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RECRUITING
NCT07014020
NA

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

Sponsor: Peking University First Hospital

View on ClinicalTrials.gov

Summary

This is a first in human, 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.

Official title: An Open-label, Single Arm, Dose-Escalation Clinical Study to Evaluating the Safety, Tolerability and Preliminary Efficacy of a Single Intracerebroventricular Injection of RB001 for the Treatment of SHANK3-related Phelan McDermid Syndrome.

Key Details

Gender

All

Age Range

3 Years - 18 Years

Study Type

INTERVENTIONAL

Enrollment

8

Start Date

2025-06-16

Completion Date

2027-12-31

Last Updated

2025-07-30

Healthy Volunteers

No

Interventions

GENETIC

RB001

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

Locations (1)

Peking University First Hospital

Beijing, Beijing Municipality, China