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.

Congenital Hyperinsulinism is the most common cause of lowblood sugar (hypoglycemia) in infants and children.  Infants who are born with this problem havelow blood sugar, which can cause seizures or brain damage. 

We are doing this study to see if the patterns ofabnormalities in controlling insulin in children and adults withhyperinsulinism are related to any underlying genetic cause and to identifypossible new genetic causes.  This willbe done by standard clinical tests of insulin control: a glucose tolerancetest, a protein tolerance test, and a fasting test.

Our team is working on a study to better understand neurological problems, including seizures and developmental delays, that occur in children with hyperinsulinism, including HI/HA syndrome, and type 1 diabetes mellitus. 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 five hours. If you are interested in learning more, please contact us at HIResearch@email.chop.edu.

Our team is working on a study to see if children with HI/HA Syndrome tolerate Vitamin E supplementation. This is an experimental, non-FDA approved use of Vitamin E which will lead to further studies to evaluate if Vitamin E can be used as a treatment for HI/HA. The study involves taking Vitamin E once a day for two weeks at home, completing a tolerability questionnaires, and two, one-day visits to the CHOP outpatient Center for Human Phenomic Science (one visit before and one visit after taking the Vitamin E supplement for two weeks). During each study visit, fasting oral protein tolerance will be performed and blood samples will be drawn.