RB001

BIOLOGICAL2 trials

Sponsors

Peking University First Hospital

Conditions

Phelan-McDermid SyndromeSHANK3 Haploinsufficiency

Phase 1

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

Active, not recruitingNCT07014020

Peking University First HospitalPhelan-McDermid Syndrome, SHANK3 Haploinsufficiency

Start: 2025-06-16End: 2027-12-31Updated: 2026-07-28

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

Enrolling by invitationNCT07690527

Peking University First HospitalPhelan-McDermid Syndrome, SHANK3 Haploinsufficiency

Start: 2026-08-01End: 2031-12-01Target: 8Updated: 2026-07-13