Clinical Trial of Efepoetin Alfa in Healthy Subjects

An Open-label, Parallel-group, Single-center, Phase I Study to Compare the Pharmacokinetic/Pharmacodynamic Characteristics, Safety, and Tolerability of a Single Intravenous Administration of Efepoetin Alfa in Healthy Caucasian and Asian Volunteers

An open-label, parallel-group, single-center, Phase I study to compare the pharmacokinetic/pharmacodynamic characteristics, safety, and tolerability of a single intravenous administration of Efepoetin Alfa in healthy subjects

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: Efepoetin Alfa

Detailed Description

This study is to objectively evaluate pharmacokinetics as well as pharmacodynamic responses after a single intravenous administration of the Efepoetin alfa, GX-E4, in healthy Caucasian and Asian volunteers.

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Phase 1

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 04763
    • Hanyang University Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: Yes

Description

Key Inclusion Criteria:

1. Adult males and females between the ages of 19-45
2. Asian or Caucasian
3. Body weight >50 kg and <90 kg, BMI 18 ~30 (BMI(kg/m2) = Weight(kg) / {Height(m)}2)
4. Normal hemoglobin range.
5. Normal Serum ferritin and transferrin saturation range.
6. Normal serum folate range
7. Normal vitamin B12 range
8. White blood cell >=3.0 X 10^3 /mm3
9. Platelet >= 150 X 10^3/mm^3 and <450 X 10^3/mm^3
10. Nonsmoker or smoker who smokes below 10 cigarettes a day.

Key Exclusion Criteria:

1. An allergy history, including drug allergies(example: aspirin, antibiotics, etc.) or clinically significant allergy.
2. Liver(including viral hepatitis), renal, respiratory, endocrine, neurological, immunological, blood, psychological, or circulatory system abnormalities, or a history of cancer.
3. Subject who had received EPO, darbepoetin, other EPO supply protein, or immunoglobulin administration, or had received intravenous iron administration.
4. Hypersensitivity to EPO and/or to the excipients of the IMP, or known hypersensitivity to supplementary iron products.
5. Hemoglobinopathy (homozygous sickle cell disease, all types of thalassemia)
6. Systolic blood pressure below 90mmHg or above 140mmHg, or diastolic blood pressure below 50mmHg or above 90mmHg after taking a rest over 3 minutes; pulse rate over 100bpm
7. C-reactive protein level >4mg/dL at 2 weeks prior to the IMP administration.
8. A drug abuse history, or positive in a urine drug-screening test(cocaine, amphetamines, barbiturates, opiates, benzodiazepine, and cannabinoids)
9. Signs of fever, with a temperature of over 38°C, within 1 week before particiation
10. History of epileptic seizure within 6 months before participation
11. Positive to HIV antibody, HBsAg, and HCV antibody test.
12. Consumes over 21 units of alcohol per week, or a person who cannot stay sober for the duration of the entire trial period.
13. Blood donation or a bleeding episode of more than 400mL within 8 weeks prior to study participation
14. The maximum length of the spleen >16cm.
15. Person thought inappropriate by the investigator in consideration of the laboratory test results.
16. Pregnant or breast-feeding.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Asian; Single-dose	Drug: Efepoetin Alfa; Single-intravenous administration; Other Names: GX-E4
Experimental: Caucasian; Single-dose	Drug: Efepoetin Alfa; Single-intravenous administration; Other Names: GX-E4

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PharmacoKinetic parameters (Cmax)	To measure the concentration of Efepoetin alfa in serum for 336 hours after administration of the investigational product for the pharmacokinetic parameters peak blood concentration	up to 4 weeks
PharmacoKinetic parameters (AUClast)	To measure the concentration of Efepoetin alfa in serum for 336 hours after administration of the investigational product for the pharmacokinetic parameters area under the blood concentration-time curve	up to 4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PharmacoKinetic parameters (AUC0-t, AUCinf, AUC%Extrap, CL, tmax, Vd, t1/2)	Pharmacokinetic parameters of Efepoetin alfa blood concentration after a single intravenous administration of the investigational product	up to 4 weeks
Pharmacodynamic parameters (Emax, ΔEmax, AUEC, ΔAUEC)	Pharmacodynamic parameters of hemoglobin, reticulocyte and reticulocyte hemoglobin content after administration of the investigational product	up to 4 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Exploratory	Assessment or iron storage function	up to 4 weeks
Incidence, nature and severity of adverse events	Graded according to NCI CTCAE v5.0	up to 4 weeks
Immunogenicity	Incidence of anti-drug antibodies (ADAs) relative to baseline	up to 4 weeks

Collaborators and Investigators

• Sponsor: Genexine, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): October 29, 2024
• Primary Completion (Actual): March 25, 2025
• Study Completion (Actual): March 25, 2025

Study Registration Dates

• First Submitted: July 1, 2024
• First Submitted That Met QC Criteria: July 1, 2024
• First Posted (Actual): July 8, 2024

Study Record Updates

• Last Update Posted (Actual): August 21, 2025
• Last Update Submitted That Met QC Criteria: August 20, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Other Study ID Numbers: GX-E4-HV-003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No