# Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

> **NCT02999984** · PHASE1,PHASE2 · COMPLETED · sponsor: **University of California, Los Angeles** · enrollment: 10 (actual)

## Conditions studied

- Severe Combined Immunodeficiency Due to ADA Deficiency

## Interventions

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

## Key facts

- **NCT ID:** NCT02999984
- **Lead sponsor:** University of California, Los Angeles
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2016-12-16
- **Primary completion:** 2018-10-11
- **Final completion:** 2019-09-26
- **Target enrollment:** 10 (ACTUAL)
- **Last updated:** 2022-08-03

## Collaborators

- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02999984

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02999984, "Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID". Retrieved via AI Analytics 2026-06-03 from https://api.ai-analytics.org/clinical/NCT02999984. Licensed CC0.

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