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

Exclusion Criteria

• •1. The patient has clinically significant non-Wolfram related CNS involvement which is judged by the investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
• •2. The patient has a known defect in oxidative phosphorylation (such as a confirmed mitochondrial myopathy)
• •3. The patient has abnormal liver function (defined as serum transaminases more than twice the upper limit of normal for the reference laboratory)
• •4. The patient has a significant medical or psychiatric co-morbidity that might affect study data or confound the integrity of study results.
• •5. The patient has received treatment with any investigational drug within the 30 days prior to study entry.
• •6. The patient has received blood product transfusions within 90 days prior to screening.
• •7. The patient is unable to comply with the protocol, (e.g. has a clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, known clinically significant psychiatric/behavioural instability, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator.
• •8. The patient has a known history of central apnea and/or ventilation requirements.
• •9. The patient has a known history of chronic obstructive pulmonary disease, pleural effusion, and/or myocardial disease.