Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
Part of paid clinical trials in Los Angeles, California.
- Sponsor
- University of California, Los Angeles
- Study ID
- NCT05432310
- Phase
- PHASE1/PHASE2
- Status
- Recruiting
Conditions
- Adenosine Deaminase Severe Combined Immune Deficiency
Eligibility Criteria
- Sex
- ALL
- Age
- 1 Month - N/A
- Healthy Volunteers
- Not accepted
Interventions
- 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 — COMBINATION_PRODUCTAutologous transplantation of EFS-ADA lentiviral vector transduced, mPB CD34+ cells by central venous infusion, following reduced intensity conditioning with busulfan
Study Details
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.
Key Dates
- Start date
- Jan 4, 2023
- Status verified
- Apr 2026
- Primary completion
- Nov 14, 2027
- Completion
- Dec 31, 2027
Study Design
- Enrollment
- 20 participants (estimated)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: Autologous mobilized peripheral blood (mPB) transduced with EFS ADA lentiviral vectorEvaluate safety and efficacy of this autologous gene therapy
Primary Outcome Measure
Survival [ Time Frame: 24 months ]
Central Contacts
- Satiro De Oliveira, MD1-310-825-6708
- Augustine Fernandes, PhD1-310-267-4948
Locations (1)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| University of California, Los Angeles (UCLA) | Los Angeles | California | 90095 |
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