Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1

What Was Tested

The trial (phase 2) is built around Tirzepatide.

Wolfram syndrome (WFS:OMIM 222300) is a group of inherited disorders that usually appear in childhood and cause diabetes, optic atrophy leading to loss of vision, deafness and often diabetes insipidus.

Wolfram syndrome affected no more than 0.2 in 10,000 people in the European Union.

It had 1 arm (groups): Interventional group (experimental).

The registry classes the interventions as drug.

Who Took Part

10 planned participants were the target.

It was open to people of any sex aged from 5 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: A definitive diagnosis of Wolfram syndrome, as determined by the following:. Documented diabetes mellitus diagnosed under 16 completed years according to WHO or ADA criteria AND. Aged 5 years or older;. Patient willing to wear a continuous glucose monitor.

The full record lists 7 inclusion and 11 exclusion criteria; among the exclusions: A history of pancreatitis;; pre-existing thyroid disease;; A personal or family history of medullary thyroid carcinoma;.

How the Study Was Run

It was run as a single-group and open-label study.

Everyone knew when Tirzepatide was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2024-01-01 with completion listed for 2024-10.

It ran at 1 site in Italy.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: changing in endogenous insulin production (time frame: [Time Frame: month 6±1, month 12±1]). changing in insulin production (time frame: [Time Frame: month 6±1, month 12±1]).

It also tracked 5 secondary outcomes, for example glucose variability, glycemic variability, change in time in range, change in insulin requirements and change in HbA1c.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Tirzepatide in 10 people.

Its size is small, so on its own it can only give an early look.

Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Tirzepatide compound page, all human trials.