# Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

> **NCT05432310** · PHASE1,PHASE2 · RECRUITING · sponsor: **University of California, Los Angeles** · enrollment: 20 (estimated)

## Conditions studied

- Adenosine Deaminase Severe Combined Immune Deficiency

## Interventions

- **COMBINATION_PRODUCT:** A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme

## Key facts

- **NCT ID:** NCT05432310
- **Lead sponsor:** University of California, Los Angeles
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2023-01-04
- **Primary completion:** 2027-11-14
- **Final completion:** 2027-12-31
- **Target enrollment:** 20 (ESTIMATED)
- **Last updated:** 2026-04-07


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05432310

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05432310, "Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector". Retrieved via AI Analytics 2026-07-15 from https://api.ai-analytics.org/clinical/NCT05432310. Licensed CC0.

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