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.
Follow this condition — get notified about new trialsBroader categories
-
One-Time gene fix could rescue 'Bubble Boy' disease
Disease control Recruiting nowThis study tests a gene therapy given by IV for ADA-SCID, a rare and life-threatening immune disorder. The treatment uses a modified virus to deliver a working gene, aiming to restore the immune system. Ten participants will be monitored for safety and immune recovery over one ye…
Phase: NA • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
-
Bubble boy disease: gene therapy trial offers hope for immune system repair
Disease control Recruiting nowThis 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: PHASE1, PHASE2 • Sponsor: University of California, Los Angeles • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
-
Scientists launch major study to unravel mysterious metabolism disorders
Knowledge-focused Recruiting nowThis study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Aug 12, 2026 00:00 UTC