Severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency
MONDO:0007064A form of SCID characterized by profound lymphopenia and very low immunoglobulin levels of all isotypes resulting in severe and recurrent opportunistic infections.
Also known as: ADA deficiency, ADA-SCID, SCID due to ADA deficiency, SCID due to ADA deficiency, early-onset, SCID due to adenosine deaminase deficiency, adenosine deaminase deficiency, adenosine deaminase deficiency, partial, Autosomal recessive, Somatic mosaicism, adenosine deaminase deficient severe combined immunodeficiency
9 clinical trials for this condition and its sub-types, 6 tagged with Severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →-
Bubble boy disease: gene therapy trial offers hope for immune system repair
Disease control OngoingThis trial tests a gene therapy for children with ADA-SCID, a severe immune disorder often called 'bubble boy disease.' Doctors take the child's own blood stem cells, add a working gene, and return them via infusion. The goal is to restore immune function and improve survival. Th…
Phase 1/2 • Sponsor: University of California, Los Angeles • Aim: Disease control
Last updated Aug 22, 2026 00:00 UTC
-
Gene therapy for 'Bubble Boy' disease under Long-Term watch
Disease control By invitation onlyThis study follows about 50 people with a rare immune disorder called ADA-SCID who have received a gene therapy treatment called Strimvelis. The goal is to track their health for many years to see if the treatment remains safe and effective. Researchers will monitor for side effe…
Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jun 27, 2026 12:36 UTC
-
Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
-
Gene therapy survivors monitored for decades in new study
Knowledge-focused By invitation onlyThis study follows 70 people who previously received gene therapy for a rare immune disorder called ADA-SCID. Researchers want to see how well the treatment worked over time and check for any long-term side effects. No new treatment is given—just regular health checkups.
Sponsor: University of California, Los Angeles • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC