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

> **NCT07014020** · NA · RECRUITING · sponsor: **Peking University First Hospital** · enrollment: 8 (estimated)

## Conditions studied

- Phelan-McDermid Syndrome
- SHANK3 Haploinsufficiency

## Interventions

- **GENETIC:** RB001

## Key facts

- **NCT ID:** NCT07014020
- **Lead sponsor:** Peking University First Hospital
- **Sponsor class:** OTHER
- **Phase:** NA
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2025-06-16
- **Primary completion:** 2027-05-01
- **Final completion:** 2027-12-31
- **Target enrollment:** 8 (ESTIMATED)
- **Last updated:** 2025-07-30

## Collaborators

- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07014020

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07014020, "RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT07014020. Licensed CC0.

---

*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*