Congenital Hyperinsulinism Center Clinical Studies

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Research Study for Individuals with Hyperinsulinism/Hyperammonemia

Our team is working on a study to better understand neurological problems, including seizures and developmental delays, that occur in many individuals with HI/HA syndrome. The study involves a one day visit to The Children’s Hospital of Philadelphia and The University of Pennsylvania for a specialized magnetic resonance imaging scan (MRI) of the brain, an electroencephalogram (EEG), blood draw, and completion of questionnaires to evaluate development and behavior. The entire study visit is expected to take about four hours. If you are interested in learning more, please contact us at HIResearch@email.chop.edu.

Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism (for children under 1 year of age)

This study is arandomized, double-blind, placebo-controlled study that will evaluate thesafety and efficacy of dasiglucagon in children between the ages of 7 days and12 months who have congenital hyperinsulinism. Dasiglucagon will be given as asubcutaneous infusion while patients are admitted to the hospital. Othertreatments for hyperinsulinism may be changed as allowed while maintaining safeblood sugar levels. For the second part of the study, patients may be able togo home on the medication.

Phenotyping Congenital Hyperinsulinism

The Congenital Hyperinsulinism Center at the Children’s Hospital of Philadelphia is working on a research study to better understand how people with hyperinsulinism may have different blood sugar responses to certain tests (like fasting or drinking a high-protein shake) when compared to people without hyperinsulinism.

This study will involve a screening visit where we will ask you about your medical history and any known episodes of low blood sugar. Depending on your responses during the interview, you will be asked to complete up to five tests at our outpatient research center. These tests include fasting, drinking a high-protein shake, drinking a high-sugar drink, eating a regular meal, and exercising on a stationary bike.

We will collect blood samples from an IV throughout the tests to measure certain blood levels like glucose and insulin. You will be compensated for some or all of your travel costs and you will receive payment for your time.  If you are interested in learning more, please contact us at HIResearch@chop.edu.

HM15136 for the Treatment of Congenital Hyperinsulinism Research Study

Thepurpose of this study is to look at the safety and tolerability (how well youwill react) of the study drug (HM15136) and to determine if it is effective forthe treatment of Congenital Hyperinsulinism (HI). HM15136 is an experimentaldrug which is not approved by the FDA for the treatment of HI. HM15136 is stable and hasa long effect in the body, only requiring it to be given (by injection) once aweek.  HM15136 is designed to act likeglucagon, a hormone produced by the pancreas that helps the body maintainnormal blood sugar levels by increasing the glucose produced in the liver andbreaking down glycogen (a form of stored glucose) into the usable glucose form.

Participationlasts for up to 22 weeks and will involve up to 13 study visits. Studyprocedures include taking the study drug for 8 weeks, havingelectrocardiograms, wearing a continuous glucose monitor (CGM) and using ahandheld glucose meter, having blood and urine tests, and completing anelectronic diary.

Themain risks of this study include: nausea, vomiting, diarrhea, abdominal pain,injection site reactions (swelling, itching, bleeding, or pain), loss ofappetite, weight loss, high blood sugar. You may benefit if the study drugproves to be more effective than your current treatment plan in treating lowblood sugar.