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
12 clinical trials for this condition and its sub-types.
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New gene therapy offers hope for older kids and adults with 'bubble boy' disease
Disease control Recruiting nowThis study tests a gene therapy for people aged 2 to 50 with X-linked severe combined immunodeficiency (SCID-X1), a condition that leaves them without a working immune system. Participants receive their own blood stem cells that are genetically corrected in a lab, then infused ba…
Phase: PHASE1, PHASE2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Disease control
Last updated Aug 15, 2026 00:00 UTC
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Bubble boy disease gene therapy trial launches
Disease control Recruiting nowThis trial tests a gene therapy given by IV for X-linked severe combined immunodeficiency (SCID-X1), a rare immune disorder. Ten participants with no matched donor will receive a lentiviral vector to correct the genetic defect. The goal is to restore immune function and improve s…
Phase: PHASE1, PHASE2 • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Gene therapy offers hope for children with SCID-X1
Disease control Recruiting nowThis trial tests a gene therapy for children with X-linked severe combined immunodeficiency (SCID-X1), also known as 'bubble boy' disease. Participants receive a low dose of busulfan followed by an infusion of their own blood stem cells that have been genetically corrected with a…
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Scientists launch major study to track rare immune diseases
Knowledge-focused Recruiting nowThis study is observing up to 3,500 people with immune system problems, including rare genetic conditions like chronic granulomatous disease and severe combined immunodeficiency. Researchers will track how these diseases change over time through blood tests, imaging, and checkups…
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated Aug 15, 2026 00:00 UTC