CompletedPhase 1NCT02022696

Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

Studying Adenosine Deaminase Deficiency

Last synced from ClinicalTrials.gov May 3, 2026

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Key facts

Sponsor: National Human Genome Research Institute (NHGRI)
Principal Investigator: Elizabeth K Garabedian, R.N.
National Human Genome Research Institute (NHGRI)
Intervention: Lentiviral Gene Transfer(genetic)
Enrollment: 1 enrolled
Eligibility: 1-65 years · All sexes
Timeline: 2013 – 2017

Study locations (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States

Collaborators

UCLA@@@Duke University Medical Center · Duke Univ. Medical Center · National Cancer Institute (NCI) · National Institutes of Health Clinical Center (CC)

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.

Open NCT02022696 on ClinicalTrials.gov

Other trials for Adenosine Deaminase Deficiency

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ENROLLING BY INVITATIONNCT04049084
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
University of California, Los Angeles

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