A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Status:
Active, not recruiting
Trial end date:
2021-12-01
Target enrollment:
Participant gender:
Summary
Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes
mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration. 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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Washington University School of Medicine
Collaborators:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) National Institutes of Health (NIH)