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NCT07014020
RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)
NA trial testing RB001 in Phelan-McDermid Syndrome in 8 participants. Currently enrolling.
1 May 2027
Quick facts
| Lead sponsor | Peking University First Hospital |
|---|---|
| Phase | NA |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 8 |
| Start date | 16 June 2025 |
| Primary completion | 1 May 2027 |
| Estimated completion | 31 December 2027 |
| Sites | 1 location across China |
Drugs / interventions tested
- RB001
Conditions studied
- Phelan-McDermid Syndrome — all drugs for Phelan-McDermid Syndrome →
- SHANK3 Haploinsufficiency — all drugs for SHANK3 Haploinsufficiency →
Sponsor
Peking University First Hospital
Who can join
Adults 3 to 18, any sex, with Phelan-McDermid Syndrome or SHANK3 Haploinsufficiency. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT07014020
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Phelan-McDermid Syndrome
Currently open trials in the same condition.
- NCT07281079 — A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome · Phase 3 · recruiting
Other Peking University First Hospital trials
Trials by the same sponsor.
- NCT07498907 — Disitamab Vedotin Plus Radiotherapy for Adjuvant Treatment of HER2-Expressing Cisplatin-Ineligible Upper Tract Urothelia · Phase 2 · not yet recruiting
- NCT07517276 — TUDCA in High-Risk Lactating Mothers Identified by Early Postpartum Milk Hydrophobicity Index · Phase 2 · not yet recruiting
- NCT07504939 — Multimodal Kidney-Sparing Strategy for High-Risk Upper Tract Urothelial Carcinoma · not yet recruiting
- NCT07464119 — A Clinical Study of iPSC-Derived Islet Cells for Diabetes Mellitus Therapy · NA · not yet recruiting
- NCT07460843 — Comparison of TB+PB and TB+6SB for Prostate Cancer Diagnosis · NA · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT07014020 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Peking University First Hospital
- Last refreshed: 30 July 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT07014020.
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