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Showing 1–15 of 15 matching trials from the live ClinicalTrials.gov search.

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Filters: Condition: Neurogenetic Disorders Clear all

Conditions: Baker Gordon Syndrome, Rare Neurodevelopmental Conditions, Rare Neurogenetic Conditions, Syt-1 Disorder, Epilepsy, Seizure, Genetic Mutations, Autism in Children, Developmental Delay (Disorder)
Interventions: No Intervention: Observational Cohort; Other
Lead sponsor: University of Missouri-Columbia; Other
Eligibility: Up to 99 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: Columbia, Missouri

Conditions: HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50, Hereditary Spastic Paraplegia Type 47, Hereditary Spastic Paraplegia Type 51, Hereditary Spastic Paraplegia Type 52, SPG47, AP4B1, Neurogenetic Disorders, Neurodevelopmental Conditions, Movement Disorders, Gene Therapy
Interventions: BFB-101 (AAV9-CBh-AP4B1); Biological
Lead sponsor: BlackfinBio Ltd; Industry
Eligibility: 12 Months to 60 Months
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Alzheimer's Disease
Interventions: NGP 555; Drug
Lead sponsor: NeuroGenetic Pharmaceuticals Inc; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015
U.S. locations: 1
States / cities: Cypress, California

Conditions: Neurodevelopmental Disorders, Intellectual Disability, Developmental Delay, Autism Spectrum Disorder, Seizures, Hypertonia, Muscle, Hypotonia
Interventions: Not listed
Lead sponsor: Columbia University; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2030
U.S. locations: 1
States / cities: New York, New York

Conditions: Alzheimer Disease
Interventions: rosiglitazone; Drug
Lead sponsor: US Department of Veterans Affairs; Federal
Eligibility: 55 Years to 80 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2003
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Neurogenetic Disorders
Interventions: Not listed
Lead sponsor: University of Pittsburgh; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2028
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Epilepsy in Children, Dyskinesias, Movement Disorders in Children, Neurologic Disorder, Chorea, Myoclonus, Ataxia, Epilepsy, Dystonia Disorder, Movement Disorders
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 0 Years to 18 Years
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Alzheimer's Disease
Interventions: NGP 555; Drug
Lead sponsor: NeuroGenetic Pharmaceuticals Inc; Industry
Eligibility: 40 Years to 65 Years
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016
U.S. locations: 1
States / cities: Cypress, California

Conditions: Spinal Muscular Atrophy (SMA)
Interventions: onasemnogene abeparvovec; Biological
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 0 Years to 100 Years
Enrollment: 175 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2031
U.S. locations: 1
States / cities: Norfolk, Virginia

Conditions: Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies, Inherited Neuromuscular Conditions
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: 1 Day to 100 Years
Enrollment: 2,323 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 2012
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Healthy Children, Children With Neurodevelopmental Disorders, Children With Neuropsychiatric Disorders, Children With Behavioral Syndromes
Interventions: Not listed
Lead sponsor: National Institute of Mental Health (NIMH); NIH
Eligibility: 6 Months to 8 Years
Enrollment: 244 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2027
U.S. locations: 5
States / cities: Bethesda, Maryland • Boston, Massachusetts • New York, New York + 2 more

Conditions: Charcot-Marie-Tooth Disease
Interventions: FLX-787-ODT (orally disintegrating tablet), Placebo ODT; Drug
Lead sponsor: Flex Pharma, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 20
States / cities: Scottsdale, Arizona • Aurora, Colorado • New Britain, Connecticut + 16 more

Conditions: Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions: Parent-Infant Inter(X)action Intervention (PIXI); Behavioral
Lead sponsor: RTI International; Other
Eligibility: Up to 99 Years
Enrollment: 120 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

Conditions: Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle, Motor Neuron Disease, Movement Disorders
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 1 Month to 30 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Epilepsy-Dyskinesia, Epilepsy, Dyskinesia, EDS, Epilepsy-Dyskinesia Syndomes, Epilepsy in Children, Dyskinesias, Movement Disorders in Children, Neurologic Disorder, Chorea, Myoclonus, Ataxia, Dystonia Disorder, Movement Disorders
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 0 Years to 30 Years
Enrollment: 700 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

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Investigating Phenotypic, Epigenetic, and NeuroGenetic Traits in Rare and Ultra-rare Neurodevelopmental Disorders (Project PENGUIN)

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

Neurogenetic Pharmaceuticals (NGP) 555 in Healthy Young Volunteers (Single-ascending Dose)

Longitudinal Study of Neurogenetic Disorders

Insulin, Neurogenetics and Memory in Alzheimer's Disease

Neurogenetics Patient Registry

A Retrospective Survey-based Multicenter Study to Delineate the Molecular and Phenotypic Spectrum of Epilepsy-dyskinesia Syndromes

Neurogenetic Pharmaceuticals (NGP) 555 in Healthy Volunteers (14 Day Multiple Ascending Dose)

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Genetic and Physical Study of Childhood Nerve and Muscle Disorders

Electrophysiologic Sleep Phenotyping and Sleep-Dependent Neuro-maturation in Clinical and Healthy Pediatric Populations

A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.

Parent and Infant Inter(X)Action Intervention (PIXI)

Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)

Registry and Natural History of Epilepsy-Dyskinesia Syndromes