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ClinicalTrials.gov public records Last synced Nov 9, 2026, 12:25 AM EST

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Showing 1–24 of 1,272 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Heredodegenerative Disorders, Nervous System Clear all

Conditions: Parkinson's Disease
Interventions: Not listed
Lead sponsor: University of Washington; Other
Eligibility: 18 Years and older
Enrollment: 5,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2026
U.S. locations: 5
States / cities: Atlanta, Georgia • Cincinnati, Ohio • Portland, Oregon + 1 more

Conditions: Fragile X Syndrome, Autism Spectrum Disorder, Autistic Disorder, Asperger Syndrome
Interventions: Alpha Auditory Entrainment, Sham; Other
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 5 Years to 10 Years
Enrollment: 180 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2028
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Acoustic Schwannoma, Adult Anaplastic Meningioma, Adult Ependymoma, Adult Grade I Meningioma, Adult Grade II Meningioma, Adult Meningeal Hemangiopericytoma, Adult Papillary Meningioma, Neurofibromatosis Type 1, Neurofibromatosis Type 2, Recurrent Adult Brain Tumor
Interventions: bevacizumab; Biological
Lead sponsor: Northwestern University; Other
Eligibility: 18 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2018
U.S. locations: 5
States / cities: Chicago, Illinois • Boston, Massachusetts • New York, New York + 2 more

Conditions: Neurofibromatosis Type 1
Interventions: Lovastatin ™, placebo; Drug · Device
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: 8 Years to 15 Years
Enrollment: 146 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2016
U.S. locations: 11
States / cities: Birmingham, Alabama • Los Angeles, California • Washington D.C., District of Columbia + 8 more

Conditions: Myotonic Dystrophy Type 1 (DM1)
Interventions: DYNE-101, Placebo; Drug
Lead sponsor: Dyne Therapeutics; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 127 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2029
U.S. locations: 8
States / cities: Stanford, California • Gainesville, Florida • Indianapolis, Indiana + 5 more

Conditions: Schwannoma, Vestibular, Neurofibromatosis 2, Meningioma
Interventions: Not listed
Lead sponsor: House Ear Institute; Other
Eligibility: 5 Years and older
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2002
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases, Mitochondrial Diseases, Mitochondrial Dysfunction, Genetic Disease
Interventions: ATI-105, Placebo; Drug
Lead sponsor: Adjuvia Therapeutics, Inc.; Industry
Eligibility: 18 Years to 60 Years
Enrollment: 60 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Interventions: Everolimus; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 3 Years and older
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2014
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Rett Syndrome, Prader-Willi Syndrome, Angelman Syndrome, Sleep Problems
Interventions: Not listed
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 1 Year to 18 Years
Enrollment: 804 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2013
U.S. locations: 9
States / cities: Birmingham, Alabama • Irvine, California • San Diego, California + 6 more

Conditions: Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions: Family Centered pediatric palliative care for family caregivers of children with rare diseases.; Behavioral
Lead sponsor: Children's National Research Institute; Other
Eligibility: 12 Months to 99 Years
Enrollment: 480 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2029
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Neurofibromas, Cutaneous, Neurofibromatosis 1
Interventions: Kybella; Drug
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 18 Years to 85 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Rett Syndrome
Interventions: Dextromethorphan; Drug
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 2 Years to 15 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2010
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Huntington Disease
Interventions: SPK-10001, Placebo Surgery Control; Genetic · Other
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 25 Years to 65 Years
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2035
U.S. locations: 5
States / cities: Boston, Massachusetts • Cincinnati, Ohio • Columbus, Ohio + 2 more

Conditions: Solid Tumors
Interventions: thioureidobutyronitrile; Drug
Lead sponsor: Cellceutix Corporation; Industry
Eligibility: 18 Years and older
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Tourette Syndrome
Interventions: Medtronic Percept Neurostimulator; Device
Lead sponsor: University of Florida; Other
Eligibility: 18 Years to 85 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: MPNST, NF1, Sarcoma
Interventions: Selumetinib + ZEN-3694 ± Durvalumab; Combination Product
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: 18 Years to 99 Years
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 1
States / cities: Birmingham, Alabama

Conditions: Fragile X Syndrome
Interventions: BPN14770/ zatolmilast, Placebo; Drug
Lead sponsor: Tetra Discovery Partners; Industry
Eligibility: 18 Years to 45 Years · Male only
Enrollment: 171 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 17
States / cities: Irvine, California • Orange, California • Sacramento, California + 14 more

Conditions: Fragile X Syndrome, Genetic Diseases
Interventions: Lovastatin, Placebo; Drug · Other
Lead sponsor: University of California, Davis; Other
Eligibility: 10 Years to 17 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 1
States / cities: Sacramento, California

Conditions: Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Neurofibromatosis Type 1, Recurrent Visual Pathway Glioma, Refractory Neurofibromatosis Type 1, Refractory Visual Pathway Glioma
Interventions: Biospecimen Collection, Selumetinib; Procedure · Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 3 Years to 21 Years
Enrollment: 217 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2027
U.S. locations: 17
States / cities: Los Angeles, California • Palo Alto, California • San Francisco, California + 11 more

Conditions: Renal Transplant
Interventions: Arm 1 Everolimus/Reduced dose tacrolimus; Drug
Lead sponsor: University of Toledo Health Science Campus; Other
Eligibility: 18 Years and older
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2020
U.S. locations: 1
States / cities: Toledo, Ohio

Conditions: Huntington Disease
Interventions: Tominersen, Placebo; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 25 Years to 50 Years
Enrollment: 301 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 18
States / cities: Birmingham, Alabama • Phoenix, Arizona • Sacramento, California + 15 more

Conditions: Vestibular Schwannoma, NF2, Neurofibromatosis 2, Acoustic Neuroma, Auditory Tumor
Interventions: lapatinib; Drug
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins; Other
Eligibility: 18 Years and older
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2014
U.S. locations: 7
States / cities: Los Angeles, California • Baltimore, Maryland • Boston, Massachusetts + 3 more

Conditions: Neurofibromatosis Type 1
Interventions: Frameshift Array blood sample test; Genetic
Lead sponsor: Children's National Research Institute; Other
Eligibility: 1 Day to 30 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 3
States / cities: Washington D.C., District of Columbia • Atlanta, Georgia • Dallas, Texas

Conditions: Cerebral Adrenoleukodystrophy (CALD)
Interventions: Lenti-D Drug Product (eli-cel); Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: Up to 17 Years · Male only
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2021
U.S. locations: 3
States / cities: Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota

Search by objective public record fields.

The Parkinson's Genetic Research Study

Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders

Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas

A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

NF2 Natural History Consortium

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults

Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex

Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW

Palliative Care Needs of Children With Rare Diseases and Their Families

Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)

Trial of Dextromethorphan in Rett Syndrome

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease

A Safety, Pharmacokinetic and Pharmacodynamic Study of Kevetrin in Patients With Advanced Solid Tumors

Tourette Deep Brain Stimulation (DBS) Target Detection & Suppression

Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome

Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma

Evaluation of Calcineurin-inhibitor Reduction With Conversion at 2 Months to Everolimus/Reduced Tacrolimus in Renal Transplant Recipients Following Campath® Induction

GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease

Concentration and Activity of Lapatinib in Vestibular Schwannomas

Frameshift Peptides of Children With NF1

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)