Congenital Hyperinsulinism Clinical Trial
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
Verified date | November 2019 |
Source | Xeris Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This is a Phase 2, multi-center, randomized, placebo-controlled, double-blind trial with open-label follow-up designed to assess the efficacy of Xeris Glucagon delivered as a continuous subcutaneous infusion to prevent hypoglycemia with lower intravenous glucose infusion rates in children < 1 year of age with congenital hyperinsulinism.
Status | Completed |
Enrollment | 5 |
Est. completion date | October 2018 |
Est. primary completion date | September 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 12 Months |
Eligibility |
Inclusion Criteria: 1. Diagnosed with hyperinsulinism: a. Biochemical; detectable insulin (i.e., =1 µIU/L) at time of hypoglycemia (i.e, blood glucose <50 mg/dl), and/or suppressed free fatty acids (FFA), and/or suppressed beta-hydroxybutyrate (BOHB) and/or glycemic response to glucagon at time of hypoglycemia. 2. Absolute necessity of intravenous glucose to prevent hypoglycemia: 1. Having failed diazoxide therapy as defined by inadequacy of 5 days maximum dose of diazoxide to eliminate the need for IV glucose, not necessarily that diazoxide has no effect. 2. May be on diazoxide and/or octreotide, but these drugs will be weaned off prior to randomization. 3. May be on dextrose feeds. 3. Patient may be a participant in other study protocols such as observational studies, as long as no investigational intervention has taken place within 24 hrs. prior to screening. 4. Less than 12 months of age at screening. Exclusion Criteria: 1. History of allergy to glucagon or excipients in the CSI-Glucagon formulation. 2. Currently receiving, or less than 12 hours removed from IV glucagon treatment that resulted in a best achievable GIR > 8 mg/(kg*min), prior to the start of study drug. 3. Diazoxide naïve or within five days of starting diazoxide. 4. Receiving steroids at doses larger than 20 mg/m2/day (hydrocortisone equivalent). 5. Patients with sepsis. 6. Receiving alpha or beta agonists for blood pressure support. 7. Received an investigational or other study drug within 5 half-lives of drug. 8. Body weight less than or equal to 2.3 kg/5.0 lbs. 9. History of pancreatectomy and GIR < 8 mg/(kg*min) after weaning of all concomitant therapies. |
Country | Name | City | State |
---|---|---|---|
United States | Cook Children's Medical Center | Fort Worth | Texas |
United States | Baylor College of Medicine, Texas Children's Hospital | Houston | Texas |
United States | UCLA Mattel Children's Hospital | Los Angeles | California |
United States | Washington University, St. Louis Children's Hospital | Saint Louis | Missouri |
United States | UCSF School of Medicine, Division of Pediatric Endocrinology | San Francisco | California |
Lead Sponsor | Collaborator |
---|---|
Xeris Pharmaceuticals | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind) | Change 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 a decrease in GIR = 20% at 24 hours, and = 33% at 48 hours will be considered to have had a clinically meaningful treatment response. | Baseline to end of blinded treatment at 24 or 48 hours | |
Secondary | Percent Change in GIR (Double-Blind) | The groups will be compared for mean percent change in GIR from baseline to the end of the double-blind study phase. | Baseline to the end of blinded treatment at 24 or 48 hours | |
Secondary | Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Open-Label) | Change from baseline in glucose infusion rate (GIR) will be determined for each subject at the end of open-label treatment. Subjects with a decrease in GIR = 33% will be considered to have had a clinically meaningful treatment response. | Baseline to the end of open-label treatment at 72 hours | |
Secondary | Percent Change in Glucose Infusion Rate (Open-Label) | The groups will be compared for mean percent change in GIR from baseline to the end of the open-label study phase. | Baseline to end of treatment at 72 hours |
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