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

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.

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