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Trial record 6 of 22 for:    congenital hyperinsulinism

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

This study is currently recruiting participants.
Verified October 2017 by Xeris Pharmaceuticals
Sponsor:
ClinicalTrials.gov Identifier:
NCT02937558
First Posted: October 18, 2016
Last Update Posted: October 17, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by (Responsible Party):
Xeris Pharmaceuticals
  Purpose
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.

Condition Intervention Phase
Congenital Hyperinsulinism Drug: Glucagon Other: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (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

Resource links provided by NLM:


Further study details as provided by Xeris Pharmaceuticals:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 12
Study Start Date: October 2016
Estimated Study Completion Date: June 2018
Estimated Primary Completion Date: May 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: CSI-Glucagon
Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr.
Drug: Glucagon
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.
Other: Placebo
Isotonic saline

Detailed Description:

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:

  1. 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.
  2. 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.
  3. 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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 12 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 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.
  Contacts and Locations
Information from the National Library of Medicine

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


Contacts
Contact: Martin J Cummins 512-498-2675 mcummins@xerispharma.com

Locations
United States, California
UCSF School of Medicine, Division of Pediatric Endocrinology Recruiting
San Francisco, California, United States, 94143
Contact: Rebecca Wesch    415-476-5984    Rebecca.Wesch@ucsf.edu   
Principal Investigator: Christine Ferrara, MD         
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 Active, not recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
Xeris Pharmaceuticals
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  More Information

Responsible Party: Xeris Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02937558     History of Changes
Other Study ID Numbers: XSGO-CH01
1R44DK105691-01 ( U.S. NIH Grant/Contract )
First Submitted: October 14, 2016
First Posted: October 18, 2016
Last Update Posted: October 17, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Xeris Pharmaceuticals:
hypoglycemia

Additional relevant MeSH terms:
Hyperinsulinism
Congenital Hyperinsulinism
Hypoglycemia
Glucose Metabolism Disorders
Metabolic Diseases
Pancreatic Diseases
Digestive System Diseases
Infant, Newborn, Diseases
Glucagon
Glucagon-Like Peptide 1
Gastrointestinal Agents
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Incretins