CSI Glucagon for Treatment of Congenital Hyperinsulinism

DESCRIPTION provided by applicant The objective of this program is to further the development of a novel stable non aqueous glucagon formulation CSI continuous subcutaneous infused Glucagon tm in combination with a Insulet OmniPod r infusion pump for prevention of hypoglycemia in patients with congenital hyperinsulinism HI The addition of glucagon will meet a significant unmet need in the management of severe persistent hypoglycemia and will allow greater control of blood glucose in this fragile pediatric patient population This program intends to demonstrate the safety of the novel glucagon in a month preclinical study and thereafter establish safety and efficacy in pediatric patients with HI The major clinical metric for this study will be the ability of CSI Glucagon tm to maintain normoglycemia delivered as a basal subcutaneous infusion The initial effort of this project will manufacture clinical supplies and put material on an ICH stability testing program Thereafter we will assess of the safety of CSI Glucagon tm in a month animal study Then we will file an Investigational New Drug IND application with the FDA Finally the safety and efficacy of our soluble glucagon will be assessed in a Phase clinical trial in pediatric patients with HI The tral will entail a day study in patients once they are stabilized with glucose and thereafter an outpatient phase wherein patients will continue therapy with CSI Glucagon tm for a period of up to six months with monthly examinations If after six months it is determined that blood glucose is being successfully managed i e they remain in a normal glycemic range on a daily basis the parents will have the option to have their child continue treatment with CSI Glucagon tm until such time that the child metabolically andquot grows intoandquot their insulin production At this point they ae typically consuming sufficient complex carbohydrates such that they can eventually discontinue treatment This can be as short as year but can extend up to years of age or longer PUBLIC HEALTH RELEVANCE The objective of this program is to further the development of a novel stable non aqueous glucagon formulation as continuous subcutaneous infusion CSI Glucagon tm delivered by an Insulet OmniPod r patch pump for prevention of severe persistent hypoglycemia in pediatric patients with congenital hyperinsulinism HI The addition of CSI Glucagon tm delivered as a basal infusion with a compatible and caregiver friendly OmniPod r infusion pump will provide a valuable and needed tool for medical management of blood glucose in HI patients CSI Glucagon tm delivered in this manner will lower the risk of severe repeated hypoglycemic events and the attendant brain damage in a very fragile patient population in both an inpatient setting and particularly in the outpatient setting for parents and in home nurses