OMENN SYNDROME
Clinical trials for OMENN SYNDROME explained in plain language.
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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…
Matched conditions: OMENN SYNDROME
Phase: PHASE2 • Sponsor: Center for International Blood and Marrow Transplant Research • Aim: Disease control
Last updated Jul 11, 2026 00:00 UTC
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Double transplant breakthrough offers hope for rare immune disorder patients
Disease control ENROLLING_BY_INVITATIONThis 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…
Matched conditions: OMENN SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC