HM15136 for the Treatment of Congenital Hyperinsulinism Research Study
Contact
Description
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.
Eligibility and criteria
What to expect
The study includes up to 13 visits(11 outpatient and 2 inpatient) over a period of up to 22 weeks as describedbelow.
·         Screening Visit:This visit helps us determine if you are eligible for the study. We will askyou questions about your medical history, take blood and urine samples, do aphysical exam (with vitals and alcohol breath test), and have you complete anECG.
·         Run-in Visits (if you meet studyrequirements): You will continue to receive your current HI medications; repeatsome of the procedures from the screening visit and be given a glucose meter,CGM, and eDiary to wear/complete throughout the rest of the study.
·         Baseline Visits: You will repeatprocedures from the previous visits as well as completing questionnaires.
·         Treatment Visits: You will receivestudy drug once a week at the inpatient and outpatient visits during the 8-weektreatment period during which previous study visit procedures will be repeated.
·         Follow-up Period: After completingstudy drug treatment, you will be asked to complete two follow-up visits. Ifyou changed your standard of care treatment or nutritional supplementationduring the Treatment Visits, you will return to your previous treatment, asinstructed by the study doctor. You will have previous tests and proceduresrepeated.
Related specialties
We need families like you
Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.