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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study of Baricitinib (LY3009104) to Preserve Beta Cell Function in Children and Adults Newly Diagnosed With Type 1 Diabetes (BARICADE-PRESERVE)Nutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitusRecruitingPhase 32026-10-08NCT07222332
A Study of Baricitinib (LY3009104) for the Delay of Stage 3 Type 1 Diabetes in At-Risk Children and AdultsNutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitusRecruitingPhase 32026-10-08NCT07222137
A Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of RO7268489 as Add-on Therapy to Ocrelizumab, in Participants With Progressive Forms of Multiple Sclerosis (MS)CNS demyelinating autoimmune disease, Chronic Disease, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, autoimmune disease, autoimmune disorder of the nervous system, chronic progressive multiple sclerosis, demyelinating disease, immune system disorder, multiple sclerosis, nervous system disorderRecruitingPhase 22026-10-07NCT07282574
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia ACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderRecruitingPhase 32026-10-06NCT07416604
A Real-World Study to Gain Clinical Insights Into Roche Ophthalmology ProductsBranch Vein Occlusion, Embolism and Thrombosis, Venous Thrombosis, cardiovascular disorder, central retinal vein occlusion, diabetic macular edema, eye disorder, retinal disorder, retinal vein occlusion, thrombotic disease, vascular disorderActive, not recruitingNot stated2026-10-02NCT05476926
A Study to Learn About the Effects of the Combination of Elranatamab, Daratumumab and Lenalidomide Compared With Daratumumab, Bortezomib, Lenalidomide, and Dexamethasone in Patients With Newly Diagnosed Multiple Myeloma Who Are Not Candidates for TransplantBlood Protein Disorders, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, cardiovascular disorder, hematologic disorder, hemorrhagic disease, immune system disorder, immunoproliferative disorder, lymphoproliferative syndrome, monoclonal gammopathy, neoplasm, plasma cell neoplasm, vascular disorder, vascular hemostatic diseaseRecruitingPhase 32026-09-25NCT05623020
A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)acute leukemiaRecruitingPhase 1 / Phase 22026-09-25NCT04811560
A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 AlterationsGranuloma, Hemic and Lymphatic Diseases, Neoplasm Metastasis, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neoplastic Processes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, T-cell non-Hodgkin lymphoma, anaplastic large cell lymphoma, astrocytoma (excluding glioblastoma), connective tissue neoplasm, epithelial neoplasm, glioblastoma, glioma, immune system disorder, immunoproliferative disorder, inflammatory myofibroblastic tumor, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumor, sarcoma, solitary fibrous tumorRecruitingPhase 1 / Phase 22026-09-25NCT04094610
A Study of Apalutamide in Participants With High-Risk, Localized or Locally Advanced Prostate Cancer Who Are Candidates for Radical ProstatectomyGenital Diseases, Male Urogenital Diseases, Neoplasms by Site, Urogenital Diseases, Urogenital Neoplasms, male reproductive organ cancer, male reproductive system disorder, neoplasm, prostate cancer, prostate disorderActive, not recruitingPhase 32026-09-25NCT03767244
A Safety Study of Ustekinumab in the Treatment of Pediatric Participants Aged 6 Years and Older With Moderate to Severe Plaque PsoriasisSkin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, psoriasis, skin disorderActive, not recruitingNot stated2026-09-25NCT03218488
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia ACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderRecruitingPhase 32026-09-23NCT07416526
Rhu-pGSN for Acute Respiratory Distress Syndrome (ARDS)Infections, Respiration Disorders, adult acute respiratory distress syndrome, lung disorder, respiratory failure, respiratory system disorderRecruitingPhase 22026-09-10NCT05947955
Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country StudyDisease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Urogenital Diseases, glomerulonephritis, kidney disorder, nephritis, urinary system disorderRecruitingPhase 42026-09-09NCT07746895
REdo Transcatheter Aortic VALVE Implantation for the Management of Transcatheter Aortic Valve FailureVentricular Outflow Obstruction, aortic valve disorder, aortic valve stenosis, cardiovascular disorder, heart disorder, heart valve disorderRecruitingNot stated2026-08-25NCT06557798
