Liraglutide for MODY

Overseen By Mansa Krishnamurthy, M.D.

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Mansa

Must not be taking: GLP-1 agonists, Stimulants

Disqualifiers: Cancer, Pancreatitis, Kidney disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how liraglutide, a drug commonly used for Type 2 diabetes, can benefit individuals with maturity-onset diabetes of the young (MODY). Researchers aim to determine if liraglutide can improve metabolism and reduce gastrointestinal issues, such as upset stomach, often experienced by MODY patients. They will also study liraglutide's mechanisms by examining cells from participants. The trial seeks individuals with a genetic diagnosis of MODY and an HgbA1c (a measure of blood sugar) over 6.5%. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are already taking GLP-1 receptor agonists or medications within the stimulant class.

Is there any evidence suggesting that liraglutide is likely to be safe for humans?

Research has shown that liraglutide is generally safe for people with diabetes. One study found that taking liraglutide, up to 1.8 mg daily, helped control blood sugar over a year. This study included children and teenagers with Type 2 diabetes, which differs from MODY, but still provides useful safety information.

Another study found that people taking liraglutide had a lower chance of experiencing low blood sugar compared to those taking glimepiride, another diabetes drug. This suggests liraglutide might be safer in some respects.

However, some individuals experience side effects, such as nausea or an upset stomach. Since many people with MODY already have stomach issues, monitoring how liraglutide affects these symptoms is important. The trial allows for dose adjustments if side effects become too uncomfortable.

Overall, while liraglutide is generally safe, it is crucial to observe its effects on each person, especially concerning stomach problems. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for MODY?

Unlike the standard treatments for Maturity-Onset Diabetes of the Young (MODY), which typically involve sulfonylureas or insulin therapies, liraglutide offers a unique approach. Liraglutide is a GLP-1 receptor agonist, which works by mimicking a hormone that increases insulin secretion in response to meals, potentially improving blood sugar control without the risk of causing low blood sugar. Researchers are excited about liraglutide because it not only helps manage glucose levels but may also offer benefits like weight loss, which is not common with traditional diabetes medications. These features could make liraglutide a more holistic approach to managing MODY, addressing both blood sugar levels and weight management.

What evidence suggests that liraglutide might be an effective treatment for MODY?

Research has shown that liraglutide, a medication often used for Type 2 diabetes, might also benefit people with maturity-onset diabetes of the young (MODY). In past studies, liraglutide effectively stabilized blood sugar levels, maintaining glycated hemoglobin below 7.0% for at least a year. Patients experienced fewer digestive problems, which are common with MODY. This improvement likely occurs because liraglutide helps the body manage insulin and digestion. Overall, liraglutide appears promising for controlling both blood sugar and digestive issues in MODY. Participants in this trial will receive liraglutide treatment to further evaluate its effectiveness for MODY. ^{1 2 6 7 8}

Who Is on the Research Team?

Mansa Krishnamurthy, M.D.

Principal Investigator

Children's Hospital Medical Center, Cincinnati

Are You a Good Fit for This Trial?

This trial is for individuals diagnosed with MODY, a form of diabetes often confused with Type 2. Participants should be interested in exploring if the diabetes medication Liraglutide can improve their metabolism and reduce gastrointestinal issues associated with MODY.

Inclusion Criteria

I have been genetically diagnosed with MODY.

HgbA1c >6.5%

Exclusion Criteria

My BMI is under 18.5, or I am a child with a BMI below the 5th percentile.

History of anaphylaxis to GLP-1 receptor agonists

HbA1c > 10.0%

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Evaluation

Comprehensive metabolic and hormonal evaluation of individuals with MODY, including baseline metabolic labs and imaging along with enteroendocrine labs

1 week

Treatment

Participants receive liraglutide with titration: 0.6 mg daily for 1 week, 1.2 mg daily for 1 week, and 1.8 mg daily thereafter. Monitoring includes repeat labs and imaging.

12 weeks

Monthly visits for monitoring and dose adjustment

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of HbA1c, enteroendocrine function, and GI symptoms

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Liraglutide

Trial Overview The study aims to understand how Liraglutide affects people with MODY, particularly its impact on metabolism, hormone levels, and gastrointestinal symptoms. It will also explore why any improvements occur by creating stem cells from participants' cells to study effects on the GI tract.

How Is the Trial Designed?

Group I: liraglutide treatment Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Mansa

Lead Sponsor

Citations

1. clinicaltrials.gov clinicaltrials.gov/search?cond=%22Maturity-Onset%20Diabetes%20of%20the%20Young%22&intr=%22Liraglutide%22&viewType=Table&rank=1

"Maturity-Onset Diabetes of the Young", "Liraglutide" | Card ... It identifies the role of the intervention that participants receive. Types of arms include experimental arm, active comparator arm, placebo comparator arm, ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7458169/

Maturity-Onset diabetes of the young type 5 treated with ... Outcomes: Liraglutide treatment (0.6 mg/d) alone maintained the patient's glycated hemoglobin level below 7.0% for at least 12 months after ...

3. ctv.veeva.com ctv.veeva.com/study/liraglutide-treatment-in-patients-with-maturity-onset-diabetes-of-the-young-mody

Liraglutide Treatment in Patients With Maturity-onset Diabetes ... 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 ...

4. nejm.org nejm.org/doi/full/10.1056/NEJMoa1903822

Liraglutide in Children and Adolescents with Type 2 Diabetes Results from this double-blind, randomized, phase 3 trial showed the superiority of liraglutide to placebo in improving glycemic control at 26 ...

5. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/pediatric/431_22341S31%20liraglutide%20clinical%20bpca.pdf

Untitled - accessdata.fda.gov “including results from the toxicity study of liraglutide in juvenile rats based on effects during ... maturity onset diabetes of the young. • Use ...

6. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK500456/

Maturity-Onset Diabetes of the Young Overview - GeneReviews Studies showed that in HNF1A-MODY nateglinide caused lower postprandial glucose levels and reduced the risk of hypoglycemia compared to the ...

7. aafp.org aafp.org/pubs/afp/issues/2022/0200/p162.html

Maturity-Onset Diabetes of the Young: Rapid Evidence ... Patients taking liraglutide had similar glycemic control as those taking glimepiride but much lower risk of hypoglycemia. Liraglutide may be considered in ...

8. patlynk.com patlynk.com/trial/NCT07029009

Maturity Onset Diabetes of the Young Researchers hope that liraglutide will not only help improve blood sugar levels and weight but also alleviate digestive symptoms that some MODY patients ...