Clinical Trial

RB001 Gene Therapy Study in Children With SHANK3-related Phelan-McDermid Syndrome (PMS)

Active, Not Recruiting Phase 1
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Summary
This is an 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.
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2025-06-02; most recent amendment 2026-07-09.
Status change: Recruiting → Active, Not Recruiting 2026-06-28
Status change: Not Yet Recruiting → Recruiting 2025-07-29
Trial Details
NCT Number NCT07014020
Lead Sponsor Peking University First Hospital
Collaborators: Shenzhen Reborngene Therapeutics Co., Ltd., Shenzhen Institutes of Advanced Technology ,Chinese Academy of Sciences
Conditions Phelan-McDermid Syndrome, SHANK3 Haploinsufficiency
Enrollment 8 participants
Start Date 2025-06-16
Primary Completion 2027-05-01 (estimated)
Study Completion 2027-12-31 (estimated)
Updated on ClinicalTrials.gov 2026-07-28