Long-Term Follow-Up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)

This is a long-term follow-up study of participants in the RB001-101 gene replacement therapy clinical trial for Children With SHANK3-related Phelan-McDermid Syndrome (PMS). Participants will roll over from the parent study into this long-term study for continuous safety monitoring and preliminary efficacy assessment for up to 5 years.

NCT Number	NCT07690527
Lead Sponsor	Peking University First Hospital Collaborators: Shenzhen Reborngene Therapeutics Co., Ltd.
Conditions	SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Enrollment	8 participants
Start Date	2026-08-01
Primary Completion	2031-07-01 (estimated)
Study Completion	2031-12-01 (estimated)
Updated on ClinicalTrials.gov	2026-07-13