HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)

A Phase 2, Multiple Ascending Dose, Open-label, Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)

This study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of HM15136 when used as add-on therapy in subjects with CHI with persistent hypoglycemia while on standard of care treatment (SoC). HM15136 will be administered once weekly in multiple doses to subjects in multiple age including pediatric to find appropriate exposure-response data.

Study Overview

• Status: Active, not recruiting
• Conditions: Congenital Hyperinsulinism
• Intervention / Treatment: Drug: HM15136

• Study Type: Interventional
• Enrollment (Estimated): 16
• Phase: Phase 2

Contacts and Locations

Study Locations

• Germany
  • Magdeburg, Germany
    • Otto-von-Guericke-Universitaet Magdeburg
• Israel
  • Jerusalem, Israel
    • Hadassah Medical Center (HMC)
• South Korea
  • Pusan, South Korea
    • Pusan National University Yangsan Hospital
  • Seoul, South Korea
    • Asan Medical Center
• United Kingdom
  • London, United Kingdom
    • Great Ormond Street Hospital (GOSH) for Children NHS Foundation Trust
  • Manchester, United Kingdom
    • Central Manchester University Hospitals NHS Foundation Trust - Royal Manchester Children's Hospital - Centre for Paediatrics and Child Health
• United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • The Children's Hospital of Philadelphia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male and female subjects aged ≥2 years with CHI with persistent hypoglycemia despite current SoC treatment according to the investigator's evaluation or documentation
• Stable therapy with SoC medications with or without nutritional supplementation
• Previously undergone near-total pancreatectomy or being treated with a nonsurgical approach, having been evaluated as not eligible for pancreatic surgery
• HbA1c <7%

Exclusion Criteria:

• Subjects with type 1 or type 2 diabetes mellitus
• Other reasons for hypoglycemia, including but not limited to drug-induced hyperinsulinemic hypoglycemia, etc
• Treatment of CHI with continuous intravenous glucose or glucagon infusion within 3 months prior to screening
• Subjects with current use of any drugs that are known to interfere with the study drug, glucose metabolism, or study procedures (eg, use of systemic glucocorticoids [excluding topical, intra-articular or ophthalmic application, nasal spray, or inhaled forms] or insulin)
• Have conditions that could affect glucose levels such as pheochromocytoma, insulinoma, and glucagonoma

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: HM15136 active; Cohort 1 / Cohort 2	Drug: HM15136; Low dose of HM15136/ High dose of HM15136, SC injection, weekly

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of incidence of AEs, TEAE, SAE as assessed by CTCAE v5.0	after multiple subcutaneous (SC) doses of 8 weeks
Number of incidence of clinical laboratory abnormalities	after multiple subcutaneous (SC) doses of 8 weeks
Maximum Serum Concentration [Cmax]	after multiple subcutaneous (SC) doses of 8 weeks
Time to reach Cmax	after multiple subcutaneous (SC) doses of 8 weeks

Collaborators and Investigators

• Sponsor: Hanmi Pharmaceutical Company Limited

Study record dates

Study Major Dates

• Study Start (Actual): May 5, 2022
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): May 1, 2027

Study Registration Dates

• First Submitted: January 20, 2021
• First Submitted That Met QC Criteria: January 26, 2021
• First Posted (Actual): February 1, 2021

Study Record Updates

• Last Update Posted (Actual): May 11, 2026
• Last Update Submitted That Met QC Criteria: May 7, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Digestive System Diseases; Infant, Newborn, Diseases; Glucose Metabolism Disorders; Pancreatic Diseases; Hyperinsulinism; Hypoglycemia; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Congenital Hyperinsulinism; HM15136
• Other Study ID Numbers: HM-GCG-201

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No