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

Phase 2

Recruiting

• Conditions: Congenital Hyperinsulinism
• Interventions: Drug: HM15136

• Registration Number: NCT04732416
• Lead Sponsor: Hanmi Pharmaceutical Company Limited

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-01-20
• Study First Submitted QC: 2021-01-26
• Study First Posted: 2021-02-01
• Study Start: 2022-05-05
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-20
• Last Update Posted: 2025-02-21
• Primary Completion: 2025-12-31
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 16

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
HM15136 active	HM15136	Cohort 1 / Cohort 2

Primary Outcome Measures

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

Trial Locations

Locations (7)

University of California Los Angeles; 🇺🇸 Los Angeles, California, United States

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Otto-von-Guericke-Universitaet Magdeburg; 🇩🇪 Magdeburg, Germany

Hadassah Medical Center (HMC); 🇮🇱 Jerusalem, Israel

Asan Medical Center; 🇰🇷 Seoul, Korea, Republic of

Great Ormond Street Hospital (GOSH) for Children NHS Foundation Trust; 🇬🇧 London, United Kingdom

Central Manchester University Hospitals NHS Foundation Trust - Royal Manchester Children's Hospital - Centre for Paediatrics and Child Health; 🇬🇧 Manchester, United Kingdom