Enrolling by invitationNCT04049084

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

Studying 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, M.D.
University of Califorina, Los Angeles
Intervention: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)(biological)
Enrollment: 70 enrolled
Eligibility: All sexes
Timeline: 2019 – 2040

Study locations (2)

Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center, Los Angeles, California, United States
UCL Great Ormond Street Institute of Child Health, London, United Kingdom

Collaborators

Great Ormond Street Hospital for Children NHS Foundation Trust

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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