Primary Immunodeficiency Diseases
Primary Immunodeficiency Diseases
Explore linked clinical trials. Standard reference: MESH:M000650094120 results · Page 1 of 5
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorder | Active, not recruiting | Phase 3 | 2026-10-08 | NCT06634420 | |
| Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion | Bone Marrow Failure Disorders, Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Disease Attributes, Hemic and Lymphatic Diseases, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Lymphohistiocytosis, Hemophagocytic, Myeloproliferative Disorders, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Primary Immunodeficiency Diseases, acute myeloid leukemia, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, histiocytosis, immune system disorder, immunoproliferative disorder, inborn error of immunity, inherited hemoglobinopathy, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, myelodysplastic syndrome, myeloid leukemia, neoplasm, non-Langerhans cell histiocytosis | Active, not recruiting | Not applicable | 2026-10-08 | NCT05236764 | |
| Send-In Sample Collection to Achieve Genetic and Immunologic Characterization of Primary Immunodeficiencies | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Primary Immunodeficiency Diseases, hereditary disease, immune system disorder, inborn error of immunity | Recruiting | Not stated | 2026-10-08 | NCT03610802 | |
| The Mechanistic Biology of Primary Immunodeficiency Disorders | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Primary Immunodeficiency Diseases, hereditary disease, immune system disorder, inborn error of immunity | Recruiting | Not stated | 2026-10-08 | NCT03394053 | |
| Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, Primary Immunodeficiency Diseases, common variable immunodeficiency, hereditary disease, immune system disorder, inborn error of immunity, metabolic disease, severe combined immunodeficiency | Enrolling by invitation | Not stated | 2026-10-08 | NCT00895271 | |
| Detection and Characterization of Infections and Infection Susceptibility | Bacterial Infections and Mycoses, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Infections, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Primary Immunodeficiency Diseases, Recurrence, Reinfection, fungal infectious disease, hereditary disease, immune system disorder, inborn error of immunity | Recruiting | Not stated | 2026-10-08 | NCT00404560 | |
| Screening Protocol for Genetic Diseases of Lymphocyte Homeostasis and Programmed Cell Death | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Primary Immunodeficiency Diseases, autoimmune disease, hereditary disease, immune system disorder, inborn error of immunity | Recruiting | Not stated | 2026-10-08 | NCT00246857 | |
| Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, Primary Immunodeficiency Diseases, X-Linked Combined Immunodeficiency Diseases, X-linked disease, hereditary disease, immune system disorder, inborn error of immunity, severe combined immunodeficiency | Recruiting | Phase 1 / Phase 2 | 2026-10-06 | NCT01306019 | |
| NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD) | Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Hemic and Lymphatic Diseases, Infections, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Primary Immunodeficiency Diseases, Toxemia, X-linked disease, chronic granulomatous disease, hematologic disorder, hereditary disease, immune system disorder, inborn error of immunity, leukocyte disorder, phagocyte bactericidal dysfunction | Recruiting | Phase 1 | 2026-10-02 | NCT05189925 | |
| NIAID Centralized Sequencing Protocol | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Primary Immunodeficiency Diseases, autoimmune disease, hereditary disease, immune system disorder, inborn error of immunity | Recruiting | Not stated | 2026-10-02 | NCT03206099 | |
| Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Primary Immunodeficiency Diseases, hematologic disorder, hereditary disease, hyper-IgE recurrent infection syndrome 1, autosomal dominant, immune system disorder, inborn error of immunity, leukocyte disorder, phagocyte bactericidal dysfunction | Active, not recruiting | Phase 2 | 2026-09-28 | NCT01176006 | |
| Genetic Basis of Immunodeficiency | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, Primary Immunodeficiency Diseases, hereditary disease, immune system disorder, inborn error of immunity, metabolic disease, severe combined immunodeficiency | Recruiting | Not stated | 2026-09-28 | NCT00055172 | |
| Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Infections, Primary Immunodeficiency Diseases, hematologic disorder, hereditary disease, hyper-IgE recurrent infection syndrome 1, autosomal dominant, immune system disorder, inborn error of immunity, leukocyte disorder, lung disorder, phagocyte bactericidal dysfunction, pneumonia, respiratory system disorder, respiratory tract infectious disorder | Recruiting | Not stated | 2026-09-28 | NCT00006150 | |
