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

NCT07029009 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 2024-0426
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Maturity Onset Diabetes of the Young

Outcome Measures

Primary Outcomes (2)

• Blood sugar control - HbA1C

  Hemoglobin A1c

  Baseline and 3 months after study treatment initiation

• BMI

  Body mass index; weight and height will be combined to report BMI in kg/m\^2

  baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation

Secondary Outcomes (6)

• Metabolic - Blood Sugar

  Baseline through three months of study treatment

• Enteroendocrine function - GI symptoms

  baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation

• Enteroendocrine function - insulin

  baseline and 3 months after study treatment initiation

• Enteroendocrine function - c-peptide

  baseline and 3 months after study treatment initiation

• Body fat percentage

  baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation

Study Arms (1)

liraglutide treatment

EXPERIMENTAL

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.

Drug: Liraglutide

Interventions

Liraglutide DRUG

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

Also known as: Victoza, Saxenda

liraglutide treatment

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Mansa: lead

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

MeSH Terms

Conditions

Diabetes Mellitus, Type 2

Interventions

Liraglutide

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Glucagon-Like Peptide 1; Glucagon-Like Peptides; Proglucagon; Gastrointestinal Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists

Study Officials

• Mansa Krishnamurthy, M.D.

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mansa Krishnamurthy, M.D.

CONTACT

513-636-4744; mansa.krishnamurthy@cchmc.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: ASSISTANT PROFESSOR

Study Record Dates

• First Submitted: August 19, 2024
• First Posted: June 19, 2025
• Study Start: May 8, 2025
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): January 1, 2027
• Last Updated: December 17, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)