T-B- severe combined immunodeficiency
MONDO:0017855T-B- severe combined immunodeficiency (SCID) is a group of rare monogenic primary immunodeficiency disorders characterized by a lack of functional peripheral T and B lymphocytes, resulting in recurrent early-onset severe respiratory viral, bacterial or fungal infections, diarrhea and failure to thrive. Hypersensitivity to ionizing radiation is a characteristic feature of some of its sub-types.
Also known as: T-B- SCID
16 clinical trials for this condition and its sub-types, 0 tagged with T-B- severe combined immunodeficiency itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of T-B- severe combined immunodeficiency
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Omenn syndrome 4 trials
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DNA ligase IV deficiency 1 trial
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Reticular dysgenesis 1 trial
1 sub-type
- Immunoerythromyeloid hypoplasia 0 trials
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Cernunnos-XLF deficiency 0 trials
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Neutrophil immunodeficiency syndrome 0 trials
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Can lower chemo doses help babies with SCID build better immunity?
Disease control OngoingThis phase 2 trial studies whether lower doses of the chemotherapy drug busulfan can help infants with severe combined immunodeficiency (SCID) achieve good immune function after a stem cell transplant, while reducing short- and long-term risks. The trial includes 56 babies receiv…
Phase 2 • Sponsor: Center for International Blood and Marrow Transplant Research • Aim: Disease control
Last updated Aug 30, 2026 00:00 UTC
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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
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Gene therapy offers hope for 'Bubble Boy' disease without a donor
Disease control PausedThis trial tests a gene therapy for infants under 2 years old with RAG1-deficient severe combined immunodeficiency (SCID), a life-threatening condition where the immune system barely works. The therapy uses the child's own blood stem cells, modified with a corrected gene, to try …
Phase 1/2 • Sponsor: Videja B.V. • Aim: Disease control
Last updated Jul 18, 2026 00:00 UTC
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Stem cell transplant offers new hope for kids with rare immune diseases
Disease control OngoingThis study tests a donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…
Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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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
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Double transplant breakthrough offers hope for rare immune disorder patients
Disease control By invitation onlyThis study tests a new approach for people aged 5 to 45 with primary immune deficiencies and end-stage lung disease. Participants receive a double lung transplant followed by a stem cell transplant from the same donor. The goal is to see if this combined procedure is safe and can…
Phase 1/2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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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
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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