- Conditions
- Adenosine Deaminase Severe Combined Immune Deficiency
- 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 Product
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 1 Month and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Los Angeles, California
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Showing 1–11
of 11
matching trials from the live ClinicalTrials.gov search.
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- 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
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- ADA PBSC, ADA Umbilical Cord Blood Cells, Transduced Lymphocytes
- Drug
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1990 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis, ADA SCID, XSCID
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- Not listed
- Enrollment
- 690 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2028
- U.S. locations
- 39
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 34 more
- 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
- Severe Combined Immunodeficiency
- Interventions
- ADA gene transfer
- Biological
- Lead sponsor
- Donald B. Kohn, M.D.
- Other
- Eligibility
- 1 Month to 18 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2015
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Adenosine Deaminase Deficiency, ADA-SCID
- Interventions
- Lentiviral Gene Transfer
- Genetic
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Year to 65 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- SCID, ADA-SCID, XSCID, Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- Not listed
- Enrollment
- 1,007 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2023
- U.S. locations
- 31
- States / cities
- Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 26 more
- Conditions
- Severe Combined Immunodeficiency Syndrome
- Interventions
- CD34+ cells transduced with ADA retrovir
- Drug
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2014
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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