T-B+ severe combined immunodeficiency due to gamma chain deficiency
MONDO:0010315Severe combined immunodeficiency (SCID) due to gamma chain deficiency, also called SCID-X1, is a form of SCID characterized by severe and recurrent infections, associated with diarrhea and failure to thrive.
Also known as: SCIDX1, T-B+ SCID due to gamma chain deficiency, T-B+ severe combined immunodeficiency due to gamma chain deficiency, T-B+ severe combined immunodeficiency, X-linked, X-linked severe combined immunodeficiency, XSCID, severe combined immunodeficiency, X-linked, X-linked recessive, SCID, X-linked
26 clinical trials for this condition and its sub-types.
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Bubble boy disease gets a gene fix: new trial offers hope
Disease control ENROLLING_BY_INVITATIONThis study tests a new gene therapy for X-linked severe combined immunodeficiency (X-SCID), a rare immune disorder. Doctors take a person's own stem cells, fix a faulty gene using a technique called base editing, and return the cells to the body. The goal is to rebuild the immune…
Phase: PHASE1, PHASE2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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SCID stem cell trial pulled before it began
Disease control TerminatedThis study aimed to see if lower doses of chemotherapy drugs, with or without radiation, could safely prepare children and adults with SCID for a stem cell transplant. The goal was to help the donor cells take hold while avoiding severe side effects. However, the trial was withdr…
Phase: PHASE1, PHASE2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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Gene therapy trial aims to fix 'Bubble Boy' disease in newborns
Disease control TerminatedThis study tests a gene therapy for infants with X-linked severe combined immunodeficiency (SCID-X1), a life-threatening condition where babies lack a working immune system. Researchers take the baby's own bone marrow stem cells, add a normal copy of the faulty gene using a lenti…
Phase: PHASE1, PHASE2 • Sponsor: St. Jude Children's Research Hospital • Aim: Disease control
Last updated Jun 27, 2026 07:56 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