Clinical trial NCT04732416: HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Congenital Hyperinsulinism
Lead sponsor: Hanmi Pharmaceutical Company Limited
Sponsor class: INDUSTRY
First posted: 2021-02-01
Enrollment: 16
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Number of incidence of AEs, TEAE, SAE as assessed by CTCAE v5.0
timeFrame: after multiple subcutaneous (SC) doses of 8 weeks
measure: Number of incidence of clinical laboratory abnormalities
timeFrame: after multiple subcutaneous (SC) doses of 8 weeks
measure: Maximum Serum Concentration [Cmax]
timeFrame: after multiple subcutaneous (SC) doses of 8 weeks
measure: Time to reach Cmax
timeFrame: after multiple subcutaneous (SC) doses of 8 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California Los Angeles
state: California
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Magdeburg
country: Germany
facility: Otto-von-Guericke-Universitaet Magdeburg
city: Jerusalem
country: Israel
facility: Hadassah Medical Center (HMC)
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: London
country: United Kingdom
facility: Great Ormond Street Hospital (GOSH) for Children NHS Foundation Trust
city: Manchester
country: United Kingdom
facility: Central Manchester University Hospitals NHS Foundation Trust - Royal Manchester Children's Hospital - Centre for Paediatrics and Child Health
Study officials:
References: