NCT07014020View on ClinicalTrials.gov

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

NARecruitingINTERVENTIONAL
Enrollment

8

Participants

Timeline

Start Date

June 16, 2025

Primary Completion Date

May 1, 2027

Study Completion Date

December 31, 2027

Conditions
Phelan-McDermid SyndromeSHANK3 Haploinsufficiency
Interventions
GENETIC

RB001

The study will enroll up to 2 cohorts, evaluating a starting dose plus a higher or lower dose

Trial Locations (1)

Unknown

RECRUITING

Peking University First Hospital, Beijing

All Listed Sponsors
collaborator

Shenzhen Reborngene Therapeutics Co., Ltd.

UNKNOWN

collaborator

Shenzhen Institutes of Advanced Technology ,Chinese Academy of Sciences

OTHER

lead

Peking University First Hospital

OTHER

NCT07014020 - RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS) | Biotech Hunter | Biotech Hunter