Ongoing Clinical Trials for Hyperinsulinism
This article provides information about 4 ongoing clinical trials investigating new treatments for hyperinsulinism, a rare condition where the body produces too much insulin, leading to dangerously low blood sugar levels. These studies are testing different medications to help manage this condition in both children and adults across several European countries.
Clinical trial locations
- Bulgaria
- Denmark
- France
- Germany
- Greece
- Netherlands
- Spain
Study on the Safety and Effects of Efpegerglucagon in Children Aged 2 and Older with Congenital Hyperinsulinism
This study is investigating HM15136, a new treatment for children with congenital hyperinsulinism. The condition occurs when the body produces too much insulin, causing dangerously low blood sugar levels.
Who can participate:
- Children aged 2 years and older with confirmed congenital hyperinsulinism
- Patients experiencing at least 3 episodes of low blood sugar per week despite current treatments
- Children aged 2-11 should have been on stable treatment with diazoxide or octreotide for at least 1 month
- Participants aged 12 and older should have been on stable treatment with diazoxide or somatostatin analogs for at least 3 months
- HbA1c level (a measure of blood sugar control) must be less than 7%
- Female participants who can become pregnant must use effective birth control, and male participants must use condoms during the study and for 60 days after
Who cannot participate:
- Patients with other serious health conditions that could interfere with the study
- Those currently participating in another clinical trial
- Patients who have had recent surgery or are planning surgery during the study
- Pregnant or breastfeeding women
- Patients with a history of allergic reactions to similar medications
- Those with certain heart conditions, uncontrolled high blood pressure, or a history of mental health disorders
What the study involves: Participants will receive HM15136 injections under the skin for 8 weeks. Researchers will monitor safety and how the body processes the medication. They will also track how the treatment affects the frequency of low blood sugar events. If no significant side effects occur, participants may continue with an optional extension treatment period.
Study on Ersodetug for Patients with Uncontrolled Low Blood Sugar Due to Tumor-Related High Insulin Levels
This trial is evaluating ersodetug for adults with tumor-associated hyperinsulinism, where tumors cause excessive insulin production. The study compares ersodetug to a placebo when added to standard treatments.
Who can participate:
- Adults aged 18 and older
- Diagnosed with tumor-associated hyperinsulinism, confirmed by laboratory tests
- Condition considered difficult to control with standard treatments
- Experiencing at least 3 episodes per week of low blood sugar (below 54 mg/dL) or episodes requiring assistance due to confusion or physical symptoms
- Life expectancy of at least 3 months
- ECOG performance status of 2 or better (able to care for yourself with limited ability to work)
- Women who can become pregnant must use effective birth control during the study and for 3 months after
- Men with female partners who can become pregnant must use effective birth control during the study and for 3 months after
Who cannot participate:
- Patients with low blood sugar not caused by tumor-associated hyperinsulinism
- Those whose condition is adequately controlled with current treatment
- Patients with other medical conditions that might interfere with study results or safety
- Those taking medications that could interact with ersodetug
- Patients with known allergies to ersodetug or its components
- Pregnant or breastfeeding women, or those planning pregnancy during the study
What the study involves: Participants will be randomly assigned to receive either ersodetug or placebo through intravenous infusion as an addition to their standard care. The main treatment period lasts approximately 8 weeks. Blood glucose levels will be monitored continuously, and researchers will track the frequency and severity of low blood sugar events. For hospitalized patients requiring IV glucose, the study will monitor changes in glucose infusion rates. An optional extension phase may be available after completing the main treatment period.
Long-term Safety Study of Dasiglucagon for Children with Congenital Hyperinsulinism
This study examines the long-term safety and effectiveness of dasiglucagon for children with congenital hyperinsulinism. Dasiglucagon is delivered through a special insulin pump to help manage blood sugar levels.
