Maturity Onset Diabetes of the YoungPhase 2NCT07029009
Liraglutide Treatment in Patients With Maturity-onset Diabetes of the Young (MODY)
A Phase 2 study testing Liraglutide in people with Maturity Onset Diabetes of the Young, run by Mansa at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Maturity Onset Diabetes of the Young
- What is being tested?
- Liraglutide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 8 May 2025to Jan 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of MODY via genetic testing
- HgbA1c >6.5%
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Lily Deng, 513-636-0002, lily.deng@cchmc.org
Mansa Krishnamurthy
About this study
Official title: Investigating Enteroendocrine Dysfunction, Metabolism, and Response to Liraglutide in Patients With Maturity-onset Diabetes of the Young (MODY)
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.
Read the detailed description
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.
- Conditions
- Maturity Onset Diabetes of the Young
- Being tested
- Liraglutide
- Medical subject headings
- Diabetes Mellitus, Type 2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LiraglutideDrug
Also called Victoza, Saxenda
Liraglutide will be prescribed following FDA approved packet insert for type 2 diabetes in our test population.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Liraglutide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Blood sugar control - HbA1C
Hemoglobin A1c
Measured: 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
Measured: baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation
Secondary outcomes (6)
Metabolic - Blood Sugar
Glycemic patterns observed utilizing a continuous glucose monitor
Measured: Baseline through three months of study treatment
Enteroendocrine function - GI symptoms
ANMS Gastroparesis Cardinal Symptom Index Daily Diary (ANMS GCSI-DD)
Measured: baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation
Enteroendocrine function - insulin
Fasting and post meal blood serum insulin levels will be assessed
Measured: baseline and 3 months after study treatment initiation
Enteroendocrine function - c-peptide
Fasting and post meal blood serum c-peptide levels will be assessed
Measured: baseline and 3 months after study treatment initiation
Body fat percentage
% body fat as identified by DXA
Measured: baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation
Liver steatosis
MRI to determine liver health
Measured: baseline and 3 months after treatment initiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2025
- Study start8 May 2025
- Primary completionAug 2026 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Maturity Onset Diabetes of the YoungStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mansa
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Mansa, Children's Hospital Medical Center, Cincinnati
- Investigators
- Mansa Krishnamurthy, M.D., Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07029009
- Sponsor's protocol ID
- 2024-0426
- Status last verified
- Dec 2024
- First submitted
- 19 Aug 2024
- Last updated
- 17 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: individual participant data will not be made available to other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigating Enteroendocrine Dysfunction, Metabolism, and Response to Liraglutide in Patients With Maturity-onset Diabetes of the Young (MODY)
- ClinicalTrials.gov ID
- NCT07029009
- Acronym / sponsor ID
- 2024-0426
- Conditions
- Maturity Onset Diabetes of the Young
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- MansaUniversity, hospital or other
- Study start
- 8 May 2025 (actual)
- Primary completion
- Aug 2026 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 19 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| liraglutide treatment | Experimental | Drug: LiraglutideAfter 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 may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of MODY via genetic testing
- HgbA1c >6.5%
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LiraglutideAlso: Victoza, SaxendaLiraglutide will be prescribed following FDA approved packet insert for type 2 diabetes in our test population. | Drug | liraglutide treatment |
Primary outcome
Blood sugar control - HbA1C
Time frame: Baseline and 3 months after study treatment initiation
Hemoglobin A1c
BMI
Time frame: 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
6 secondary outcomes
Metabolic - Blood Sugar
Time frame: Baseline through three months of study treatment
Enteroendocrine function - GI symptoms
Time frame: baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation
Enteroendocrine function - insulin
Time frame: baseline and 3 months after study treatment initiation
Enteroendocrine function - c-peptide
Time frame: baseline and 3 months after study treatment initiation
Body fat percentage
Time frame: baseline, 1 month, 2 months, and final study visit 3 months after study treatment initiation
Liver steatosis
Time frame: baseline and 3 months after treatment initiation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2025.
- Individual participant dataSharing planned: No. individual participant data will not be made available to other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Mansa Krishnamurthy, M.D. principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Liraglutide in people with Maturity Onset Diabetes of the Young, run by Mansa at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mansa Krishnamurthy, M.D. (513-636-4744, mansa.krishnamurthy@cchmc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Blood sugar control - HbA1C (time frame: Baseline and 3 months after study treatment initiation). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Dec 2025.