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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study of MZE782 in Adults With PKUBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuriaRecruitingPhase 22026-10-08NCT07694440
An Open-label Study of XEN1101 in EpilepsyNeurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Tonic-Clonic Seizures, brain disorder, central nervous system disorder, epilepsy, focal epilepsy, nervous system disorder, visual epilepsyEnrolling by invitationPhase 32026-10-08NCT05718817
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
A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Metabolic Dysfunction-Associated Steatohepatitis (MASH) in Adult Participants at Increased Genetic Risk for This ConditionDigestive System Diseases, fatty liver disease, liver disorder, metabolic dysfunction-associated steatotic liver diseaseRecruitingPhase 22026-10-08NCT05519475
A Study to Confirm Safety and Efficacy of Lecanemab in Participants With Early Alzheimer's DiseaseAlzheimer disease, Cognition Disorders, Cognitive Dysfunction, brain disorder, central nervous system disorder, cognitive disorder, dementia, nervous system disorder, neurodegenerative disease, psychiatric disorder, tauopathyActive, not recruitingPhase 32026-10-06NCT03887455
Cognitive and Behavioral Approaches to Reduce Binge Eating and Excess Weight in Adolescents (Online)Body Weight, Feeding and Eating Disorders, Hyperphagia, Nutritional and Metabolic Diseases, Overweight, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Signs and Symptoms, Digestive, binge eating disorder, nutritional disorder, obesity disorder, overnutrition, psychiatric disorderNot yet recruitingNot applicable2026-10-05NCT07856784
Finding Immune Nascent Type 1 DiabetesNutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitusRecruitingNot applicable2026-10-02NCT07663136
Effects of Dietary Phosphorus on Phosphorus and Calcium Whole-Body Balance and Kinetics in Moderate CKDCKDNot yet recruitingNot applicable2026-10-02NCT06712719
A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing CholangitisCholangitis, Sclerosing, Digestive System Diseases, bile duct disorder, biliary tract disorder, cholangitis, primary sclerosing cholangitisRecruitingPhase 32026-10-01NCT07387549
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary AtresiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Abnormalities, Digestive System Diseases, bile duct disorder, biliary atresia, biliary tract disorderActive, not recruitingPhase 32026-10-01NCT05426733
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseActive, not recruitingPhase 1 / Phase 22026-09-23NCT05481879
Bionic Pancreas in CFRDcystic fibrosis-related diabetesActive, not recruitingPhase 32026-09-18NCT06449677
Study to Assess the Use of Tezampanel for Opioid Withdrawal Syndrome in Treatment-Seeking Patients With Opioid Use DisorderChemically-Induced Disorders, Narcotic-Related Disorders, Opioid Use Disorder, opiate dependence, psychiatric disorder, substance-related disorderRecruitingPhase 12026-09-17NCT06538558
Open-Label Extension of EDG-5506 in Participants With Becker Muscular DystrophyBecker muscular dystrophy, 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 diseaseEnrolling by invitationPhase 22026-09-17NCT06066580
Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant DiseasesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, hematologic disorder, hereditary disease, inherited hemoglobinopathy, metabolic diseaseRecruitingPhase 1 / Phase 22026-09-16NCT00920972
A Study to Evaluate the Safety and Efficacy of Ruxolitinib Cream in Pediatric Participants With Nonsegmental VitiligoNonSegmental VitiligoRecruitingPhase 32026-09-14NCT06548360
Positron Emission Tomography Using 64Cu-SAR-bisPSMA in Participants With High-risk Prostate Cancer Prior to 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 disorderRecruitingPhase 32026-09-11NCT06056830
Safety and Efficacy of OTL78 (Zopocianine)Prostate Cancer Metastatic Disease; Prostate Cancer SurgeryNot yet recruitingPhase 2 / Phase 32026-09-10NCT07725523
Assessment of Worldwide Acute Kidney Injury Epidemiology in Neonates 2.0Chronic Disease, Disease Attributes, Edema, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Obstetric Labor Complications, Obstetric Labor, Premature, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Premature Birth, Signs and Symptoms, Urogenital Diseases, acute kidney injury, cardiovascular disorder, chronic renal failure syndrome, hypertensive disorder, kidney disorder, kidney failure, pregnancy disorder, urinary system disorder, vascular disorderEnrolling by invitationNot stated2026-09-04NCT06823115
Surveillance HeartCare® Outcomes RegistryHeart Transplant RejectionActive, not recruitingNot stated2026-09-03NCT03695601
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuriaRecruitingPhase 32026-08-28NCT06302348
Observational Study of Pediatric Rheumatic Diseases: The CARRA RegistrySkin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, Spondylarthritis, Spondylarthropathies, arthritic joint disease, arthropathy, autoimmune disease, bone disorder, connective tissue disorder, immune system disorder, juvenile idiopathic arthritis, musculoskeletal system disorder, negative rheumatoid factor polyarthritis, psoriasis, psoriatic arthritis, rheumatic disorder, skin disorder, spondylitis, vertebral column disorderRecruitingNot stated2026-08-27NCT02418442
Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmRecruitingPhase 32026-08-24NCT05183035
The Gut Microbiome in Type 1 Diabetes and Mechanism of Metformin ActionBody Weight, Nutritional and Metabolic Diseases, Overweight, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, nutritional disorder, obesity disorder, overnutrition, type 1 diabetes mellitusRecruitingPhase 22026-08-21NCT05414409
Interaction of CYP2B6 Genotype and Efavirenz With Methadone and Tizanidine PKDrug-Drug InteractionRecruitingEarly phase 12026-08-20NCT05789173