NCT04049084
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
Enrolling by Invitation
Not specifiedAll AgesObservationalUniversity of California, Los AngelesInvestigator-initiated
~70 participants
Updated 2025-12-18 on ClinicalTrials.gov
What's tested:autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
At a glance
Recruiting sites
0 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Overall Survival
Measured over 15 years post-treatment
+2 more outcomes measured
Conditions
Where it's being run
2 sites across 2 statesCalifornia1
United Kingdom1
Study leadership
- Donald B. Kohn, M.D. · STUDY_DIRECTOR · University of Califorina, Los Angeles
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
What this trial measures
- Overall Survival15 years post-treatment
Establishing efficacy through overall survival
- Event-Free Survival15 years post-treatment
Establishing efficacy through event-free survival
- Incidence of Adverse Events15 years post-treatment
Long-Term Safety incidence of AEs and SAEs