Who can participate:
- Children who have completed treatment in either Trial ZP4207-17103 or ZP4207-17109
- Expected to continue benefiting from dasiglucagon treatment based on previous trial experience
- Female participants who can become pregnant must have a negative pregnancy test at the start
- Sexually active female participants and their partners must use acceptable contraception or abstain from sexual activity during the trial and for 30 days after the last dose
- Able and willing to follow trial procedures
- One or both parents or guardian must provide signed consent
What the study involves: Dasiglucagon is given as a subcutaneous infusion (under the skin) using the Accu-Chek Spirit Combo insulin pump. The trial focuses on monitoring long-term safety, tracking any adverse events, and measuring outcomes such as the amount of carbohydrates needed to treat low blood sugar, time until removal of feeding tubes, and timing of any necessary pancreatic surgeries. Continuous glucose monitoring tracks the percentage of time blood sugar levels remain below 70 mg/dL and the rate of hypoglycemia episodes. The trial is expected to continue until November 2026.
Study on RZ358 for Treating Congenital Hyperinsulinism in Patients
This trial evaluates RZ358, a medication designed to block the insulin receptor, for patients with congenital hyperinsulinism. The study compares RZ358 to a placebo over 24 weeks.
Who can participate:
- Aged between 3 months (corrected for gestational age if under 9 months) and 45 years
- Confirmed clinical diagnosis of congenital hyperinsulinism, with or without a known genetic cause
- Have not achieved adequate blood sugar control despite trying standard treatments such as diazoxide and somatostatin analogs
- Experiencing at least 3 episodes of low blood sugar (less than 70 mg/dL or 3.9 mmol/L) per week
- Average daily time with low blood sugar of at least 8% during screening
- Liver ultrasound showing no significant issues
- Female participants who can become pregnant must have a negative pregnancy test and agree to use highly effective birth control during the study and for at least 3 months after
- Sexually active male participants must use contraceptive measures during the study and for at least 3 months after
Who cannot participate:
- Patients with other serious health conditions that might interfere with the study
- Pregnant or breastfeeding women
- Those who have participated in another clinical trial recently
- Patients with allergies to the study medication or its ingredients
- Those unable to follow study procedures or instructions
- Patients with a history of drug or alcohol abuse
- Those with certain heart conditions, severe kidney or liver problems
- Patients taking medications that might interfere with the study drug
What the study involves: This is a double-blind study, meaning neither participants nor researchers know who receives RZ358 or placebo. The medication is given intravenously (directly into a vein) for 24 weeks. Throughout the study, blood sugar levels are regularly monitored using self-monitoring devices and continuous glucose monitoring. Regular safety assessments include laboratory tests, physical exams, and possibly additional ultrasounds. After 24 weeks, a final assessment evaluates the treatment’s effects on blood sugar levels and overall health.
Summary
These four clinical trials represent important research efforts to develop new treatments for hyperinsulinism across different age groups and causes. Three trials focus specifically on congenital hyperinsulinism in children and young adults, while one addresses tumor-associated hyperinsulinism in adults.
Geographically, the trials are concentrated primarily in Germany, which hosts three of the four studies, followed by France with two trials. Other participating countries include the Netherlands, Denmark, Bulgaria, Greece, and Spain. This multi-country approach helps ensure diverse patient populations and broader applicability of results.
The investigational medications being tested represent different therapeutic approaches. HM15136 and dasiglucagon work to counteract the effects of excess insulin by raising blood sugar levels, while RZ358 and ersodetug block insulin receptors to prevent low blood sugar episodes. Each medication is administered differently—some through injections under the skin, others through intravenous infusion—and treatment periods range from 8 weeks to 24 weeks, with some offering optional extension phases.
All trials emphasize safety monitoring as a primary concern, particularly for pediatric participants. Participants must meet specific criteria regarding blood sugar control, treatment history, and overall health status. The studies aim to determine whether these new treatments can reduce the frequency and severity of dangerous low blood sugar episodes while maintaining acceptable safety profiles.
