2024-11-27
Locations
29 sites across 8 countries: United States, Canada, Denmark, France, Germany, Italy, Netherlands, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04093349. Inclusion in this directory is not an endorsement.