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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination TherapyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingPhase 32026-10-09NCT05444257
The AIRTIVITY™ Study: A Study to Find Out Whether BI 1291583 Helps People With Bronchiectasisbronchial disorder, bronchiectasis, respiratory system disorderRecruitingPhase 32026-10-01NCT06872892
Randomized Study in Children and Adolescents With Migraine: Acute TreatmentEar Diseases, Nausea, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Photophobia, Sensation Disorders, Signs and Symptoms, Signs and Symptoms, Digestive, brain disorder, central nervous system disorder, eye disorder, headache disorder, hearing disorder, hyperacusis, hypnic headache, migraine disorder, nervous system disorder, otorhinolaryngologic disease, vision disorderRecruitingPhase 32026-09-24NCT04649242
Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial DiseaseNutritional and Metabolic Diseases, inborn mitochondrial metabolism disorder, metabolic diseaseRecruitingPhase 22026-09-16NCT07514338
Prospective Natural History Study of POLG DiseaseChronic Disease, Disease Attributes, Dyskinesias, Neurologic Manifestations, Nutritional and Metabolic Diseases, Ocular Motility Disorders, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cranial nerve neuropathy, epilepsy, eye disorder, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, metabolic disease, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, ophthalmoplegia, palsy, progressive external ophthalmoplegia, visual epilepsyRecruitingNot stated2026-09-15NCT07775872
I-DECIDE After Bronchiolitis HospitalizationBronchiolitis AcuteRecruitingNot applicable2026-09-10NCT07243652
A Study of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the KneeOsteochondritis, bone disorder, musculoskeletal system disorder, osteochondritis dissecansRecruitingPhase 32026-09-10NCT03588975
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)Abnormalities, 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 22026-09-09NCT07157254
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
Third Party Viral Specific T-cells (VSTs)DNA Virus Infections, Epstein-Barr virus infection, Herpesviridae infectious disease, Infections, Tumor Virus Infections, adenoviridae infectious disease, viral infectious diseaseRecruitingPhase 22026-09-04NCT02532452
Donor-Derived Viral Specific T-cells (VSTs)DNA Virus Infections, Epstein-Barr virus infection, Herpesviridae infectious disease, Infections, Tumor Virus Infections, adenoviridae infectious disease, viral infectious diseaseRecruitingPhase 1 / Phase 22026-09-04NCT02048332
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular DystrophyCongenital, 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 32026-08-28NCT05126758
Post Approval Study for Treatment of Drug-resistant Adult and Pediatric Primary FSGS Using the LIPOSORBER® LA-15 SystemFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Diseases, focal segmental glomerulosclerosis, glomerulonephritis, kidney disorder, nephritis, urinary system disorderRecruitingNot applicable2026-08-28NCT04065438
Post Approval Study of Liposorber LA-15 System for the Treatment of Focal Segmental Glomerulosclerosis in ChildrenDisease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, Urogenital Diseases, focal segmental glomerulosclerosis, glomerulonephritis, kidney disorder, nephritis, urinary system disorderRecruitingNot applicable2026-08-28NCT02235857
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
Pro-2-Cool Pivotal Trial IIBrain Concussion, Brain Injuries, Traumatic, Concussion, Mild, Craniocerebral Trauma, Head Injuries, Closed, Wounds, Nonpenetrating, brain disorder, brain injury, central nervous system disorder, injury, nervous system disorder, nervous system injuryRecruitingNot applicable2026-08-12NCT06929923
International Rare Brain Tumor RegistryBCOR ITD sarcoma, Disease Attributes, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, astroblastoma, central nervous system sarcoma, epithelial neoplasm, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingNot stated2026-08-07NCT05697874
ARDS in Children and ECMO Initiation Strategies Impact on Neurodevelopment (ASCEND)Respiration Disorders, adult acute respiratory distress syndrome, lung disorder, respiratory system disorderActive, not recruitingNot stated2026-07-14NCT05388708
An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)Acid-Base Imbalance, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, MELAS syndrome, Mitochondrial encephalopathy, Nutritional and Metabolic Diseases, acidosis disorder, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, lactic acidosis, metabolic disease, mitochondrial encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, vascular disorderActive, not recruitingPhase 22026-07-07NCT06961344
A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to GrowCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Musculoskeletal Abnormalities, Noonan syndrome, Sex Chromosome Disorders, Skin and Connective Tissue Diseases, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, cardiovascular disorder, chromosomal disorder, congenital heart disease, connective tissue disorder, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, musculoskeletal system disorder, sex chromosome disorder of sex developmentActive, not recruitingPhase 32026-06-12NCT05330325
HeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal TumorsNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, epithelial neoplasm, glioma, medulloblastoma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 42026-06-03NCT02875314
A Study of Uveitis in Children <18 Years of Ageeye disorder, uveal disorder, uveitisRecruitingNot stated2026-05-08NCT07518134
Safety Study of Unlicensed IND Cord Blood Units Manufactured by the National Cord Blood Program for Unrelated TransplantationInfusion ReactionsRecruitingPhase 22026-04-13NCT01656603
LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell HistiocytosisHemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, histiocytosis, interstitial lung disease, lung disorder, lymphatic system disorder, respiratory system disorderActive, not recruitingPhase 2 / Phase 32026-04-09NCT02205762
Targeted Reversal of Inflammation in Pediatric Sepsis-induced MODSPediatric Sepsis-induced Multiple Organ Dysfunction Syndrome (MODS)RecruitingPhase 2 / Phase 32026-03-13NCT05267821