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Study on Tirzepatide for Improving Insulin Production in Patients with Wolfram Syndrome Type 1

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare condition known as Wolfram syndrome type 1. This syndrome is a genetic disorder that can lead to diabetes and other health issues. The study is exploring the use of a medication called tirzepatide, which is also known by its code name LY3298176. Tirzepatide is a solution for injection that is being investigated for its potential to help increase the body's natural production of insulin in patients with Wolfram syndrome type 1.

The purpose of this study is to determine if tirzepatide can effectively improve insulin production in individuals with Wolfram syndrome type 1. Participants in the study will receive tirzepatide injections, which are administered under the skin. The study will compare different doses of tirzepatide, specifically 2.5 mg, 5 mg, and 7.5 mg, to see which is most effective. Some participants may receive a placebo for comparison. The study will last for a period of up to 12 months, during which participants will be monitored to assess their response to the treatment.

Throughout the study, participants will undergo various assessments to evaluate the effects of tirzepatide on their insulin production. The main goal is to see if there is an increase in insulin levels, which will be measured using a specific test. This research aims to provide valuable insights into whether tirzepatide can be a beneficial treatment option for managing Wolfram syndrome type 1, potentially improving the quality of life for those affected by this condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient will have a confirmed diagnosis of Wolfram syndrome type 1. This includes having diabetes diagnosed before the age of 16 and specific genetic mutations.

    The patient must be at least 5 years old and have signed an informed consent form. If applicable, consent from a guardian is also required.

    Women of childbearing age must have a negative pregnancy test and agree to use effective contraception if sexually active.

    The patient must be willing to wear a continuous glucose monitor.

  2. Step 2

    Medication administration

    The patient will receive tirzepatide, which is a solution for injection. The medication is administered subcutaneously, meaning it is injected under the skin.

    The available dosages are 2.5 mg, 5 mg, and 7.5 mg. The specific dosage and frequency will be determined by the study protocol.

  3. Step 3

    Monitoring and evaluation

    Throughout the study, the patient's response to the medication will be monitored. This includes measuring the production of insulin in the body.

    The primary goal is to see if the medication increases insulin production, which is assessed by a specific test called MMTT. A positive response is defined as a certain level of C-peptide, a marker of insulin production, at 90 minutes after the test.

  4. Step 4

    Study duration

    The study is expected to end by July 15, 2025. The recruitment of participants is estimated to start on May 16, 2024.

Who can join the trial?

5 criteria

  • The patient must have a confirmed diagnosis of Wolfram syndrome. This includes having diabetes diagnosed before the age of 16 and specific genetic mutations related to the condition.
  • The patient must be aged 5 years or older.
  • The patient, or their parents or legal guardians, must sign a consent form after understanding the study details. This form is approved by an ethics committee.
  • Women who can have children must have a negative pregnancy test and agree to use effective birth control if they are sexually active.
  • The patient must be willing to wear a device that continuously monitors blood sugar levels.
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Investigated drugs

Tirzepatide is a medication being studied for its potential to help patients with Wolfram syndrome type 1. It works by activating two important receptors in the body, which can help increase the production of insulin. This is important for people with Wolfram syndrome type 1, as they often have problems with insulin production. The goal of using tirzepatide in this trial is to see if it can improve the body's ability to produce insulin on its own.

What is already known about the treatment

Tirzepatide – Tirzepatide is administered as an injection under the skin, typically in the abdomen, thigh, or upper arm. It is currently being studied in clinical trials for its potential use in treating Wolfram syndrome type 1, a rare genetic disorder. The main therapeutic indication being explored is its ability to increase the body's own insulin production in these patients. At the molecular level, tirzepatide works by activating receptors for two hormones, GIP and GLP-1, which help regulate blood sugar levels. It is classified pharmacologically as a dual agonist for these receptors.

Investigated diseases

Wolfram syndrome type 1 – Wolfram syndrome type 1 is a rare genetic disorder that primarily affects the nervous system and the endocrine glands. It is characterized by the onset of diabetes mellitus and optic atrophy, usually during childhood. As the disease progresses, individuals may experience hearing loss, diabetes insipidus, and neurological complications. The condition is caused by mutations in the WFS1 gene, which plays a role in the function of cells in the pancreas and brain. Over time, the symptoms can become more severe, affecting various bodily functions. The progression of the disease can vary significantly among individuals.
Trial detailsLast updated 9 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-514909-64-00Estimated enrolment10 patientsSponsorOspedale San Raffaele S.r.l.

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