# Long-Term Follow-Up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)

> **NCT07690527** · PHASE1 · ENROLLING_BY_INVITATION · sponsor: **Peking University First Hospital** · enrollment: 8 (estimated)

## Conditions studied

- SHANK3 Haploinsufficiency
- Phelan-McDermid Syndrome

## Interventions

- **BIOLOGICAL:** RB001

## Key facts

- **NCT ID:** NCT07690527
- **Lead sponsor:** Peking University First Hospital
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** ENROLLING_BY_INVITATION
- **Start date:** 2026-08-01
- **Primary completion:** 2031-07-01
- **Final completion:** 2031-12-01
- **Target enrollment:** 8 (ESTIMATED)
- **Last updated:** 2026-07-13

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07690527

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07690527, "Long-Term Follow-Up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)". Retrieved via MORVS 2026-10-20 from https://api.morvs.ai/clinical/NCT07690527. Licensed CC0.

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