Boston Children's Hospital
Boston Children's Hospital
Boston, Massachusetts, United States343 results · Page 1 of 14
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| THiamine for Recovery In Vulnerable Early-life Infections | Bacterial Infections and Mycoses, Infections, Inflammation, Neuroinflammatory Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Sepsis, Systemic Inflammatory Response Syndrome, bacterial infectious disease, bacterial infectious disease with sepsis, meningitis, nervous system disorder | Not yet recruiting | Phase 2 | 2026-10-09 | NCT07868679 | |
| Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Juvenile Macular Degeneration, Stargardt Disease 1, Stargardt disease, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, macular degeneration, retinal degeneration, retinal disorder | Recruiting | Not stated | 2026-10-09 | NCT06445322 | |
| A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorder | Active, not recruiting | Phase 3 | 2026-10-09 | NCT05444257 | |
| A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy | central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Recruiting | Phase 2 | 2026-10-08 | NCT07287982 | |
| ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Rett syndrome, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT06430385 | |
| A Platform Trial for Pediatric Participants With Obesity or Overweight (LY900040) | Body Weight, Nutritional and Metabolic Diseases, Overweight, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, nutritional disorder, obesity disorder, overnutrition | Recruiting | Phase 3 | 2026-10-07 | NCT06672549 | |
| A Study of Mirikizumab (LY3074828) in Pediatric Participants With Crohn's Disease | Crohn disease, Digestive System Diseases, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorder | Recruiting | Phase 3 | 2026-10-07 | NCT05509777 | |
| A Master Protocol (AMAZ): A Study of Mirikizumab (LY3074828) in Pediatric Participants With Ulcerative Colitis or Crohn's Disease (SHINE-ON) | Crohn disease, Digestive System Diseases, Pediatric ulcerative colitis, colitis, colonic disorder, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorder, pediatric Crohn disease, ulcerative colitis | Recruiting | Phase 3 | 2026-10-07 | NCT04844606 | |
| A Study to Evaluate the Efficacy and Safety of KarXT for the Treatment of Schizophrenia in Adolescents (EMERGENT TEEN) | Schizophrenia Spectrum and Other Psychotic Disorders, psychiatric disorder, schizophrenia | Recruiting | Phase 3 | 2026-10-06 | NCT07288567 | |
| PEEL-224, Vincristine and Temozolomide in Pediatric Solid Tumors | Disease Attributes, Myosarcoma, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, epithelial neoplasm, myomatous neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumor, rhabdomyosarcoma, sarcoma | Recruiting | Phase 1 / Phase 2 | 2026-10-06 | NCT06721689 | |
| A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease | Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Danon disease, Hypertrophy, Hypertrophy, Left Ventricular, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, X-linked disease, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, disorder of glycogen metabolism, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, inborn carbohydrate metabolic disorder, inborn errors of metabolism, intellectual disability, lysosomal storage disease, metabolic disease, nervous system disorder, subvalvular aortic stenosis | Recruiting | Phase 2 | 2026-10-06 | NCT06092034 | |
| ALTERRA Post-Approval Study | Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorder, heart valve disorder, pulmonary valve insufficiency, tetralogy of fallot | Active, not recruiting | Not stated | 2026-10-05 | NCT05378386 | |
| Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Recruiting | Phase 1 | 2026-10-02 | NCT06502171 | |
| Implementation of Whole Genome Sequencing as Screening in a Diverse Cohort of Healthy Infants | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, disease susceptibility, hereditary disease, inherited disease susceptibility | Active, not recruiting | Not applicable | 2026-10-02 | NCT05161169 | |
| Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Malformations of Cortical Development, Group I, Neoplasms, Multiple Primary, brain disorder, central nervous system disorder, central nervous system malformation, cerebral cortical dysplasia, epilepsy, hamartoma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, tuberous sclerosis | Recruiting | Phase 2 | 2026-10-02 | NCT05104983 | |
| Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome | Abnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorder | Active, not recruiting | Phase 3 | 2026-10-02 | NCT05035030 | |
| An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Abnormalities, Digestive System Diseases, bile duct disorder, biliary atresia, biliary tract disorder | Active, not recruiting | Phase 3 | 2026-10-01 | NCT05426733 | |
| Harmony TPV Post-Approval Study | Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorder, heart valve disorder, pulmonary valve insufficiency, tetralogy of fallot | Active, not recruiting | Not applicable | 2026-10-01 | NCT05077774 | |
| A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition | congenital structural myopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Recruiting | Not stated | 2026-09-30 | NCT06581146 | |
| A Study to Find Out How Nerandomilast is Tolerated, Handled by the Body, and if it Helps Children and Adolescents With Interstitial Lung Disease (FIBRONEER-chILD) | Fibrosing Interstitial Lung Disease | Recruiting | Phase 3 | 2026-09-29 | NCT07366034 | |
| Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM) | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, X-linked disease, chromosomal disorder, fragile X syndrome, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder | Recruiting | Phase 2 | 2026-09-29 | NCT07209462 | |
| REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome | 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 disorder | Active, not recruiting | Phase 3 | 2026-09-29 | NCT06914609 | |
| ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis | eye disorder, retinal degeneration, retinal disorder, retinoschisis | Recruiting | Phase 3 | 2026-09-29 | NCT05878860 | |
| Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss | Deafness, Deafness, Autosomal Recessive 9, Ear Diseases, Hearing Loss, Bilateral, Hearing Loss, Sensorineural, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, auditory neuropathy, hearing disorder, hearing loss disorder, nervous system disorder, otorhinolaryngologic disease | Recruiting | Phase 3 | 2026-09-29 | NCT05821959 | |
| A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period | Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, hematologic disorder, metabolic disease, pyruvate kinase deficiency of red cells | Active, not recruiting | Phase 3 | 2026-09-28 | NCT05144256 |