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Long-term Follow-up of RB001 in SHANK3-related Phelan-McDermid Syndrome
NCT07690527Phase 1Enrolling by invitation
- Sponsor
- Peking University First Hospital
- Start
- Aug 2026
- Primary completion
- Jul 2031
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of RB001 in Phelan-McDermid syndrome (PMS), sponsored by Peking University First Hospital. Primary endpoint: Safety and tolerability of a single intracerebroventricular injection of RB001. Started Aug 2026; status: enrolling by invitation.
02
Drugs
RB001
03
Conditions
Phelan-McDermid syndrome (PMS)
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