Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1

Exclusion Criteria

• •1. 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. A history of pancreatitis;
• •3. Pre-existing thyroid disease;
• •4. A personal or family history of medullary thyroid carcinoma;
• •5. Multiple Endocrine Neoplasia syndrome type 2;
• •6. Active liver or renal disease, personal or family history of liver/kidney dysfunction related to known genetic disorders;
• •7. Treatment with any investigational drug within the 30 days prior to Trial entry;
• •8. Current therapy with of GLP-1 agonist or DDP-4 inhibitor or a known hypersensitivity to GLP-1 agonist;
• •9. Any other acute or chronic medical, psychiatric, social situation or laboratory result that, based on investigator's judgment, would jeopardize patient safety during trial participation, cause inability to comply with the protocol, or affect the Trial outcome;
• •10. Breastfeeding;
• •11. Pre-existing ocular disease (corneal or lens diseases and any other retinal or optic nerve non-Wolfram related diseases).