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NCT07014020
RB001 Gene Therapy Study in Children With SHANK3-related Phelan-McDermid Syndrome (PMS)
Conditions: Phelan-McDermid Syndrome, SHANK3 Haploinsufficiency
Sex: All
Ages: 3 Years – 18 Years
Healthy volunteers: No
Phase: PHASE1
Enrollment: 8
Sponsor: Peking University First Hospital
Location: Peking University First Hospital Beijing Beijing Municipality
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.
Eligibility Criteria
Inclusion Criteria:
* Age ≥3 years and \
Source: ClinicalTrials.gov (NCT07014020). StuddyBuddy aggregates publicly available trial information.