| Knowledge of Inborn Errors of Immunity Among Pediatric Physicians | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Primary Immunodeficiency Diseases, hereditary disease, immune system disorder, inborn error of immunity | Not yet recruiting | Not stated | 2026-09-25 | NCT07837518 | |
| NTLA-2002 in Adults With Hereditary Angioedema (HAE) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorder | Active, not recruiting | Phase 1 / Phase 2 | 2026-09-25 | NCT05120830 | |
| Genetic Analysis of Immune Disorders | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Primary Immunodeficiency Diseases, hereditary disease, immune system disorder, immunodeficiency disease, inborn error of immunity | Recruiting | Not stated | 2026-09-25 | NCT00001467 | |
| Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, Primary Immunodeficiency Diseases, X-Linked Combined Immunodeficiency Diseases, X-linked disease, hereditary disease, immune system disorder, inborn error of immunity, severe combined immunodeficiency | Enrolling by invitation | Phase 1 / Phase 2 | 2026-09-23 | NCT06851767 | |
| Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorder | Enrolling by invitation | Phase 3 | 2026-09-22 | NCT06679881 | |
| Metabolic Profiling of Immune Responses in Immune-mediated Diseases | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin Diseases, Eczematous, Skin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, atopic eczema, dermatitis, hereditary disease, hereditary skin disorder, immune system disorder, inborn error of immunity, psoriasis, skin disorder | Recruiting | Not stated | 2026-09-22 | NCT04864886 | |
| Evaluation of Patients With Immune Function Abnormalities | Bronchiolitis Obliterans, Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Infections, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Primary Immunodeficiency Diseases, Recurrence, Reinfection, X-Linked Combined Immunodeficiency Diseases, X-linked disease, bronchial disorder, bronchiolitis, bronchiolitis obliterans syndrome, bronchitis, chronic granulomatous disease, cryptogenic organizing pneumonia, graft versus host disease, hematologic disorder, hereditary disease, immune system disorder, inborn error of immunity, leukocyte disorder, lung disorder, obstructive lung disease, phagocyte bactericidal dysfunction, respiratory system disorder, severe combined immunodeficiency | Recruiting | Not stated | 2026-09-22 | NCT00128973 | |
| Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, acquired angioedema, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorder | Recruiting | Phase 3 | 2026-09-21 | NCT07266805 | |
| Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorder | Recruiting | Phase 4 | 2026-09-17 | NCT07654829 | |
| Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, Primary Immunodeficiency Diseases, graft versus host disease, hereditary disease, immune system disorder, inborn error of immunity, metabolic disease, severe combined immunodeficiency | Active, not recruiting | Phase 1 / Phase 2 | 2026-09-16 | NCT03597594 | |
| The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics | Chronic Disease, Colorectal Neoplasms, Hereditary Nonpolyposis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cronkhite-Canada syndrome, Cysts, DNA repair disease, Digestive System Diseases, Digestive System Neoplasms, Disease Attributes, Dyskinesias, Familial Atypical Multiple Mole-Melanoma, Hyperpigmentation, Lynch syndrome, Melanosis, Neoplasms by Histologic Type, Neoplasms by Site, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pancreatic Cyst, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peutz-Jeghers syndrome, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, Spinocerebellar Ataxias, atactic disorder, non-human animal, ataxia telangiectasia, brain disorder, cardiovascular disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, chronic pancreatitis, classic familial adenomatous polyposis, colonic disorder, colorectal neoplasm, digestive system disorder, digestive system neoplasm, hereditary disease, hereditary neoplastic syndrome, immune system disorder, inborn error of immunity, intestinal disorder, lentigo, melanocytic neoplasm, melanocytic nevus, melanoma, cutaneous malignant, susceptibility to, 2, metabolic disease, neoplasm, nervous system disorder, neurocutaneous syndrome, pancreas disorder, pancreatic ductal adenocarcinoma, pancreatitis, skin disorder, skin pigmentation disorder, telangiectasis, vascular disorder | Active, not recruiting | Not stated | 2026-09-15 | NCT05692596 | |
| STOP-HAE: A Phase 3 Study of ADX-324 in HAE | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorder | Recruiting | Phase 3 | 2026-09-10 | NCT06960213 |