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Study identifier: NCT05432310 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

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

Condition: Adenosine Deaminase Severe Combined Immune Deficiency  ·  Sponsor: University of California, Los Angeles

PhasePhase 1/Phase 2
Planned participants20
Who can joinAll sexes, 1 Month to no upper limit
Healthy volunteersNo

About this study

The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.

This description comes directly from the study's public registry record.

Talk to the study team

Satiro De Oliveira, MD  ·  1-310-825-6708  ·  sdeoliveira@mednet.ucla.edu

Augustine Fernandes, PhD  ·  1-310-267-4948  ·  afernandes@mednet.ucla.edu

Always discuss trial participation with your own doctor first.

Locations (1)

University of California, Los Angeles (UCLA)Los Angeles, California, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT05432310