- Conditions
- Severe Combined Immunodeficiency Due to ADA Deficiency
- Interventions
- OTL-101
- Biological
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 30 Days to 17 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Los Angeles, California
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Showing 1–4
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- Conditions
- Severe Combined Immunodeficiency Due to ADA Deficiency
- Interventions
- Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT
- Genetic · Drug
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 30 Days to 17 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- ADA-SCID
- Interventions
- Infusion of autologous EFS-ADA LV CD34+ (OTL-101), busulfan, PEG-ADA ERT
- Genetic · Drug
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 1 Month to 17 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Bethesda, Maryland
- Conditions
- Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)
- Interventions
- autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
- Biological
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- Not listed
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2040
- U.S. locations
- 1
- States / cities
- Los Angeles, California