# Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy

> **NCT06851767** · PHASE1,PHASE2 · ENROLLING_BY_INVITATION · sponsor: **National Institute of Allergy and Infectious Diseases (NIAID)** · enrollment: 18 (estimated)

## Conditions studied

- X-linked Severe Combined Immunodeficiency
- X-SCID
- XSCID

## Interventions

- **GENETIC:** Plerixafor
- **DRUG:** Filgrastim
- **DRUG:** Palifermin
- **DRUG:** Busulfan
- **BIOLOGICAL:** Base-edited hematopoietic stem and progenitor cells

## Key facts

- **NCT ID:** NCT06851767
- **Lead sponsor:** National Institute of Allergy and Infectious Diseases (NIAID)
- **Sponsor class:** NIH
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ENROLLING_BY_INVITATION
- **Start date:** 2025-05-09
- **Primary completion:** 2034-12-31
- **Final completion:** 2034-12-31
- **Target enrollment:** 18 (ESTIMATED)
- **Last updated:** 2026-02-17


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT06851767

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT06851767, "Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy". Retrieved via AI Analytics 2026-06-25 from https://api.ai-analytics.org/clinical/NCT06851767. Licensed CC0.

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