# An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

> **NCT04049084** · — · ENROLLING_BY_INVITATION · sponsor: **University of California, Los Angeles** · enrollment: 70 (estimated)

## Conditions studied

- Adenosine Deaminase Deficiency
- Severe Combined Immunodeficiency (SCID)

## Interventions

- **BIOLOGICAL:** autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)

## Key facts

- **NCT ID:** NCT04049084
- **Lead sponsor:** University of California, Los Angeles
- **Sponsor class:** OTHER
- **Phase:** —
- **Study type:** OBSERVATIONAL
- **Status:** ENROLLING_BY_INVITATION
- **Start date:** 2019-09-26
- **Primary completion:** 2040-08
- **Final completion:** 2040-08
- **Target enrollment:** 70 (ESTIMATED)
- **Last updated:** 2025-12-18

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04049084

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04049084, "An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID". Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/clinical/NCT04049084. Licensed CC0.

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