U.S., June 11 -- ClinicalTrials.gov registry received information related to the study (NCT07014020) titled 'RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)' on May 21.
Brief 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.
Study Start Date: June 01
Study Type: INTERVENTIONAL
Condition:
Phelan-McDermid Syndrome
SHANK3 Haploinsufficiency
Interventi...