Dasiglucagon for Congenital Hyperinsulinism

This trial tests the long-term safety and effectiveness of dasiglucagon, administered as an under-the-skin infusion, for children with Congenital Hyperinsulinism (CHI). CHI causes the body to produce excessive insulin, leading to low blood sugar levels. The trial includes children who have completed specific previous studies with dasiglucagon and are expected to benefit from it without serious side effects. As a Phase 3 trial, this study is the final step before FDA approval, allowing participants to contribute to the potential availability of a new treatment option.

The trial information does not specify if you need to stop your current medications. However, if you require medication that was prohibited in the lead-in trial, you may not be eligible to participate.

Research has shown that dasiglucagon is generally safe for children with congenital hyperinsulinism (CHI). Studies indicate its safety profile is similar to glucagon, a related medication, meaning the side effects align with expectations for this drug type, with no major surprises.

An independent committee regularly reviews the safety data to quickly identify any potential issues. So far, results indicate that dasiglucagon is safe for children with CHI.

Unlike the standard of care for congenital hyperinsulinism, which often involves medications like diazoxide or surgical interventions, dasiglucagon offers a novel approach by acting as a glucagon analog. This means it can quickly raise blood sugar levels by stimulating the liver to release glucose, which is particularly useful in managing hypoglycemia. Researchers are excited about dasiglucagon because it is administered via a subcutaneous infusion, allowing for continuous and more stable glucose management, potentially reducing the frequency and severity of hypoglycemic episodes. This delivery method, combined with its fast-acting properties, positions dasiglucagon as a promising treatment option for those affected by this challenging condition.

Research shows that dasiglucagon, which participants in this trial will receive, can help children with congenital hyperinsulinism (CHI). Studies have found that it greatly reduces the need for IV glucose to stabilize blood sugar levels. One study demonstrated a 43% reduction in low blood sugar episodes compared to standard care alone. This indicates that dasiglucagon helps maintain healthier blood sugar levels, reducing the risk of dangerously low blood sugar. These findings suggest dasiglucagon could be a promising treatment for managing CHI.¹³⁶⁷⁸