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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Treatment With Bempedoic Acid and/or Its Fixed-dose Combination With Ezetimibe in Primary Hypercholesterolemia or Mixed DyslipidemiaDyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Nutritional and Metabolic Diseases, hyperlipidemia, metabolic diseaseActive, not recruitingNot stated2026-09-30NCT04579367
Study to Evaluate Switching to Brelovitug for the Treatment of CHD in Participants Receiving BulevirtideChronic Disease, Digestive System Diseases, Disease Attributes, Hepatitis D, Chronic, Hepatitis, Chronic, Infections, Pathologic Processes, Pathological Conditions, Signs and Symptoms, RNA Virus Infections, hepatitis, hepatitis D virus infection, liver disorder, viral hepatitis, viral infectious diseaseRecruitingPhase 2 / Phase 32026-09-10NCT07454837
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 32026-09-01NCT06904261
Study of WAL0921 in Patients With Glomerular Kidney DiseasesDiabetes Complications, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, IgA glomerulonephritis, Male Urogenital Diseases, Urogenital Diseases, autoimmune disease, diabetes mellitus, diabetic kidney disease, endocrine system disorder, glomerulonephritis, immune system disorder, kidney disorder, minimal change disease, nephritis, nephrosis, primary focal segmental glomerulosclerosis, urinary system disorderRecruitingPhase 22026-08-03NCT06466135
Chronic Limb-Threatening Ischemia Treated With Intravascular Lithotripsy Observational StudyArterial Occlusive Diseases, Chronic Disease, Chronic Limb-Threatening Ischemia, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, arteriosclerosis disorder, atherosclerosis, cardiovascular disorder, ischemic disease, peripheral arterial disease, peripheral vascular disease, vascular disorderActive, not recruitingNot stated2026-07-30NCT06149650
Monitoring Oedema in Heart Failure to Improve Function and Reduce Hospitalisation Riskcardiovascular disorder, heart disorder, heart failureRecruitingNot applicable2026-07-28NCT07067658
Adalimumab Dose Adjustment for Pediatric Uveitis Treatment Trialanterior uveitis, eye disorder, intermediate uveitis, panuveitis, uveal disorder, uveitisNot yet recruitingPhase 42026-07-13NCT07699107
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, achondroplasia, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hereditary endocrine growth disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasiaRecruitingPhase 22026-07-13NCT07169279
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett SyndromeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rett syndrome, Signs and Symptoms, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder, neurodevelopmental disorder, psychiatric disorderActive, not recruitingPhase 32026-06-10NCT05898620
Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXSCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, X-linked disease, chromosomal disorder, fragile X syndrome, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorderActive, not recruitingPhase 2 / Phase 32026-05-27NCT03802799
Positron Emission Tomography to Assess the Effect of Camzyos on Ischaemia in HOCM: PEACH TrialArterial Occlusive Diseases, Ventricular Outflow Obstruction, Ventricular Outflow Obstruction, Left, aortic valve disorder, aortic valve stenosis, arteriosclerosis disorder, cardiomyopathy, cardiovascular disorder, coronary artery disorder, heart disorder, heart valve disorder, hypertrophic cardiomyopathy, myocardial ischemia, subvalvular aortic stenosis, vascular disorderRecruitingNot applicable2026-05-22NCT07120776
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular DiseasesAlport syndrome, Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Hemorrhage, IgA glomerulonephritis, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, Urogenital Abnormalities, Urogenital Diseases, autoimmune disease, blood coagulation disease, cardiovascular disorder, connective tissue disorder, focal segmental glomerulosclerosis, glomerulonephritis, hematologic disorder, hemorrhagic disease, hereditary nephritis, immune system disorder, immunoglobulin A vasculitis, kidney disorder, macular corneal dystrophy, minimal change disease, nephritis, nephrosis, purpura, skin disorder, skin vascular disease, type III hypersensitivity disease, urinary system disorder, vascular disorder, vascular hemostatic disease, vasculitisRecruitingPhase 22026-05-12NCT05003986
Mothers Working to Prevent Early Stillbirth Study 20-28Death, Female Urogenital Diseases and Pregnancy Complications, Fetal Death, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Perinatal Death, Urogenital Diseases, pregnancy disorder, pregnancy disorder with abortive outcomeRecruitingNot stated2026-02-27NCT06005272
DENTOFACIAL-PBT Stage 2 - Feasibility StudyCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Head and Neck Neoplasms, Neoplasms by Site, childhood malignant neoplasm, developmental disability, head and neck cancer, injury, neoplasm, neurodevelopmental disorder, psychiatric disorder, radiation injuryEnrolling by invitationNot stated2026-01-26NCT07057284
Treating People With Idiopathic Pulmonary Fibrosis With the Addition of LansoprazoleCough, Pathological Conditions, Signs and Symptoms, Respiration Disorders, Signs and Symptoms, Signs and Symptoms, Respiratory, idiopathic pulmonary fibrosis, interstitial lung disease, lung disorder, pulmonary fibrosis, respiratory system disorderRecruitingPhase 32025-12-22NCT04965298
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)Dyslipidemias, Lipid Metabolism Disorders, Nutritional and Metabolic Diseases, hyperlipidemia, hypertriglyceridemia, metabolic diseaseActive, not recruitingPhase 32025-12-01NCT05681351
Validation of Sensors for Long-term Non-Invasive Fetal MonitoringCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases and Pregnancy Complications, Fetal Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, fetal growth restriction, hereditary endocrine growth disease, pregnancy disorderRecruitingNot applicable2025-05-01NCT06952777
Intestinal Microbiota Transplant Prior to Allogeneic Stem Cell Transplant (MAST) TrialChronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myeloproliferative Disorders, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute biphenotypic leukemia, acute leukemia of ambiguous lineage, acute myeloid leukemia, bone marrow disorder, chronic myelomonocytic leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, myelodysplastic syndrome, myelodysplastic/myeloproliferative disease, myeloid leukemia, neoplasmRecruitingPhase 22025-04-17NCT06355583
Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3AActive, not recruitingPhase 1 / Phase 22025-03-30NCT04201405
Methylated Biomarkers Predictive of Endometrial Cancer RiskFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Neoplasms by Site, Urogenital Diseases, Urogenital Neoplasms, endometrium neoplasm, female reproductive system disorder, female reproductive system neoplasm, neoplasm, uterine disorderActive, not recruitingNot stated2025-01-30NCT06800677
UNICORNS: Uveitis in Childhood Prospective National Cohort Studyeye disorder, uveal disorder, uveitisActive, not recruitingNot stated2024-10-04NCT05385757
Cardiometabolic Health in First Time PregnancyNutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, cardiovascular disorder, diabetes mellitus, disease, endocrine system disorder, glucose metabolism disease, heart disorder, metabolic disease, syndromic diseaseRecruitingNot stated2024-04-09NCT05856318