Investigating Enteroendocrine Dysfunction, Metabolism, and Response to Liraglutide in Patients With Maturity-onset Diabetes of the Young (MODY)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Blood sugar control - HbA1C
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of MODY via genetic testing
- •HgbA1c >6.5%
排除标准
- •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
研究组 & 干预措施
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.
干预措施: Liraglutide (Drug)
结局指标
主要结局
Blood sugar control - HbA1C
时间窗: Baseline and 3 months after study treatment initiation
Hemoglobin A1c
BMI
时间窗: baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation
Body mass index; weight and height will be combined to report BMI in kg/m\^2
次要结局
- 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)
- Liver steatosis(baseline and 3 months after treatment initiation)
研究者
Mansa
ASSISTANT PROFESSOR
Children's Hospital Medical Center, Cincinnati