Liraglutide for people with maturity-onset diabetes of the young (MODY)
Investigating Enteroendocrine Dysfunction, Metabolism, and Response to Liraglutide in Patients With Maturity-onset Diabetes of the Young (MODY)
This trial will try liraglutide in people with genetically confirmed MODY to see if it improves blood sugar, body weight, and gastrointestinal symptoms.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 10 Years and up |
| Sex | All |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 1 site (Cincinnati, Ohio) |
| Trial ID | NCT07029009 on ClinicalTrials.gov |
What this trial studies
Participants with genetically confirmed MODY will undergo baseline metabolic, hormonal, and imaging evaluations before starting liraglutide. Investigators will initiate liraglutide and repeat labs and imaging to measure changes in HbA1c, BMI, enteroendocrine biomarkers, and GI symptoms. Up to about 50 participants will be enrolled, and a subset will consent to provide cells for generation of human induced pluripotent stem cells (iPSC) to study cellular mechanisms of enteroendocrine dysfunction. The study tests the hypothesis that heterozygous MODY gene variants contribute to GI and enteroendocrine abnormalities that may respond to GLP‑1 receptor agonist therapy.
Who should consider this trial
Good fit: People with a genetic diagnosis of MODY and HbA1c between 6.5% and 10% who are not already taking GLP‑1 receptor agonists and who do not have contraindications such as recent DKA, pancreatitis, active kidney disease, pregnancy, prior bariatric surgery, very low BMI, or family/personal history of medullary thyroid cancer would be ideal candidates.
Not a fit: Patients already using GLP‑1 receptor agonists, those with HbA1c over 10% or recent DKA, active kidney disease, prior pancreatitis, relevant cancer risk, recent bariatric surgery, pregnancy, or very low BMI are unlikely to be eligible or to benefit from this protocol.
Why it matters
Potential benefit: If successful, liraglutide could offer people with MODY better glucose control, possible weight benefit, and relief of gastrointestinal symptoms.
How similar studies have performed: GLP‑1 receptor agonists are proven to improve glycemia and reduce weight in type 2 diabetes, but their use specifically in MODY has limited evidence and is largely supported by small case reports and emerging data.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Cincinnati, Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
Study contacts
- Principal investigator: Mansa Krishnamurthy, M.D. — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Mansa Krishnamurthy, M.D.
- Email: mansa.krishnamurthy@cchmc.org
- Phone: 513-636-4744
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.