Investigational Therapy for the Potential Treatment of Congenital Hyperinsulinism

Jairo Noreña, MD, endocrinology fellow at Stanford University, discusses an investigational therapy for the potential treatment of congenital hyperinsulinism.
Congenital hyperinsulinism is a rare pediatric genetic disorder characterized by excessive production of insulin by the pancreas. If left untreated, elevated insulin levels can cause extreme hypoglycemic (low blood sugar) events, increasing the risk of neurologic and developmental complications, including persistent feeding problems, learning disabilities, recurrent seizures, brain damage or even death.
Current management involves reducing insulin production, increasing resistance in the tissue to insulin, and reducing the tumor via surgery or other therapies.
RZ358 is a monoclonal antibody that binds to insulin receptors, counteracting its excess activation. It is currently being investigated for the treatment of congenital hyperinsulinism. The sunRIZE study is a phase 3, randomized, double-blind, placebo-controlled, parallel-arm study investigating the safety and efficacy of the treatment.
The development of this novel treatment may be an alternative treatment option for patients with hyperinsulinism caused by neuroendocrine tumors that may be unresponsive to traditional management.
Chapters:
Congenital Hyperinsulinism Overview 00:00
Current Management 1:14
RZ358 Clinical Trials 2:27