Inborn error of immunity
MONDO:0003778A disorder in which the immune system is unable to mount an adequate immune response.
Also known as: IEI, inborn errors of immunity, primary immunodeficiency disease, antibody deficiency syndrome, antibody deficiency syndromes, deficiency syndrome, antibody, deficiency syndrome, immunologic, deficiency syndrome, immunological
344 clinical trials for this condition and its sub-types, 121 tagged with Inborn error of immunity itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
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Sub-types of Inborn error of immunity
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Lymphoproliferative syndrome 70 trials · 100 incl. sub-types
8 sub-types
- Castleman disease 8 trials · 18 incl. sub-types Sub-types →
- Autoimmune lymphoproliferative syndrome 11 trials · 12 incl. sub-types Sub-types →
- X-linked lymphoproliferative syndrome 7 trials · 11 incl. sub-types Sub-types →
- Dianzani autoimmune lymphoproliferative disease 0 trials
- Atypical lymphoproliferative disorder 0 trials
- Lymphoproliferative syndrome 1 0 trials
- Lymphoproliferative syndrome 2 0 trials
- Severe combined immunodeficiency due to CD70 deficiency 0 trials
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B cell deficiency 5 trials · 85 incl. sub-types
5 sub-types
- Agammaglobulinemia 16 trials · 52 incl. sub-types Sub-types →
- Hyperimmunoglobulin syndrome 0 trials · 33 incl. sub-types Sub-types →
- Selective immunoglobulin deficiency disease 1 trial · 4 incl. sub-types Sub-types →
- PAX5-related B lymphopenia and autism spectrum disorder 0 trials
- Immunoglobulin beta deficiency 0 trials
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Phagocyte bactericidal dysfunction 0 trials · 37 incl. sub-types
1 sub-type
- Chronic granulomatous disease 37 trials Sub-types →
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Complement deficiency 2 trials · 28 incl. sub-types
8 sub-types
- Atypical hemolytic-uremic syndrome 19 trials Sub-types →
- Classic complement early component deficiency 0 trials · 7 incl. sub-types Sub-types →
- Disorder of lectin complement activation pathway 0 trials · 1 incl. sub-types Sub-types →
- Complement factor I deficiency 0 trials
- Complement receptor deficiency 0 trials
- Immunodeficiency due to a classical component pathway complement deficiency 0 trials Sub-types →
- Immunodeficiency due to a late component of complement deficiency 0 trials Sub-types →
- Recurrent Neisseria infections due to factor D deficiency 0 trials
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Hereditary hemophagocytic lymphohistiocytosis 8 trials · 16 incl. sub-types
11 sub-types
- Chediak-Higashi syndrome 9 trials
- Griscelli syndrome type 2 1 trial
- Familial hemophagocytic lymphohistiocytosis 3 1 trial
- Hermansky-Pudlak syndrome 2 0 trials
- Hermansky-Pudlak syndrome 9 0 trials
- Familial hemophagocytic lymphohistiocytosis 2 0 trials
- Familial hemophagocytic lymphohistiocytosis 4 0 trials
- Familial hemophagocytic lymphohistiocytosis 5 0 trials
- Familial hemophagocytic lymphohistiocytosis type 1 0 trials
- Hemophagocytic lymphohistiocytosis due to RhoG deficiency 0 trials
- Hemophagocytic lymphohistiocytosis, familial, 6 0 trials
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Aicardi-Goutieres syndrome 9 trials
10 sub-types
- Aicardi-Goutieres syndrome 1 2 trials Sub-types →
- Aicardi-Goutieres syndrome 2 0 trials
- Aicardi-Goutieres syndrome 3 0 trials
- Aicardi-Goutieres syndrome 4 0 trials
- Aicardi-Goutieres syndrome 5 0 trials
- Aicardi-Goutieres syndrome 6 0 trials
- Aicardi-Goutieres syndrome 7 0 trials
- Aicardi-Goutieres syndrome 8 0 trials
- Aicardi-Goutieres syndrome 9 0 trials
- Basal ganglia calcification, idiopathic, childhood-onset 0 trials
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Chronic mucocutaneous candidiasis 4 trials
12 sub-types
- Autoimmune enteropathy and endocrinopathy - susceptibility to chronic infections syndrome 0 trials
- Candidiasis, familial, 1 0 trials
- Candidiasis, familial, 3 0 trials
- Candidiasis, familial, 4 0 trials
- Candidiasis, familial, 6 0 trials
- Candidiasis, familial, 8 0 trials
- Candidiasis, familial, 9 0 trials
- Chronic mucocutaneous candidiasis due to inhibition of lymphoblastic transformation 0 trials
- Chronic mucocutaneous candidiasis due to intrinsic defect in lymphoblastic transformation 0 trials
- Chronic mucocutaneous candidiasis due to lymphokine deficiency 0 trials
- Chronic mucocutaneous candidiasis due to monocyte chemotactic disorder 0 trials
- Immunodeficiency 51 0 trials
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Immunoglobulin heavy chain deficiency 0 trials · 2 incl. sub-types
1 sub-type
- Selective IgG subclass deficiency 2 trials
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A20 haploinsufficiency 1 trial
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BENTA disease 1 trial
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Immuno-osseous dysplasia 0 trials · 1 incl. sub-types
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2 sub-types
- Inflammatory bowel disease 25 0 trials