A Prospective Registry Study to Assess Real-world Patient Characteristics, Treatment Patterns, and Longitudinal Outcomes in Patients Receiving Mavacamten and Other Treatments for Symptomatic Obstructive Hypertrophic Cardiomyopathy (Obstructive-HCM)aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, heart disorder, heart failure, heart valve disorder, hypertrophic cardiomyopathy, subvalvular aortic stenosisRecruitingNot stated2026-08-19NCT05489705
A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)Frontotemporal Lobar Degeneration, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, TDP-43 proteinopathy, aphasia, brain disorder, central nervous system disorder, cognitive disorder, communication disorder, dementia, frontotemporal dementia, language disorder, metabolic disease, nervous system disorder, neurodegenerative disease, primary progressive aphasia, proteostasis deficiencies, psychiatric disorder, speech disorderRecruitingPhase 1 / Phase 22026-08-13NCT06064890
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious InfectionsAbnormalities, Multiple, Barth syndrome, Bone Marrow Failure Disorders, Cardiovascular Abnormalities, Cohen syndrome, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Digestive System Diseases, Hemic and Lymphatic Diseases, Kostmann syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Shwachman-Diamond syndrome, X-linked disease, agranulocytosis, autoimmune disease, bone marrow disorder, cardiovascular disorder, congenital heart disease, exocrine pancreatic insufficiency, heart disorder, hematologic disorder, hereditary disease, immune system disorder, inborn errors of metabolism, leukocyte disorder, leukopenia, lipomatosis, metabolic disease, neutropenia, pancreas disorderRecruitingPhase 32026-08-06NCT06056297
A Study Comparing Anitocabtagene Autoleucel to Standard of Care Therapy in Participants With Relapsed/ Refractory Multiple MyelomaBlood Protein Disorders, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, cardiovascular disorder, hematologic disorder, hemorrhagic disease, immune system disorder, immunoproliferative disorder, lymphoproliferative syndrome, monoclonal gammopathy, neoplasm, plasma cell neoplasm, vascular disorder, vascular hemostatic diseaseActive, not recruitingPhase 32026-07-29NCT06413498
Comparison Between Rituximab Plus Zanubrutinib Versus Rituximab Monotherapy in Untreated SMZL Patientssplenic marginal zone lymphomaActive, not recruitingPhase 32026-07-22NCT05735834
Registry for Patients With X-Linked HypophosphatemiaBone Diseases, Metabolic, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hypophosphatemia, Familial, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Rickets, X-Linked Hypophosphatemic, Urogenital Diseases, X-linked hypophosphatemic rickets, bone disorder, calcium metabolic disease, hereditary disease, hypophosphatemia, hypophosphatemic rickets, inborn errors of metabolism, inborn metal metabolism disorder, kidney disorder, metabolic disease, musculoskeletal system disorder, nutritional deficiency disease, nutritional disorder, phosphorus metabolism disease, renal tubular transport disease, rickets, urinary system disorder, vitamin D deficiency, vitamin deficiency disorderRecruitingNot stated2026-07-09NCT03193476
A Study Assessing HMB-002 in Participants With Von Willebrand DiseaseCongenital, 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), von Willebrand disease 3RecruitingPhase 1 / Phase 22026-06-30NCT06754852
A Study to Investigate APL-4098 Alone and in Combination in Adults With AML or MDSCongenital, Hereditary, and Neonatal Diseases and Abnormalities, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Myelodysplastic Syndrome Acute Myeloid Leukemia, Neoplasms by Histologic Type, acute myeloid leukemia, anemia, bone marrow disorder, hematologic disorder, hereditary disease, leukemia, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts, myelodysplastic syndrome with single lineage dysplasia, myeloid leukemia, neoplasmRecruitingPhase 12026-06-01NCT06372717
A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host DiseaseBronchiolitis Obliterans, bronchial disorder, bronchiolitis, bronchiolitis obliterans syndrome, bronchitis, cryptogenic organizing pneumonia, graft versus host disease, immune system disorder, lung disorder, obstructive lung disease, respiratory system disorderActive, not recruitingPhase 22026-05-22NCT06388564
MITHRIDATE: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High Risk Polycythemia VeraHemic and Lymphatic Diseases, Myeloproliferative Disorders, Neoplasms by Site, acquired polycythemia vera, bone marrow disorder, bone marrow neoplasm, hematologic disorder, hematopoietic and lymphoid system neoplasm, neoplasmRecruitingPhase 32026-04-28NCT04116502
A Global Prospective Observational Registry of Patients With Pompe DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorderRecruitingNot stated2026-03-10NCT06121011