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

Enrolling by invitationOBSERVATIONAL
Enrollment

70

Participants

Timeline

Start Date

September 26, 2019

Primary Completion Date

August 31, 2035

Study Completion Date

August 31, 2035

Conditions
Adenosine Deaminase DeficiencySevere Combined Immunodeficiency (SCID)
Interventions
BIOLOGICAL

autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)

ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)

Trial Locations (2)

90095

Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center, Los Angeles

WC1N 1EH

UCL Great Ormond Street Institute of Child Health, London

All Listed Sponsors
collaborator

Great Ormond Street Hospital for Children NHS Foundation Trust

OTHER

lead

University of California, Los Angeles

OTHER