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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Evaluate XEN1101 as Adjunctive Therapy in Primary Generalized Tonic-Clonic Seizuresbrain disorder, central nervous system disorder, epilepsy, nervous system disorderRecruitingPhase 32026-10-08NCT05667142
Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and Compensated CirrhosisDigestive System Diseases, Fibrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, bile duct disorder, biliary tract disorder, cholestasis, cirrhosis of liver, intrahepatic cholestasis, liver disorder, primary biliary cholangitisRecruitingPhase 32026-10-06NCT06051617
A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)Neoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndromeRecruitingPhase 22026-10-05NCT06987539
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeAbnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderActive, not recruitingPhase 32026-09-29NCT06914609
A Study of Combination Therapy With Guselkumab and Golimumab in Participants With Moderately to Severely Active Ulcerative ColitisDigestive System Diseases, colitis, colonic disorder, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorder, ulcerative colitisActive, not recruitingPhase 22026-09-28NCT05242484
A Study of Combination Therapy With Guselkumab and Golimumab in Participants With Moderately to Severely Active Crohn's DiseaseCrohn disease, Digestive System Diseases, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorderActive, not recruitingPhase 22026-09-28NCT05242471
A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, blood platelet disease, hematologic disorder, neonatal thrombocytopenia, thrombocytopeniaRecruitingPhase 32026-09-25NCT06533098
A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's DiseaseCrohn disease, Digestive System Diseases, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorderRecruitingPhase 32026-09-25NCT05923073
A Study of Nipocalimab in Pregnancies at Risk for Severe Hemolytic Disease of the Fetus and Newborn (HDFN)Hemolytic Disease of the Fetus and NewbornRecruitingPhase 32026-09-25NCT05912517
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseActive, not recruitingPhase 22026-09-09NCT06128564
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 2 / Phase 32026-09-09NCT05115110
Research Study on Whether Semaglutide Works in People With Non-alcoholic Steatohepatitis (NASH)Digestive System Diseases, fatty liver disease, liver disorder, metabolic dysfunction-associated steatohepatitis, metabolic dysfunction-associated steatotic liver diseaseActive, not recruitingPhase 32026-09-04NCT04822181
DETERMINE Trial Treatment Arm 07: Dabrafenib in Combination With Trametinib in Adult, Paediatric and Teenage/Young Adult Patients With BRAF V600 Mutation-Positive Cancers.Adnexal Diseases, Blood Protein Disorders, Cancer of Gastrointestinal Tract, Digestive System Diseases, Digestive System Neoplasms, Erdheim-Chester disease, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Head and Neck Neoplasms, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Non-Melanoma Skin Neoplasms, Otorhinolaryngologic Neoplasms, Thoracic Neoplasms, Thyroid Cancer, Papillary, Urogenital Diseases, Urogenital Neoplasms, adenocarcinoma, bronchial neoplasm, bronchogenic carcinoma, cancer, carcinoma, cardiovascular disorder, colonic disorder, colorectal neoplasm, digestive system disorder, digestive system neoplasm, endocrine system disorder, epithelial neoplasm, female reproductive system disorder, female reproductive system neoplasm, glioma, gonadal disorder, hematologic disorder, hematopoietic and lymphoid system neoplasm, hemorrhagic disease, histiocytosis, immune system disorder, immunoproliferative disorder, interstitial lung disease, intestinal disorder, laryngeal disorder, laryngeal neoplasm, lung disorder, lung neoplasm, lymphatic system disorder, lymphoproliferative syndrome, malignant endocrine neoplasm, monoclonal gammopathy, neoplasm, neuroepithelial neoplasm, non-Langerhans cell histiocytosis, non-small cell lung carcinoma, otorhinolaryngologic disease, ovarian cancer, ovarian disorder, pancreas disorder, papillary adenocarcinoma, plasma cell neoplasm, primitive neuroectodermal tumor, rectal disorder, respiratory system disorder, respiratory tract neoplasm, thyroid gland disorder, thyroid gland undifferentiated (anaplastic) carcinoma, thyroid tumor, vascular disorder, vascular hemostatic diseaseRecruitingPhase 2 / Phase 32026-08-17NCT07440290
GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's DiseaseCognition Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Huntington disease, basal ganglia disorder, brain disorder, central nervous system disorder, choreatic disease, cognitive disorder, dementia, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, psychiatric disorderActive, not recruitingPhase 22026-08-10NCT05686551
DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial) - Master Screening ProtocolHemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms by Site, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoproliferative syndrome, neoplasmRecruitingPhase 2 / Phase 32026-07-01NCT05722886
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 32026-06-01NCT05337553
Capillary OGTT StudyNutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitus, type 2 diabetes mellitusRecruitingNot stated2026-04-13NCT06815081
Identifying the Causes and Risk Factors of Pulmonary Exacerbations in Cystic FibrosisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2026-03-19NCT06940531
Cooling in Mild EncephalopathyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, perinatal asphyxiaRecruitingPhase 32026-02-06NCT05889507
DETERMINE Trial Treatment Arm 04: Trastuzumab in Combination With Pertuzumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With HER2 Amplification or Activating MutationsAdnexal Diseases, Biliary Tract Neoplasms, Digestive System Diseases, Digestive System Neoplasms, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Head and Neck Neoplasms, Hemic and Lymphatic Diseases, Male Urogenital Diseases, Mouth Neoplasms, Neoplasms by Site, Skin and Connective Tissue Diseases, Stomatognathic Diseases, Thoracic Neoplasms, Urogenital Diseases, Urogenital Neoplasms, benign urinary system neoplasm, biliary tract disorder, colonic disorder, colorectal neoplasm, digestive system disorder, digestive system neoplasm, endocrine system disorder, female reproductive system disorder, female reproductive system neoplasm, gallbladder disorder, gallbladder neoplasm, gonadal disorder, hematologic disorder, hematopoietic and lymphoid system neoplasm, intestinal disorder, lung disorder, lung neoplasm, male reproductive organ cancer, male reproductive system disorder, malignant endocrine neoplasm, mouth disorder, neoplasm, ovarian cancer, ovarian disorder, pancreas disorder, pancreatic neoplasm, prostate cancer, prostate disorder, rectal disorder, respiratory system disorder, respiratory tract neoplasm, salivary gland cancer, salivary gland disorder, skin disorder, skin neoplasm, urinary bladder disorder, urinary bladder neoplasm, urinary system disorderRecruitingPhase 2 / Phase 32025-12-02NCT05786716
DETERMINE Trial Treatment Arm 03: Entrectinib in Adult, Paediatric and Teenage/Young Adult Patients With ROS1 Gene Fusion-Positive Cancers.Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Skin and Connective Tissue Diseases, brain disorder, cancer, central nervous system cancer, central nervous system disorder, epithelial neoplasm, glioma, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoproliferative syndrome, melanocytic neoplasm, melanoma, neoplasm, nervous system disorder, neuroendocrine neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumor, skin disorder, skin neoplasmRecruitingPhase 2 / Phase 32025-11-24NCT05770544
DETERMINE Trial Treatment Arm 02: Atezolizumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With High Tumour Mutational Burden (TMB) or Microsatellite Instability-high (MSI-high) or Proven Constitutional Mismatch Repair Deficiency (CMMRD) DispositionDigestive System Diseases, Digestive System Neoplasms, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Skin and Connective Tissue Diseases, Urogenital Diseases, Urogenital Neoplasms, cancer, colonic disorder, colorectal neoplasm, digestive system disorder, digestive system neoplasm, endometrium neoplasm, female reproductive system disorder, female reproductive system neoplasm, immune system disorder, immunoproliferative disorder, intestinal disorder, lymphatic system disorder, lymphoproliferative syndrome, melanocytic neoplasm, melanoma, neoplasm, neuroendocrine neoplasm, primitive neuroectodermal tumor, rectal disorder, skin disorder, skin neoplasm, uterine disorderRecruitingPhase 2 / Phase 32025-11-24NCT05770102
DETERMINE Trial Treatment Arm 01: Alectinib in Adult, Paediatric and Teenage/Young Adult Patients With ALK Positive CancersFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Male Urogenital Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, T-cell non-Hodgkin lymphoma, Urogenital Diseases, Urogenital Neoplasms, adenocarcinoma, anaplastic large cell lymphoma, benign urinary system neoplasm, cancer, carcinoma, epithelial neoplasm, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, kidney cancer, kidney disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumor, renal cell carcinoma, urinary system disorderRecruitingPhase 2 / Phase 32025-11-24NCT05770037
Cardiac Resynchronization Therapy in Previously Untreatable and High Risk Upgrade Patientscardiovascular disorder, heart disorder, heart failureActive, not recruitingNot applicable2025-07-09NCT02922036