Clinical Trials Funded by the National Institute of Neurological Disorders and Stroke



  • An Exploratory Phase II Study to Determine the Tolerability, Safety, and Activity of a Novel Vasopressin 1a Receptor Antagonist (SRX246) in Irritable Subjects With Huntington’s Disease (HD) (website)
  • Primary Prevention of Stroke in Children With Sickle Cell Disease in Sub-Saharan Africa II (website)


  • A Phase I Study of Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy (website)
  • Exercise intervention for slowing HD progression (website)
  • The effect of vitamin d3 on markers of oxidative stress in boys with x-linked ALD (website)
  • Tolerability of srx246 in Huntington’s disease patients (website)


  • A Phase II Trial of Rituximab In Myasthenia Gravis (website)


  • A Dose-Escalation, Safety And Feasibility Study Of Enteral Levetiracetam For Seizure Control In Pediatric Cerebral Malaria (website)
  • Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen (website)
  • Phase I/IIa Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA (website)
  • Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency (website)
  • Sickle Cell Disease – Stroke Prevention in Nigeria Trial (website)
  • Transcranial Magnetic Stimulation (TMS) in Spino-Cerebellar Ataxia (website)