# Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

> **NCT01306019** · PHASE1,PHASE2 · RECRUITING · sponsor: **National Institute of Allergy and Infectious Diseases (NIAID)** · enrollment: 40 (estimated)

## Conditions studied

- X-linked Severe Combined Immunodeficiency (XSCID)

## Interventions

- **BIOLOGICAL:** Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector
- **DRUG:** Busulfan
- **DRUG:** Palifermin

## Key facts

- **NCT ID:** NCT01306019
- **Lead sponsor:** National Institute of Allergy and Infectious Diseases (NIAID)
- **Sponsor class:** NIH
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2012-09-25
- **Primary completion:** 2032-12-31
- **Final completion:** 2032-12-31
- **Target enrollment:** 40 (ESTIMATED)
- **Last updated:** 2026-05-01


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01306019

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01306019, "Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)". Retrieved via AI Analytics 2026-07-01 from https://api.ai-analytics.org/clinical/NCT01306019. Licensed CC0.

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