- Inflammatory bowel disease 28 0 trials
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NK cell deficiency 0 trials
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Say-Barber-Miller syndrome 0 trials
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T cell and NK cell immunodeficiency 0 trials
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X-linked immunoneurologic disorder 0 trials
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Dendritic cell deficiency 0 trials
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2 sub-types
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Immunodeficiency 28 0 trials
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Immunodeficiency 33 0 trials
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Immunodeficiency 37 0 trials
1 sub-type
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Immunodeficiency 39 0 trials
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Immunodeficiency 47 0 trials
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Immunodeficiency 49 0 trials
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Properdin deficiency, X-linked 0 trials
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Trichohepatoenteric syndrome 0 trials
2 sub-types
- Trichohepatoenteric syndrome 1 0 trials
- Trichohepatoenteric syndrome 2 0 trials
Most studied deeper sub-types
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Rewriting the code: a One-Time gene fix for a rare immune disease?
Cure By invitation onlyThis trial tests a one-time gene therapy for WHIM syndrome, a rare inherited immune disorder that causes severe infections. Doctors collect a person's own blood stem cells, use base editing to correct the faulty CXCR4 gene, and return the edited cells to the body. The goal is to …
Phase 1/2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Cure
Last updated Sep 21, 2026 13:00 UTC
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MRNA HIV vaccine trial launches in healthy volunteers
Vaccine OngoingThis early-stage trial tests whether three experimental mRNA vaccines can safely teach the immune system to recognize and fight HIV. About 108 healthy, HIV-negative adults aged 18-55 will receive one of the vaccines. The main goal is to check for side effects and see if the vacci…
Phase 1 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Vaccine
Last updated Jun 27, 2026 07:54 UTC
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Stem cell transplant offers hope for rare immune disorder
Disease control OngoingThis study tests whether a stem cell transplant from a matched sibling, unrelated donor, or half-matched parent can fix the immune system in people with DOCK8 deficiency, a genetic condition that causes severe infections and cancer. Participants aged 4 to 35 receive chemotherapy …
Phase 2 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Aug 26, 2026 00:00 UTC
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New drug could make stem cell transplants safer for rare immune disorder
Disease control OngoingThis phase II trial tests whether a new drug called Briquilimab can make stem cell transplants safer for people with GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of infections and blood cancers. The study involves 13 participants a…
Phase 2 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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New shot could help Immune-Compromised patients fight infections
Disease control OngoingThis phase 3 trial is testing a new medicine called Newnorm, a concentrated antibody solution given as a shot under the skin, in 50 people with primary immunodeficiency (a condition where the immune system doesn't work properly). The goal is to see if Newnorm can prevent serious …
Phase 3 • Sponsor: Octapharma • Aim: Disease control
Last updated Aug 08, 2026 00:03 UTC
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New cell therapy targets Epstein-Barr virus in immune-compromised patients
Disease control OngoingThis study tests an experimental cell therapy called tabelecleucel in 190 people with diseases caused by the Epstein-Barr virus (EBV), including certain lymphomas and post-transplant complications. The treatment uses specially prepared immune cells to fight the virus. The main go…
Phase 2 • Sponsor: Pierre Fabre Medicament • Aim: Disease control
Last updated Aug 02, 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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New antibody therapy aims to shield kids with rare immune disease from deadly infections
Disease control OngoingThis study tests a medicine called Kedrion IVIG 10% in 30 children with primary immunodeficiency, a condition where the immune system doesn't work properly. The treatment is made from donated antibodies and is given through a vein to help prevent serious bacterial infections. Res…
Phase 3 • Sponsor: Kedrion S.p.A. • Aim: Disease control
Last updated Jun 27, 2026 13:00 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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Quick-Push immune therapy studied for safety and ease
