drugset / Trial / NCT07014020
RB001 Gene Therapy Study in Children With SHANK3-related Phelan-McDermid Syndrome (PMS)
Phase 1
Active not recruiting
8 enrolled
Peking University First Hospital
Shenzhen Institutes of Advanced Technology ,Chinese Academy of Sciences · collabShenzhen Reborngene Therapeutics Co., Ltd. · collab
NaSingle-groupOpen-labelTreatment
Summary
This is a first in human, open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of RB001 administered via intracerebroventricular (ICV) injection in pediatric with SHANK3 related Phelan-McDermid Syndrome. Clinical data will be evaluated for safety, tolerability, and preliminary efficacy of RB001 in participants with SHANK3 related PMS.
Timeline
- Start
- 2025-06-16
- Primary completion
- 2027-05-01
- Completion
- 2027-12-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | RB001 | Gene therapy (AAV / viral vector) | — | Other |