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Dantrolene Sodium in Wolfram Syndrome
NCT02829268Phase 2
Key facts
- Status
- Completed
- Start
- Jan 2017
- Primary completion
- Feb 2023
- Countries
- 1
- Sponsor
- Washington University School of Medicine
- Modality
- Classical Binding Small Molecules
- Drugs
- dantrolene
- Conditions
- Wolfram syndrome
01
Overview
Phase 2, open-label trial of dantrolene in Wolfram syndrome (2 arms), sponsored by Washington University School of Medicine. Primary endpoint: Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests at 6 months. Started Jan 2017; status: completed.
02
Design
2 arms- Study design
- Non-randomizedOpen-label
Pediatric
Pediatric patients treated with dantrolene sodium
Adult
Adult patients treated with dantrolene sodium
03
Who it enrols
Patients with definitive diagnosis of Wolfram syndrome (WFS1 mutations), at least 5 years old. Excludes patients with significant non-Wolfram CNS involvement, mitochondrial myopathy, abnormal liver function, or significant co-morbidities.
04
Endpoints
Primary
Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests
Secondary
Changes in C-peptide Levels
Changes in Visual Functioning
Changes in Best-corrected Visual Acuity
Changes in Neurological Functions
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