Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1

Launched by OSPEDALE SAN RAFFAELE · Dec 12, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a medication called tirzepatide for patients with Wolfram Syndrome Type 1, a rare genetic condition that causes diabetes, vision loss, and other serious health issues. The goal is to see if tirzepatide can help increase the body's insulin production and improve blood sugar control in these patients. This trial is part of an effort to provide better treatment options for people with this challenging condition, as there is currently no cure.

To participate in the trial, applicants must be at least 5 years old and have a confirmed diagnosis of Wolfram Syndrome, which includes having diabetes diagnosed before age 16 and specific genetic mutations. Participants will be closely monitored and may need to wear a continuous glucose monitor to track their blood sugar levels throughout the study. This trial aims to offer hope for those affected by Wolfram Syndrome and to better understand how to manage this complex disease. If you or someone you know is interested in learning more about participating, please reach out for additional information.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• 1. A definitive diagnosis of Wolfram syndrome, as determined by the following:
• • 1. Documented diabetes mellitus diagnosed under 16 completed years according to WHO or ADA criteria AND
• • 2. Documented functionally relevant recessive mutations on both alleles of the WFS1 gene or dominant mutation on one allele of the WFS1 gene based on historical test results (if available) or from a qualified laboratory at screening;
• • 2. Aged 5 years or older;
• • 3. The patient, patient's parent(s), or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. The guardians' consent and patient's assent, as relevant, must be obtained;
• • 4. Females of child bearing potential will only be included after a negative highly sensitive urine pregnancy test. If sexually active, they must agree to use a highly effective contraception measure;
• • 5. Patient willing to wear a continuous glucose monitor.
• 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).