Disease control OngoingThis study looks at whether giving Cutaquig® (a medicine that helps fight infections) by a faster manual push method is safe and easy for adults with primary or secondary immunodeficiency. About 36 participants will use the rapid push method at home. The goal is to see if this ap…
Sponsor: University of Alberta • Aim: Disease control
Last updated Jun 27, 2026 12:31 UTC
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Peer support could boost HIV med adherence and curb substance use
Disease control OngoingThis study tests a program called Khanya, where trained peers (people with lived substance use experience) help HIV patients stick to their medication and reduce drug or alcohol use. The program is offered in steps: a basic version first, then a more intensive one if needed. Rese…
Sponsor: University of Maryland, College Park • Aim: Disease control
Last updated Jun 27, 2026 12:30 UTC
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New antibody therapy tested for long-term safety in immune-deficient patients
Disease control OngoingThis study tests the long-term safety of TAK-881, a subcutaneous immunoglobulin treatment, in 53 adults and children with primary immunodeficiency diseases (PIDD) who completed a prior study. Participants receive regular infusions every 2-4 weeks and are monitored for side effect…
Phase 3 • Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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Milder stem cell transplant shows promise for kids with immune disorders
Disease control OngoingThis study tests a stem cell transplant using a milder chemotherapy-like regimen to treat children and young adults (up to age 28) with various immune system disorders. The goal is to help the donor stem cells settle in the body with fewer side effects. The study involves 20 part…
Phase 2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
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New drug aims to calm overactive immune system in rare disorders
Disease control OngoingThis study tests a drug called leniolisib in 12 people aged 12 to 75 with certain primary immunodeficiency disorders. The goal is to see if the drug is safe and can help control immune system problems. Participants will receive three different doses of the drug, and researchers w…
Phase 2 • Sponsor: Pharming Technologies B.V. • Aim: Disease control
Last updated Jun 27, 2026 09:05 UTC
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New stem cell approach could fix immune systems in kids
Disease control OngoingThis Phase II trial tests a stem cell transplant from unrelated or partially matched family donors for children and young adults with severe immune deficiencies. The stem cells are specially processed to remove certain immune cells, aiming to reduce complications. The goal is to …
Phase 2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Gene therapy trial aims to fix 'Bubble Boy' disease in newborns
Disease control PausedThis 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 1/2 • Sponsor: St. Jude Children's Research Hospital • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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New transplant method aims to cut dangerous immune reaction in half-matched donors
Disease control OngoingThis study tests a stem cell transplant method that removes certain immune cells from the donor's blood to lower the chance of graft-versus-host disease (GvHD), a serious complication. It involves 3 patients with blood cancers or non-cancerous blood disorders who lack a perfectly…
Sponsor: Baylor College of Medicine • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Gene-Edited stem cells aim to tame HIV
Disease control OngoingThis study tests whether giving HIV patients their own genetically modified stem cells is safe and feasible. The cells are changed to resist HIV, and patients receive a mild chemotherapy drug before infusion. The goal is to see if the modified cells can help control the virus, bu…
Phase 1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
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Scientists collect skin cells to pave way for future immune disease cures
Knowledge-focused By invitation onlyThis study collects small skin samples from people with primary immunodeficiency or immune regulation disorders, as well as from healthy volunteers. Researchers will use these cells to create cell lines for laboratory research. The goal is to better understand immune diseases and…
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated Sep 02, 2026 00:00 UTC
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Holistic education study for stress withdrawn before starting
Knowledge-focused CancelledThis study was designed to test a holistic education program for stress management in people from colonized English-speaking countries. It aimed to help with conditions like stress, PTSD, anxiety, and fatigue using pastoral counseling and self-care activities. However, the study …
Sponsor: Goddess Zena I. Jones • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:01 UTC