Hereditary Complement Deficiency Diseases
Hereditary Complement Deficiency Diseases
Explore linked clinical trials. Standard reference: MESH:M00065009533 results · Page 1 of 2
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 | |
| 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 | |
| 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 | |
| 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 | |
| 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 | |
| Phase 3 Extension Study of ADX-324 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 | Recruiting | Phase 3 | 2026-09-04 | NCT07428499 | |
| Donidalorsen Treatment in Children With Hereditary Angioedema | 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 | 2026-09-02 | NCT07298447 | |
| Angioedema Biomarker Research Study | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Mast Cell Activation Disorders, Mastocytosis, Systemic, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, connective tissue neoplasm, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, indolent systemic mastocytosis, mastocytosis, neoplasm, skin disorder, skin vascular disease, urticaria, vascular disorder | Recruiting | Not stated | 2026-08-24 | NCT06210698 | |
| A Survey of Lanadelumab in Participants With Hereditary Angioedema | 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 | Not stated | 2026-08-04 | NCT05397431 | |
| A Long-Term Study of Navenibart in Participants With Hereditary Angioedema | 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 | 2026-07-30 | NCT07204938 | |
| Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary angioedema type 3, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorder | Enrolling by invitation | Phase 2 / Phase 3 | 2026-07-21 | NCT05396105 | |
| A Study of Navenibart in Participants With Hereditary Angioedema | 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 | 2026-06-30 | NCT06842823 | |
| A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea | 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 | Not yet recruiting | Not stated | 2026-06-18 | NCT07445087 | |
| A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt | 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 | Not yet recruiting | Not stated | 2026-06-18 | NCT07218393 | |
| Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients | 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 | 2026-06-16 | NCT05691361 | |
| A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients 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 | Recruiting | Not stated | 2026-06-03 | NCT07001280 | |
| A Study to Learn About the C1-Inhibitor Function as Diagnosis for Hereditary Angioedema | 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 | Not applicable | 2026-05-19 | NCT07293364 | |
| A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia | 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 | Not stated | 2026-05-11 | NCT07263685 | |
| A Phase 2 in Adult Subjects With Hereditary Angioedema | 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 2 | 2026-04-17 | NCT06846398 | |
| Real-life Ecological Momentary Assessment of Lived Burden in Hereditary AngioEdema | 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 | Not stated | 2026-03-04 | NCT07448181 | |
| Improving Deceased-Donor Kidney Transplant Outcomes Via a Single Intragraft Injection of C1 Esterase Inhibitor (IMPROVE TRIAL) | 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 2 | 2026-02-27 | NCT06919003 | |
| Berotralstat Treatment in Children With Hereditary Angioedema | 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 | 2026-02-25 | NCT05453968 | |
| 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 | Active, not recruiting | Phase 3 | 2026-02-13 | NCT06669754 | |
| Institutional Registry of Rare Diseases | Abnormalities, Multiple, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cushing syndrome, Digestive System Abnormalities, Digestive System Diseases, Disease Attributes, Eosinophilia, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Complement Deficiency Diseases, Hirschsprung disease, Hypersensitivity, Delayed, Hypersensitivity, Immediate, Immunoglobulin G4-Related Disease, Imprinting Disorders, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Male Urogenital Diseases, Mast Cell Activation Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Multiple Primary, Neoplasms, Nerve Tissue, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Overweight, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Prader-Willi syndrome, Primary Immunodeficiency Diseases, Rare Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, Urogenital Abnormalities, Urogenital Diseases, Wilson disease, adrenal gland disorder, adrenal gland hyperfunction, adrenogenital syndrome, amyloidosis, angioedema, angiomatosis, aortic valve disorder, aortic valve stenosis, autoimmune disease, basal ganglia disorder, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, chromosomal disorder, colonic disorder, congenital adrenal hyperplasia, connective tissue neoplasm, demyelinating disease, digestive system disorder, disorder of sexual differentiation, ectodermal dysplasia syndrome, endocrine system disorder, eosinophilic esophagitis, esophageal disorder, esophagitis, gastroenteritis, gonadal disorder, heart disorder, heart valve disorder, hematologic disorder, hemorrhagic disease, hereditary angioedema, hereditary disease, hereditary hemorrhagic telangiectasia, hereditary neoplastic syndrome, hereditary skin disorder, hypertensive disorder, hypertrophic cardiomyopathy, immune system disorder, inborn error of immunity, inborn errors of metabolism, inborn metal metabolism disorder, inflammatory bowel disease, inherited neurodegenerative disorder, intellectual disability, intestinal disorder, leukocyte disorder, liver disorder, lung disorder, lymphatic system disorder, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, malignant endocrine neoplasm, mastocytosis, megacolon, metabolic disease, movement disorder, multiple endocrine neoplasia, neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroendocrine neoplasm, nutritional disorder, obesity disorder, overnutrition, paraganglioma, pheochromocytoma, primitive neuroectodermal tumor, proteostasis deficiencies, pulmonary hypertension, respiratory system disorder, sarcoidosis, skin disorder, skin vascular disease, sphingolipidosis, steroid inherited metabolic disorder, subvalvular aortic stenosis, telangiectasis, urticaria, vascular disorder, vascular hemostatic disease, vascular malformation, von Hippel-Lindau disease | Recruiting | Not stated | 2026-01-14 | NCT06573723 |