CompletedPHASE1, PHASE2NCT01852071

Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

Studying Severe combined immunodeficiency due to adenosine deaminase deficiency

Last synced from ClinicalTrials.gov May 3, 2026

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Key facts

Sponsor: University of California, Los Angeles
Principal Investigator: Donald B Kohn, MD, D.D.S
University of California, Los Angeles
Intervention: Infusion of autologous EFS-ADA LV CD34+ (OTL-101)(genetic)
Enrollment: 46 enrolled
Eligibility: 17 years · All sexes
Timeline: 2013 – 2018

Study locations (2)

Mattel Children's Hospital, UCLA, Los Angeles, California, United States
Mark O. Hatfield Clinical Research Center, NIH, Bethesda, Maryland, United States

Collaborators

National Institute of Allergy and Infectious Diseases (NIAID) · National Human Genome Research Institute (NHGRI) · National Heart, Lung, and Blood Institute (NHLBI) · Orchard Therapeutics

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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