Treatment for Monogenic Diabetes

EnrollingByInvitation · Any Age · All Sexes · Boston, MA

Oral Hypoglycemic Therapy for Monogenic Variant Carriers of the Joslin Medalist Study

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About the trial for Monogenic Diabetes

Eligible Conditions
Monogenic Diabetes · Diabetes Mellitus · Type 1 Diabetes Mellitus

Treatment Groups

This trial involves a single treatment. Treatment is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 & 3 and have had some early promising results.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.


This trial is for patients born any sex of any age. There are 4 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
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Odds of Eligibility
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 3 months and 6 months
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 3 months and 6 months.
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Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Treatment will improve 1 primary outcome and 8 secondary outcomes in patients with Monogenic Diabetes. Measurement will happen over the course of 6 months.

Area under the plasma concentration versus time curve (AUC) of C-peptide
Change in AUC of C-peptide between the two study groups, as measured by the hyperglycemic/arginine clamp, at the end of the study
Change in C-peptide (ng/mL) between the two study groups
High density lipoprotein (HDL)-cholesterol
Change in HDL-cholesterol (mg/dL) between the two study groups
Daily insulin dose
Change in daily insulin dose (units/kg body weight) between the two study groups
Glycated hemoglobin (HbA1c)
Change in HbA1c (%) between the two study groups
Low density lipoprotein (LDL)-cholesterol
Change in LDL-cholesterol (mg/dL) between the two study groups
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people get diabetes mellitus a year in the United States?

Almost 30 million Americans have been diagnosed with diabetes mellitus. Nearly half of those diagnosed with diabetes die from complications of the disease. The disease is associated with significant financial and moral consequences. Therefore, the disease is a major public health burden in the United States.

Anonymous Patient Answer

What causes diabetes mellitus?

Diabetes mellitus is mostly due to either genetic predisposition or environmental factors such as diet or lifestyle. It is estimated that two thirds of the cases of diabetes can be prevented through lifestyle modification. Diabetes, however, can occur at all ages and presents a big challenge in global health because it affects the quality of human life.

Anonymous Patient Answer

What is diabetes mellitus?

In contrast to other well-known pathologies like leukemia, there is no indication for the use of a general cancer guideline for diabetes mellitus. It may be used in case of pulmonary, gastrointestinal and vaginal cancer. Further, diabetes is not an absolute indication for surgical treatment.

Anonymous Patient Answer

What are common treatments for diabetes mellitus?

Current treatment options for diabetes include oral agents, insulin, and metformin. Most patients are treated with the oral agents or their combinations. Insulin therapy is rarely used alone, but if combined with metformin it is effective. Other agents including thiazides and sulfonylureas are used for blood sugar control. There are no other widely accepted treatments for diabetes.

Anonymous Patient Answer

What are the signs of diabetes mellitus?

Many people will have a few sign or symptoms of diabetes but not necessarily be aware of them. Some symptoms may require careful investigation for an early diagnosis. \n\nAn important group to identify and monitor in order to prevent cardiovascular disease are those with diabetes and with an additional risk factor. In addition to blood glucose levels, triglyceride levels and blood pressure should be recorded by a doctor.

Anonymous Patient Answer

Can diabetes mellitus be cured?

Diabetes may be curable not in a person with hyperglycemia but in a person with normal glucose levels; in a person with hypoglycemia; and in a person with hyperglycemia and hypoglycemia. Diabetes mellitus may be cured. This is the subject of further exploration.

Anonymous Patient Answer

What is the average age someone gets diabetes mellitus?

Type II diabetes first appear at a relatively young age, with a peak at 55 years of age for men and 45 years of age for women. The absolute number of people who develop diabetes is declining in all age groups. [ Diabetes_Mellitus ( Diabetes_Mellitus) Diabetes mellitus is a chronic disease that can be successfully managed using a combination of diet, exercise, and regular monitoring of blood glucose and blood pressure levels. Diabetes can be prevented by decreasing risk factors such as obesity, high blood pressure and sedentary lifestyles. There are many diet and physical activity recommendations for improving health and preventing diabetes.

Anonymous Patient Answer

What are the common side effects of treatment?

Findings from a recent study indicated that the most common side effects of medication were nausea, dry mouth, and diarrhea. Diarrhea and dizziness were the most commonly reported (in more than 80%) side effects of antidiabetic agents.

Anonymous Patient Answer

How serious can diabetes mellitus be?

Serious complications from DM relate mainly to the duration of DM, the age of onset of DM, the presence of microalbuminuria, and the presence of retinopathy. However, with modern treatment, complications may cease from time to time and with good adherence to treatment.

Anonymous Patient Answer

Is treatment safe for people?

A small proportion of people who undergo treatment with DPP-4 inhibitors or GLP-1 agonists develop serious adverse events during therapy. No clear association was found between the occurrence of adverse events and age or duration of treatment and all patients appear to have benefited from long-term treatment. However, further research is required to verify the long-term safety of this new class of therapeutics, particularly in patients who are aged >75 years or who are taking concomitant therapies.

Anonymous Patient Answer

How does treatment work?

The type 2 diabetic population enrolled in the study received a higher level of care than the patients in general. In a recent study, findings of this study, therefore, are more specific to those with type 2 diabetes than they are to patients with type 2 diabetes in general. Nevertheless, although it should be acknowledged that type 2 diabetes is a broad category, the overall findings from this study are meaningful to patients with type 2 diabetes.

Anonymous Patient Answer

Does diabetes mellitus run in families?

Hyperglycemia is an important risk factor for the development of CAD. Therefore, hyperglycemia may be one of the common causes of CAD in families with a high prevalence of hyperglycemia/diabetes mellitus, such as the Pakistani population.

Anonymous Patient Answer
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