# Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

> **NCT01852071** · PHASE1,PHASE2 · COMPLETED · sponsor: **University of California, Los Angeles** · enrollment: 46 (actual)

## Conditions studied

- ADA-SCID

## Interventions

- **GENETIC:** Infusion of autologous EFS-ADA LV CD34+ (OTL-101)
- **DRUG:** busulfan
- **DRUG:** PEG-ADA ERT

## Key facts

- **NCT ID:** NCT01852071
- **Lead sponsor:** University of California, Los Angeles
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2013-08-02
- **Primary completion:** 2018-08-27
- **Final completion:** 2018-08-27
- **Target enrollment:** 46 (ACTUAL)
- **Last updated:** 2022-08-03

## Collaborators

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## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01852071

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01852071, "Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene". Retrieved via AI Analytics 2026-06-03 from https://api.ai-analytics.org/clinical/NCT01852071. Licensed CC0.

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