- 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
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Showing 1–5
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- 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
- Adenosine Deaminase Severe Combined Immunodeficiency
- Interventions
- elapegademase-lvlr
- Biological
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 0 Months to 65 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 14
- States / cities
- Los Angeles, California • San Diego, California • San Francisco, California + 11 more
- 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
- 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