Ongoing Clinical Trials for Monogenic Diabetes
Currently, there are 2 ongoing clinical trials investigating new approaches to managing monogenic diabetes and related conditions. These studies are taking place across several European countries, including Denmark, Poland, Germany, Austria, Sweden, and Belgium. The trials focus on different aspects of diabetes prevention and treatment, with one examining a COVID-19 vaccine’s potential role in preventing Type 1 Diabetes in genetically susceptible children, and another testing the effectiveness of empagliflozin in controlling blood sugar levels in patients with a specific genetic form of diabetes known as HNF1A-MODY.
Clinical trial locations
- Austria
- Belgium
- Denmark
- Germany
- Poland
- Sweden
Study on the Effect of Raxtozinameran Vaccine in Reducing Type 1 Diabetes Risk in Children with High Genetic Susceptibility
This trial is investigating whether vaccinating young children against COVID-19 can help prevent the development of Type 1 Diabetes in those with a high genetic risk. The study focuses on a critical window early in life when the immune system may begin attacking insulin-producing cells in the pancreas.
Who can participate: Children between 3 and 4 months old at the time of enrollment are eligible if they have a high genetic risk (more than 10%) of developing islet autoantibodies by age 6. This risk is determined by specific genetic markers called HLA DR/DQ genotype, a polygenic risk score, and having a close family member with Type 1 Diabetes. Parents or guardians must provide written informed consent for their child to participate.
Who cannot participate: The study does not specify detailed exclusion criteria in the provided information, but standard clinical trial exclusions would likely apply.
What the trial involves: The trial uses the Comirnaty Omicron XBB.1.5 COVID-19 mRNA Vaccine, which is administered as an injection starting when children reach 6 months of age. The vaccine dose is 3 micrograms per administration. Some children will receive the actual vaccine, while others will receive a saline solution (placebo) for comparison. The study will follow participants over time to measure how long it takes for islet autoantibodies or Type 1 Diabetes to develop. Regular follow-up visits will monitor the children’s health and track any signs of the immune system beginning to attack the pancreas. The trial is scheduled to continue until October 2029, allowing researchers sufficient time to understand the vaccine’s impact on diabetes prevention.
Trial medication: The investigational treatment is the COVID-19 Vaccine, specifically designed to help the body develop immunity against the COVID-19 virus. In this trial context, researchers are exploring whether preventing COVID-19 infections in genetically susceptible children might reduce the risk of triggering the autoimmune response that leads to Type 1 Diabetes.
Study on the Effects of Empagliflozin on Blood Sugar Levels in Patients with HNF1A-MODY Diabetes
This clinical trial examines how well empagliflozin can control blood sugar levels in patients with a specific genetic form of diabetes called HNF1A-MODY (Maturity-onset diabetes of the young type 3). This form of diabetes is caused by a genetic mutation in the HNF1A gene that affects insulin production and typically appears in adolescence or early adulthood.
Who can participate: Adults aged 18 years or older with a confirmed heterozygous mutation in the HNF1A gene can join the study. Participants must have a Hemoglobin A1c level of at least 48 mmol/mol, which reflects their average blood sugar level over the past 2 to 3 months. They must also have been on stable blood sugar-lowering treatment for at least 60 days before the first study visit and be taking at least one glucose-lowering medication at a fixed dose. All participants must provide informed consent after understanding the study details.
Who cannot participate: Patients who do not have HNF1A-MODY cannot take part in this trial. Those outside the specified age range or who belong to vulnerable populations requiring special protection are also excluded from participation.
What the trial involves: This is a double-blind, crossover trial, meaning participants will receive both the active medication and a placebo at different times without knowing which one they are taking at any point. The trial medication is Jardiance 25 mg film-coated tablets containing empagliflozin, taken once daily by mouth. Empagliflozin works by helping the kidneys remove excess sugar from the body through urine, which helps maintain more balanced blood sugar levels. During the study, participants will wear a continuous glucose monitoring device that tracks blood sugar levels throughout the day and night. After completing the first treatment period, participants enter a washout period where no trial medication is taken, allowing the effects of the first treatment to clear before switching to the second treatment. The study will measure whether there is a significant difference in blood sugar control between taking empagliflozin and taking the placebo, while also monitoring factors such as body weight and episodes of low blood sugar.
Trial medication: Empagliflozin is classified as an SGLT2 inhibitor. It works by blocking a protein in the kidneys called SGLT2, which reduces how much glucose is reabsorbed back into the bloodstream, thereby lowering blood sugar levels. This medication is being tested specifically for its effectiveness in managing the genetic form of diabetes caused by HNF1A mutations.
Summary
These two ongoing clinical trials represent different approaches to managing diabetes and related conditions. The first trial takes an innovative preventive approach, investigating whether COVID-19 vaccination in infancy might reduce the risk of Type 1 Diabetes in genetically susceptible children. This multi-country study across Poland, Germany, Austria, Sweden, and Belgium reflects a long-term commitment to understanding diabetes prevention, with follow-up extending until 2029.
The second trial, conducted in Denmark, focuses on treatment options for a specific genetic form of diabetes. By testing empagliflozin in HNF1A-MODY patients, researchers aim to determine whether this medication class, already used in other forms of diabetes, can effectively manage blood sugar levels in this genetically distinct population.
Together, these trials highlight the diversity of research approaches in diabetes care, from early prevention strategies in high-risk infants to targeted treatment options for adults with specific genetic mutations. Both studies use rigorous scientific methods, including placebo controls and blinding procedures, to ensure reliable results that can guide future medical practice.
