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Clinical trial

Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency

NCT02999984 is registered in ClinicalTrials.gov with University of California, Los Angeles as lead sponsor.

NCT NCT02999984
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Registry record

As filed in ClinicalTrials.gov.

Title
Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency
Lead sponsor
University of California, Los Angeles
Phase
Phase 1/2
Overall status
Completed
Start date
December 16, 2016
Completion date
September 26, 2019
Enrollment
10 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

severe combined immunodeficiency due to ada deficiency