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

Study Overview
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Brief Summary
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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.

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

Conditions
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Congenital Hyperinsulinism

Intervention / Treatment
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* Drug: HM15136


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 2 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: HM15136 active<br>Cohort A / Cohort C | Drug: HM15136<br>* Low dose of HM15136/ High dose of HM15136, SC injection, weekly<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 |