21
Participants
Start Date
January 31, 2017
Primary Completion Date
February 28, 2023
Study Completion Date
February 28, 2023
dantrolene sodium
"The purpose of this study is to assess the safety and tolerability of dantrolene sodium in patients with Wolfram syndrome. In addition, we will assess the efficacy of dantrolene sodium on the cardinal manifestations of Wolfram syndrome, including visual acuity, remaining beta cell functions, and neurological functions.~There is a screening period up to 56 days, a 6-month treatment period with an optional extension phase up to 24 months, and a 4-week safety follow-up period. Study assessments include medical \& medication history, physical exams, neurological exams, eye exams, endocrine exams, vital signs, height, weight, electrocardiograms, blood and urine tests, pregnancy test if applicable, and questionnaires."
Washington University School of Medicine, St Louis
National Institutes of Health (NIH)
NIH
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Washington University School of Medicine
OTHER