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

NCT07014020 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2025-07-30

No results posted yet for this study

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.

Conditions

  • Phelan-McDermid Syndrome
  • SHANK3 Haploinsufficiency

Interventions

GENETIC

RB001

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

Sponsors & Collaborators

  • Shenzhen Reborngene Therapeutics Co., Ltd.

    collaborator UNKNOWN

  • Shenzhen Institutes of Advanced Technology ,Chinese Academy of Sciences

    collaborator OTHER

  • Peking University First Hospital

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-16
Primary Completion
2027-05-01
Completion
2027-12-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07014020 on ClinicalTrials.gov