CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
|Official Title:||A Phase 2 Proof-of-Concept Study of CSI-Glucagon™ (Continuous Subcutaneous Glucagon Infusion) to Prevent Hypoglycemia With Lower Intravenous Glucose Infusion Rates in Children up to One Year of Age With Congenital Hyperinsulinism|
- Reduction in Glucose Infusion Rate [ Time Frame: Baseline to end of treatment at 24 or 48 hours ]Reduction from baseline in glucose infusion rate (GIR) will be determined for each subject at 24 and 48 hours from the start of blinded treatment. Subjects with GIR ≥ 20% at 24 hours, and ≥ 33% at 48 hours will be considered to have had a positive treatment response.
- Mean Reduction in GIR [ Time Frame: Baseline to the end of treatment at 24 or 48 hours ]The groups will be compared for average proportional GIR reduction from baseline.
- Targeted GIR reduction [ Time Frame: Baseline to the end of treatment at 24 or 48 hours ]The groups will be compared for the percentage of subjects that achieve GIR ≤ 8 mg/(kg*min).
- Targeted carbohydrate reduction [ Time Frame: Baseline to the end of treatment at 24 or 48 hours ]The groups will be compared for the percentage of subjects that achieve a daily caloric intake from carbohydrate, combining oral, tube and IV sources, ≤ 8 mg/(kg*min).
- Time in range [ Time Frame: Baseline to the end of treatment at 24 or 48 hours ]The groups will be compared for the proportional time in hypoglycemia, with blood glucose < 70 mg/dL, and in euglycemia, with blood glucose in the range of 70-180 mg/dL.
|Study Start Date:||October 2016|
|Estimated Study Completion Date:||December 2017|
|Estimated Primary Completion Date:||September 2017 (Final data collection date for primary outcome measure)|
Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr.
Room-temperature-stable, non-aqueous injectable liquid formulation of synthetic glucagon peptide
Other Name: CSI-Glucagon (continuous subcutaneous glucagon infusion)
Placebo Comparator: Placebo
Normal saline delivered as a 24-hour continuous subcutaneous infusion via a patch pump.
This is a Phase 2, multi-center, randomized, placebo-controlled, double-blind parallel group study with open-label follow-up designed to evaluate the efficacy of CSI-Glucagon™ for the prevention of hypoglycemia with lower IV glucose infusion rates when delivered subcutaneously to patients up to 1 year of age with congenital hyperinsulinism. CSI-Glucagon™ is expected to provide a better inpatient treatment option compared to the current standard of care.
The study will consist of three phases:
- Baseline Phase: First is a baseline stabilization phase of at least 24 hours, during which concomitant therapy with octreotide and diazoxide will be safely weaned and continuous enteric feed will be held constant to the degree possible, with the only factors varying being meal size and IV glucose infusion rate (GIR) adjusted by a set plasma glucose measurement driven algorithm.
- Blinded, Randomized Treatment Phase: Following the stabilization phase, subjects will be randomly assigned to blinded treatment with either glucagon or placebo, which will be delivered for up to 48 hours with an OmniPod® infusion pump with the controller set to a starting basal rate for glucagon of 5 μg/kg/hr and GIR adjustments used to maintain euglycemia. After 48 hours of blinded treatment, all subjects will transition to open-label active treatment. However, if GIR reduction from baseline is < 20% at 24 hours, subjects will be transitioned early to the open-label phase.
- Open-label Treatment Phase: The third study period will involve use of CSI-Glucagon™ to manage blood glucose with minimal GIR for up to 28 days of cumulative exposure.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02937558
|Contact: Martin J Cumminsemail@example.com|
|United States, Texas|
|Cook Children's Medical Center||Recruiting|
|Fort Worth, Texas, United States, 76104|
|Contact: Larry Rodriguez, BSN, RN, CRC 682-885-7208 Larry.Rodriguez@cookchildrens.org|
|Principal Investigator: Paul Thornton, MD|
|Baylor College of Medicine||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Jacqueline Castello, CMA 832-824-4126 Jxcastel@texaschildrens.org|
|Principal Investigator: George Jeha, MD|