A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

21

Participants

Timeline

Start Date

January 31, 2017

Primary Completion Date

February 28, 2023

Study Completion Date

February 28, 2023

Conditions
Wolfram SyndromeDiabetes MellitusOptic Nerve AtrophyAtaxia
Interventions
DRUG

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."

Trial Locations (1)

63110

Washington University School of Medicine, St Louis

All Listed Sponsors
collaborator

National Institutes of Health (NIH)

NIH

collaborator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

lead

Washington University School of Medicine

OTHER

NCT02829268 - A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome | Biotech Hunter | Biotech Hunter