Liraglutide Treatment in Patients With Maturity-onset Diabetes of the Young (MODY)

What Was Tested

In this phase 2 trial, researchers set out to test Liraglutide.

The reason for this research study is to better understand the use of liraglutide, a commonly prescribed Type 2 diabetes medication, in patients with a diagnosis of maturity-onset diabetes of the young (MODY).

The investigators are interested in better understanding the way that this drug affects the metabolism and hormone levels of a person with MODY.

Many people with MODY report having gastrointestinal (GI) issues such as an upset stomach.

It had 1 arm (groups): liraglutide treatment (experimental).

The registry classes the interventions as drug.

More on the Plan

The objective of this study is to perform a comprehensive metabolic and hormonal evaluation of individuals with Maturity-onset diabetes of the young (MODY) at baseline and then assess response to initiation of liraglutide.

Select individuals will be consented for human induced pluripotent stem cell (iPSC) generation to identify the cellular basis of enteroendocrine cell (EEC) dysfunction.

The hypothesis is that heterozygous mutations in MODY-related genes contribute to a range of GI pathologies, including regional disruptions in epithelial identity and disturbances in the function of EECs.

Investigators plan to enroll approximately 50 patients with MODY and obtain baseline metabolic labs and imaging along with enteroendocrine labs before initiating liraglutide and assessing response with repeat labs and imaging.

Who Took Part

50 planned participants were the target.

It was open to people of any sex aged from 10 years with no upper age limit.

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

Examples of who could join: Diagnosis of MODY via genetic testing.

The full record lists 2 inclusion and 12 exclusion criteria; among the exclusions: history of anaphylaxis to glp-1 receptor agonists; already taking glp-1 receptor agonists; on medications within the stimulant class.

How the Study Was Run

The set-up was single-group and open-label.

Nothing was hidden in this open-label Liraglutide study: the participants and staff both knew the treatment.

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

It started in 2025-05-08 with completion listed for 2026-08.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Blood sugar control - HbA1C (time frame: Baseline and 3 months after study treatment initiation). BMI (time frame: baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation).

It also tracked 6 secondary outcomes, for example Metabolic - Blood Sugar, Enteroendocrine function - GI symptoms, Enteroendocrine function - insulin, Enteroendocrine function - c-peptide, Body fat percentage and Liver steatosis.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Liraglutide in 50 people.

It is still recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

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