46 results · Page 1 of 2

Swipe the table sideways for more columns.

TrialConditionsStatusPhaseUpdatedMore infoNCT ID
An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut SyndromeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Epileptic Syndromes, Lennox-Gastaut syndrome, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, hereditary disease, nervous system disorder, visual epilepsyRecruitingPhase 32026-10-09NCT05066217
A Study Evaluating the Efficacy and Safety of Xywav Expanded Dosing vs Placebo in Participants With Narcolepsy or IHDisorders of Excessive Somnolence, Dyssomnias, Sleep Disorders, Intrinsic, idiopathic hypersomnia, narcolepsy, nervous system disorder, psychiatric disorder, sleep-wake disorderRecruitingPhase 32026-10-08NCT07625280
Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care TreatmentBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorderRecruitingPhase 32026-10-08NCT06247085
Efficacy and Safety of Ribupatide Administered Once Weekly Compared With Semaglutide and Placebo in Participants Living With Obesity Who Do Not Have DiabetesBody Weight, Nutritional and Metabolic Diseases, Overweight, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, nutritional disorder, obesity disorder, overnutritionActive, not recruitingPhase 32026-10-06NCT07284979
Efficacy and Safety of Ribupatide Administered Once Weekly in Participants Living With Obesity or Overweight and DiabetesBody Weight, Nutritional and Metabolic Diseases, Overweight, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, diabetes mellitus, endocrine system disorder, glucose metabolism disease, metabolic disease, nutritional disorder, obesity disorder, overnutritionActive, not recruitingPhase 32026-10-06NCT07284901
Inclisiran Versus Placebo for the Prevention of Major Adverse Cardiovascular and Limb Events in Patients Undergoing Percutaneous Coronary Intervention or Peripheral Endovascular InterventionArterial Occlusive Diseases, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Nutritional and Metabolic Diseases, arteriosclerosis disorder, cardiovascular disorder, coronary artery disorder, heart disorder, hyperlipidemia, metabolic disease, myocardial ischemia, vascular disorderRecruitingPhase 42026-10-05NCT06909565
SUNOSI® (Solriamfetol) Pregnancy RegistryApnea, Disorders of Excessive Somnolence, Dyssomnias, Respiration Disorders, Sleep Disorders, Intrinsic, narcolepsy, nervous system disorder, obstructive sleep apnea syndrome, psychiatric disorder, respiratory system disorder, sleep apnea syndrome, sleep-wake disorderRecruitingNot stated2026-10-05NCT06413420
A Study to Evaluate Efficacy, Safety, and PK of XEMBIFY®+Standard Medical Treatment (SMT) Compared to Placebo+SMT to Prevent Infections in Participants With HGG and Recurrent or Severe Infections Associated With B-cell Chronic Lymphocytic Leukemia, Multiple Myeloma, and Non-Hodgkin LymphomaB-cell chronic lymphocytic leukemia, Bacterial Infections and Mycoses, Blood Protein Disorders, Chronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Hypogammaglobulinemia, Infections, Leukemia, B-Cell, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, agammaglobulinemia, bacterial infectious disease, hematologic disorder, immune system disorder, immunoproliferative disorder, inborn error of immunity, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, neoplasm, non-Hodgkin lymphomaRecruitingPhase 32026-09-30NCT05645107
Efficacy, Safety, and Tolerability Study of Lunsekimig Compared With Placebo in Adult Participants With Inadequately Controlled Chronic Obstructive Pulmonary Disease (COPD) Characterized by an Eosinophilic PhenotypeChronic Disease, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, chronic obstructive pulmonary disease, lung disorder, obstructive lung disease, respiratory system disorderRecruitingPhase 32026-09-22NCT07190209
A Study to Assess the Efficacy and Safety of Empasiprubart Versus IVIg in Adults With Multifocal Motor Neuropathymultifocal motor neuropathyActive, not recruitingPhase 32026-09-22NCT06742190
Study of Olverembatinib (HQP1351) in Patients With CML-CPChronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Chronic-Phase, Myeloproliferative Disorders, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, bone marrow disorder, chronic myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmRecruitingPhase 32026-09-15NCT06423911
