# Gene Therapy for Wiskott-Aldrich Syndrome (WAS)

> **NCT01347242** · PHASE1,PHASE2 · COMPLETED · sponsor: **Genethon** · enrollment: 6 (actual)

## Conditions studied

- Wiskott-Aldrich Syndrome

## Interventions

- **GENETIC:** Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene

## Key facts

- **NCT ID:** NCT01347242
- **Lead sponsor:** Genethon
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2011-03
- **Primary completion:** 2019-11-13
- **Final completion:** 2019-11-13
- **Target enrollment:** 6 (ACTUAL)
- **Last updated:** 2021-06-03

## Collaborators

- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01347242

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01347242, "Gene Therapy for Wiskott-Aldrich Syndrome (WAS)". Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/clinical/NCT01347242. Licensed CC0.

---

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