CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism

NCT02937558 | Completed Phase 2 Results DMC

• 2 other identifiers: XSGO-CH011R44DK105691-01
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Congenital Hyperinsulinism

Keywords

hypoglycemia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Percent Change in GIR (Double-Blind)

  Baseline to the end of blinded treatment at 24 or 48 hours

• Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Open-Label)

  Baseline to the end of open-label treatment at 72 hours

• Percent Change in Glucose Infusion Rate (Open-Label)

  Baseline to end of treatment at 72 hours

Study Arms (3)

CSI-Glucagon (Double-Blind Phase - 2 days)

EXPERIMENTAL

Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr.

Drug: Glucagon

Placebo (Double-Blind Phase - 2 days)

PLACEBO COMPARATOR

Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.

Other: Placebo

CSI-Glucagon (Open-label Phase - Up to 28 days)

EXPERIMENTAL

Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr.

Drug: Glucagon

Interventions

Glucagon DRUG

Room-temperature-stable, non-aqueous injectable liquid formulation of synthetic glucagon peptide

Also known as: CSI-Glucagon (continuous subcutaneous glucagon infusion)

CSI-Glucagon (Double-Blind Phase - 2 days); CSI-Glucagon (Open-label Phase - Up to 28 days)

Placebo OTHER

Isotonic saline

Placebo (Double-Blind Phase - 2 days)

Eligibility Criteria

• Age: Up to 12 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Absolute necessity of intravenous glucose to prevent hypoglycemia:
• 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.
• May be on diazoxide and/or octreotide, but these drugs will be weaned off prior to randomization.
• May be on dextrose feeds.
• 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.
• Less than 12 months of age at screening.

You may not qualify if:

• History of allergy to glucagon or excipients in the CSI-Glucagon formulation.
• 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.
• Diazoxide naïve or within five days of starting diazoxide.
• Receiving steroids at doses larger than 20 mg/m2/day (hydrocortisone equivalent).
• Patients with sepsis.
• Receiving alpha or beta agonists for blood pressure support.
• Received an investigational or other study drug within 5 half-lives of drug.
• Body weight less than or equal to 2.3 kg/5.0 lbs.
• History of pancreatectomy and GIR \< 8 mg/(kg\*min) after weaning of all concomitant therapies.

Sponsors & Collaborators

• Xeris Pharmaceuticals: lead
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator

Study Sites (5)

UCLA Mattel Children's Hospital

Los Angeles, California, 90095, United States

Location

UCSF School of Medicine, Division of Pediatric Endocrinology

San Francisco, California, 94143, United States

Location

Washington University, St. Louis Children's Hospital

St Louis, Missouri, 63130, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Baylor College of Medicine, Texas Children's Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Congenital Hyperinsulinism; Hypoglycemia

Interventions

Glucagon

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperinsulinism; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Proglucagon; Pancreatic Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Khaled Junaidi
• Organization: Xeris Pharmaceuticals, Inc.

xerisclintrials@xerispharma.com

312-517-1461

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 14, 2016
• First Posted: October 18, 2016
• Study Start: October 1, 2016
• Primary Completion: September 1, 2018
• Study Completion: October 1, 2018
• Last Updated: December 10, 2019
• Results First Posted: December 10, 2019

Record last verified: 2019-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)