# Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

> **NCT03315078** · PHASE1,PHASE2 · UNKNOWN · sponsor: **National Institute of Allergy and Infectious Diseases (NIAID)** · enrollment: 13 (estimated)

## Conditions studied

- X-Linked Combined Immunodeficiency Diseases

## Interventions

- **BIOLOGICAL:** CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT
- **DRUG:** Palifermin
- **DRUG:** Busulfan

## Key facts

- **NCT ID:** NCT03315078
- **Lead sponsor:** National Institute of Allergy and Infectious Diseases (NIAID)
- **Sponsor class:** NIH
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** UNKNOWN
- **Start date:** 2012-04
- **Primary completion:** 2022-12
- **Final completion:** 2022-12
- **Target enrollment:** 13 (ESTIMATED)
- **Last updated:** 2019-12-04


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03315078

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03315078, "Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency". Retrieved via AI Analytics 2026-05-31 from https://api.ai-analytics.org/clinical/NCT03315078. Licensed CC0.

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