# Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

> **NCT02333760** · PHASE1,PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **Genethon** · enrollment: 10 (estimated)

## Conditions studied

- Wiskott-Aldrich Syndrome

## Interventions

- **GENETIC:** Autologous CD34+ cells transduced with WASP lentiviral vector

## Key facts

- **NCT ID:** NCT02333760
- **Lead sponsor:** Genethon
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2014-09
- **Primary completion:** 2032-10
- **Final completion:** 2032-10
- **Target enrollment:** 10 (ESTIMATED)
- **Last updated:** 2021-06-03


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02333760

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02333760, "Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome". Retrieved via AI Analytics 2026-06-03 from https://api.ai-analytics.org/clinical/NCT02333760. Licensed CC0.

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