NCT05432310View on ClinicalTrials.gov

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

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

20

Participants

Timeline

Start Date

January 4, 2023

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2026

Conditions
Adenosine Deaminase Severe Combined Immune Deficiency
Interventions
COMBINATION_PRODUCT

A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme

Autologous transplantation of EFS-ADA lentiviral vector transduced, mPB CD34+ cells by central venous infusion, following reduced intensity conditioning with busulfan

Trial Locations (1)

90095

RECRUITING

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

All Listed Sponsors
lead

University of California, Los Angeles

OTHER