Kennedy Krieger Institute
Kennedy Krieger Institute
Baltimore, Maryland, United States41 results · Page 1 of 2
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Setrusumab vs Placebo for Osteogenesis Imperfecta | Bone Diseases, Developmental, Collagen Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia, osteogenesis imperfecta | Active, not recruiting | Phase 2 / Phase 3 | 2026-10-06 | NCT05125809 | |
| Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 3 | 2026-10-05 | NCT07486934 | |
| Evaluation of SPN-812 (Viloxazine Extended-release Capsule) in Preschool-age Children With ADHD | attention deficit hyperactivity disorder, inattentive type, neurodevelopmental disorder, oppositional defiant disorder, psychiatric disorder | Recruiting | Phase 4 | 2026-10-05 | NCT04781140 | |
| Epidiolex Trial for Presymptomatic Treatment of Sturge-Weber Syndrome | Infarction, Necrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, brain disorder, brain infarction, brain ischemia, brain stem infarction, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, ischemic disease, nervous system disorder, stroke disorder, vascular disorder | Recruiting | Phase 2 | 2026-10-02 | NCT07778810 | |
| Biomarker Validation in Motor System Physiology in Attention Deficit Hyperactivity Disorder | Attention Deficit Hyperactivity Disorder Combined | Recruiting | Phase 4 | 2026-09-25 | NCT04421248 | |
| A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, facioscapulohumeral muscular dystrophy, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 2 | 2026-09-24 | NCT05548556 | |
| Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy | Becker 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 disease | Enrolling by invitation | Phase 2 | 2026-09-17 | NCT06066580 | |
| Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, facioscapulohumeral muscular dystrophy, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Not stated | 2026-09-17 | NCT03458832 | |
| Home Exercise for Individuals With Neurodegenerative Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Signs and Symptoms, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, adrenomyeloneuropathy, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, leukodystrophy, leukoencephalopathy with brain stem and spinal cord involvement-high lactate syndrome, metabolic disease, nervous system disorder, neurodegenerative disease | Recruiting | Not applicable | 2026-09-16 | NCT05443906 | |
| Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I) | autosomal recessive limb-girdle muscular dystrophy type 2I | Active, not recruiting | Phase 3 | 2026-09-09 | NCT05775848 | |
| Defining Endpoints in Becker Muscular Dystrophy | Becker 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 disease | Active, not recruiting | Not stated | 2026-09-03 | NCT05257473 | |
| A Clinical Trial for Self-Injurious Behavior | Self-Injurious Behavior | Active, not recruiting | Not applicable | 2026-08-31 | NCT03995966 | |
| A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, autosomal recessive limb-girdle muscular dystrophy type 2A, autosomal recessive limb-girdle muscular dystrophy type 2I, hereditary disease, limb-girdle muscular dystrophy, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 1 / Phase 2 | 2026-08-26 | NCT05230459 | |
| Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Enrolling by invitation | Phase 3 | 2026-08-19 | NCT07008469 | |
| Measuring Brain Changes Following Cognitive Intervention in Pediatric Patients With Brain Tumors | Neoplasms by Site, brain disorder, central nervous system cancer, central nervous system disorder, neoplasm, nervous system disorder | Not yet recruiting | Not applicable | 2026-08-07 | NCT07558473 | |
| Suramin for the Treatment of Autism Trial: KZ101 in a Male Pediatric Population With Autism Spectrum Disorder (ASD) | autism spectrum disorder, neurodevelopmental disorder, pervasive developmental disorder, psychiatric disorder | Recruiting | Phase 2 | 2026-07-30 | NCT06866275 | |
| Medication Treatment of ADHD in Pediatric Epilepsy | attention deficit hyperactivity disorder, inattentive type, brain disorder, central nervous system disorder, epilepsy, nervous system disorder, neurodevelopmental disorder, oppositional defiant disorder, psychiatric disorder | Recruiting | Phase 4 | 2026-07-29 | NCT07594652 | |
| A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy | Cerebral Adrenoleukodystrophy (cALD) | Active, not recruiting | Phase 3 | 2026-07-29 | NCT05819866 | |
| A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, facioscapulohumeral muscular dystrophy, facioscapulohumeral muscular dystrophy 1, facioscapulohumeral muscular dystrophy 2, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Recruiting | Phase 3 | 2026-07-27 | NCT07038200 | |
| A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Tourette syndrome, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, neurodevelopmental disorder, psychiatric disorder, tic disorder | Active, not recruiting | Phase 3 | 2026-07-22 | NCT06021522 | |
| A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rett syndrome, Signs and Symptoms, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder, neurodevelopmental disorder, psychiatric disorder | Active, not recruiting | Phase 3 | 2026-07-07 | NCT05606614 | |
| Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9 | autosomal recessive limb-girdle muscular dystrophy type 2I | Active, not recruiting | Phase 3 | 2026-07-02 | NCT07678775 | |
| Rett Syndrome Registry | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Rett Syndrome, Atypical, Rett syndrome, Signs and Symptoms, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder, neurodevelopmental disorder, psychiatric disorder | Recruiting | Not stated | 2026-06-30 | NCT05432349 | |
| ARC-IM System to Manage Symptomatic Blood Pressure Instability Secondary to Chronic Spinal Cord Injury | central nervous system disorder, injury, nervous system disorder, nervous system injury, spinal cord disorder, spinal cord injury | Recruiting | Not applicable | 2026-06-16 | NCT07147296 | |
| A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rett syndrome, Signs and Symptoms, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder, neurodevelopmental disorder, psychiatric disorder | Active, not recruiting | Phase 3 | 2026-06-10 | NCT05898620 |