Treatment for Congenital Hyperinsulinism in Children and Adults

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 testing a new medication called HM15136 to see if it can help children and adults with congenital hyperinsulinism who still have low blood sugar even with standard treatments.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 16 (estimated)
• Ages: 2 Years and up
• Sex: All
• Sponsor: Hanmi Pharmaceutical Company Limited (Industry-sponsored)
• Locations: 7 sites (Los Angeles, California and 6 other locations)
• Trial ID: NCT04732416 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety, tolerability, pharmacokinetics, and efficacy of HM15136 as an add-on therapy for patients aged 2 years and older with congenital hyperinsulinism (CHI) who experience persistent hypoglycemia despite standard treatment. Participants will receive weekly doses of HM15136 over an 8-week period to gather data on its effects. The study aims to determine the appropriate exposure-response relationship for this medication in a pediatric and adult population.

Who should consider this trial

Good fit: Ideal candidates include males and females aged 2 years and older with CHI and persistent hypoglycemia despite standard care.

Not a fit: Patients with type 1 or type 2 diabetes mellitus or those with other causes of hypoglycemia will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve blood sugar control in patients with congenital hyperinsulinism.

How similar studies have performed: While this approach is novel, similar studies targeting hypoglycemia management have shown promise in other contexts.

Eligibility criteria

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

Where this trial is running

Los Angeles, California and 6 other locations

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

Study contacts

• Study coordinator: JinHee Byeon

Conditions Congenital Hyperinsulinism