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

NCT ID: NCT07029009

Last Updated: 2025-12-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-05-08

Study Completion Date

2027-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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. Investigators also are interested in finding out if this drug will help with GI issues.

If liraglutide does help with this symptom of MODY, the investigators want to know why this happens. If this drug is effective for participants, the investigators will use participants cells to make human induced pluripotent stem cell (iPSC). This means that the investigators will use participant cells to create what are called stem cells, which are cells in the body that are able to be told what their job is. Investigators will use these cells to see what happens in gastrointestinal (GI) tract.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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. Thus, patients with MODY may experience improvement not only in HbA1c and BMI but also in GI symptoms with initiation of GLP-1 receptor agonists. 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. This project will provide further guidance for clinical care and treatment of patients with MODY.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Maturity Onset Diabetes of the Young

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

liraglutide treatment

After baseline evaluation of enteroendocrine levels and metabolism, study participants will be prescribed liraglutide. Titration of liraglutide will be as follows: 1) Starting dose of liraglutide 0.6 mg daily for 1 week, 2) Increase to liraglutide 1.2 mg daily for 1 week, and 3) Maintain at final dose of liraglutide 1.8 mg daily. If study participants are unable to tolerate liragultide 1.8 mg daily due to side effects for 1 week, then will decrease back to liraglutide 1.2 mg daily.

Group Type EXPERIMENTAL

Liraglutide

Intervention Type DRUG

Liraglutide will be prescribed following FDA approved packet insert for type 2 diabetes in our test population.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Liraglutide

Liraglutide will be prescribed following FDA approved packet insert for type 2 diabetes in our test population.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Victoza Saxenda

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Diagnosis of MODY via genetic testing
* HgbA1c \>6.5%

Exclusion Criteria

* History of anaphylaxis to GLP-1 receptor agonists
* Already taking GLP-1 receptor agonists
* on medications within the stimulant class
* have had bariatric surgery
* personal or family history of cancer, especially medullary thyroid cancer
* personal history of pancreatitis or pathogenic variants associated with increased risk of pancreatitis
* known active kidney disease
* severe hypoglycemia (glucose \< 50 mg/dL) within 3 months of study enrollment
* HbA1c \> 10.0%
* episode of diabetic ketoacidosis (DKA) in the past 3 months
* are currently pregnant
* BMI \< 18.5 kg/m2 and pediatric patients with BMI \< 5th percentile
Minimum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Mansa

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Mansa

ASSISTANT PROFESSOR

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Mansa Krishnamurthy, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United States

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Mansa Krishnamurthy, M.D.

Role: CONTACT

513-636-4744

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Lily Deng

Role: primary

513-636-0002

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2024-0426

Identifier Type: -

Identifier Source: org_study_id