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, nervous system disorder, visual epilepsyRecruitingPhase 32026-09-15NCT04462770
A Phase 3 Efficacy and Safety Study of HBS-301 in Participants With Idiopathic Hypersomnia (IH)Disorders of Excessive Somnolence, Dyssomnias, Fatigue, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Sleep Disorders, Intrinsic, idiopathic hypersomnia, nervous system disorder, psychiatric disorder, sleep-wake disorderRecruitingPhase 32026-09-14NCT07500090
Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, blood platelet disease, coagulation protein disease, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, von Willebrand disease (hereditary or acquired)RecruitingPhase 32026-09-14NCT07115004
A Study to Determine the Efficacy and Safety of Finerenone and SGLT2i in Combination in Hospitalized Patients With Heart Failure (CONFIRMATION-HF)cardiovascular disorder, heart disorder, heart failureRecruitingPhase 32026-09-10NCT06024746
A Study to Evaluate the Efficacy and Safety of Sotagliflozin in Symptomatic Obstructive and Non-obstructive Hypertrophic CardiomyopathyCardiomyopathy, Hypertrophic Obstructive, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, heart disorder, heart valve disorder, hypertrophic cardiomyopathy, subvalvular aortic stenosisActive, not recruitingPhase 32026-08-26NCT06481891
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)Cholangitis, Sclerosing, Digestive System Diseases, Pathological Conditions, Signs and Symptoms, Pruritus, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, bile duct disorder, biliary tract disorder, cholangitis, cholestasis, primary sclerosing cholangitis, skin disorderActive, not recruitingPhase 22026-08-26NCT04663308
A Study of Pitolisant in Patients With Prader-Willi SyndromeAbnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disorders of Excessive Somnolence, Dyssomnias, Imprinting Disorders, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Overweight, Prader-Willi syndrome, Sleep Disorders, Intrinsic, chromosomal disorder, hereditary disease, intellectual disability, nervous system disorder, nutritional disorder, obesity disorder, overnutrition, psychiatric disorder, sleep-wake disorderRecruitingPhase 32026-08-25NCT06366464
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary CholangitisDigestive System Diseases, Fibrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pruritus, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, bile duct disorder, biliary tract disorder, cholangitis, cholestasis, cirrhosis of liver, intrahepatic cholestasis, liver disorder, primary biliary cholangitis, skin disorderActive, not recruitingPhase 22026-08-25NCT05050136
A Phase 2/3 Study of Osivelotor in Adult and Adolescent Participants With SCDCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseRecruitingPhase 2 / Phase 32026-08-18NCT05431088
Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous DiseaseChronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, chronic granulomatous disease, hematologic disorder, hereditary disease, immune system disorder, inborn error of immunity, leukocyte disorder, phagocyte bactericidal dysfunctionRecruitingNot stated2026-08-17NCT06605378
A Global Clinical Study Investigating the Safety and Effectiveness of Smith and Nephew's Porous Knee System in Patients Who Need a Total Knee Replacement Due to Degenerative Arthritis, Post-traumatic Arthritis or Inflammatory ArthritisSkin and Connective Tissue Diseases, arthritic joint disease, arthropathy, autoimmune disease, connective tissue disorder, immune system disorder, musculoskeletal system disorder, osteoarthritis, rheumatic disorder, rheumatoid arthritisActive, not recruitingNot stated2026-08-11NCT05197036
Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorderRecruitingPhase 1 / Phase 22026-08-10NCT03406611
A Study of EDG-7500 in Adults With Hypertrophic Cardiomyopathy (CIRRUS-HCM)aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, heart disorder, heart valve disorder, hypertrophic cardiomyopathy, subvalvular aortic stenosisActive, not recruitingPhase 22026-07-31NCT06347159
A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their BabiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderRecruitingNot stated2026-07-10NCT06628687