Immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome
MONDO:0010580Immunodysregulation - polyendocrinopathy - enteropathy - X-linked (IPEX) syndrome is a severe congenital systemic autoimmune disease characterized by refractory diarrhea, endocrinopathies, cutaneous involvement, and infections.
Also known as: DMSD, IDDM-secretory diarrhea syndrome, IDDM-secretory diarrhoea syndrome, IPEX, X linked polyendocrinopathy, X-linked autoimmunity-allergic dysregulation syndrome, XLAAD, XPID
48 clinical trials for this condition and its sub-types, 10 tagged with Immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome itself.
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Stem cell transplant offers new hope for rare immune diseases
Disease control Recruiting nowThis study tests a stem cell transplant for people with common variable immunodeficiency (CVID) and other immune disorders. The goal is to replace the faulty immune system with a healthy donor's stem cells. Participants are aged 5 to 40 and will receive a reduced-intensity transp…
Phase 2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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New stem cell transplant option for patients without a perfect donor match
Disease control Recruiting nowThis expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…
Early phase 1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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Milder chemo before stem cell transplant shows promise for rare blood disorders
Disease control Recruiting nowThis study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…
Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Milder transplant method aims to help young patients with rare blood diseases
Disease control Recruiting nowThis study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…
Phase 2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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Scientists seek clues to fungal infections in immune system study
Knowledge-focused Recruiting nowThis study aims to understand why some people are more prone to fungal infections, especially from Candida yeast. Researchers will collect blood, saliva, urine, and tissue samples from people with immune disorders, their family members, and healthy volunteers. No treatment is giv…
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated Aug 26, 2026 00:00 UTC