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)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04732416

Enrollment

Registered

2021-02-01

Start date

2022-05-05

Completion date

2027-05-01

Last updated

2026-08-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital Hyperinsulinism

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.

Interventions

DRUGHM15136

Low dose of HM15136/ High dose of HM15136, SC injection, weekly

Study design

Allocation

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

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

Countries

Germany, Israel, South Korea, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Aug 27, 2026