CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism
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|ClinicalTrials.gov Identifier: NCT02937558|
Recruitment Status : Completed
First Posted : October 18, 2016
Last Update Posted : November 9, 2018
|Condition or disease||Intervention/treatment||Phase|
|Congenital Hyperinsulinism||Drug: Glucagon Other: Placebo||Phase 2|
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 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|Study Start Date :||October 2016|
|Actual Primary Completion Date :||September 2018|
|Actual Study Completion Date :||October 2018|
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
Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02937558
|United States, California|
|UCLA Mattel Children's Hospital|
|Los Angeles, California, United States, 90095|
|UCSF School of Medicine, Division of Pediatric Endocrinology|
|San Francisco, California, United States, 94143|
|United States, Missouri|
|Washington University, St. Louis Children's Hospital|
|Saint Louis, Missouri, United States, 63130|
|United States, Texas|
|Cook Children's Medical Center|
|Fort Worth, Texas, United States, 76104|
|Baylor College of Medicine, Texas Children's Hospital|
|Houston, Texas, United States, 77030|