An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients

A Phase 1/3, Open-label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of Human Plasma-derived Factor VIII (SKP-0141) for the Treatment and Prophylaxis in Male Patients with Severe Hemophilia a

This is a prospective, multicenter, open-label study to assess efficacy, safety, pharmacokinetics (PK), and immunogenicity of human plasma-derived Factor VIII (FVIII) in previously treated patients (PTPs) with severe hemophilia A. Overall, 55 male PTPs aged 12 to 65 years old with a FVIII level of < 1% and at least 150 treatment exposure days (EDs) with a previous FVIII product will be enrolled. Patients will receive SKP-0141 at a dose of 25 to 50 IU/kg every second day or 3 times per week for at least 50 EDs and/or 6 months from the start of prophylactic treatment. Efficacy of SKP-0141 will be primarily evaluated in bleeding prophylaxis with annualized bleeding rate from start of treatment and until end of treatment (Visit 10).

Study Overview

• Status: Not yet recruiting
• Conditions: Hemophilia A, Severe
• Intervention / Treatment: Biological: SKP-0141

• Study Type: Interventional
• Enrollment (Estimated): 55
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Byung Nam Chung; Phone Number: 82-2-2008-2567; Email: byung-nam.chung@sk.com
• Study Contact Backup: Name: Garam Kim, M.S.; Phone Number: +82-2-2008-2062; Email: kgram@sk.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• A patient or parent/legal guardian who is capable of giving signed informed consent
• Patients assigned male at birth and must be 12 to 65 years old at the time of Screening
• Diagnosis of severe congenital hemophilia A, defined as an FVIII level of <1% as documented in the patient's medical records at the time of Screening
• Patients who have received or are currently receiving plasma-derived and/or recombinant FVIII products and have had at least 150 EDs with a FVIII product
• Patients who can produce viable sperm and have a partner of childbearing potential must agree to take appropriate contraceptive measures consistently during the study, starting at Screening and until 30 days after the end of study

Exclusion Criteria:

• Any history of or current FVIII inhibitors or any first order family history of FVIII inhibitors in terms of detectable FVIII inhibitors (ie, ≥0.6 Bethesda Units [BU]) using the Nijmegen-modification of the Bethesda assay
• Any known congenital or acquired coagulation disorder other than the congenital hemophilia A
• Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction, and arterial embolus within 3 months prior to Visit 1
• Experienced life-threatening bleeding episode or had major surgery or an orthopedic surgical procedure during the 3 months prior to Visit 1
• Has been tested positive for HIV with a CD4+ count ≤200/μL at Screening (if available, hepatitis B surface antigen, or hepatitis C virus antibodies, and/or positive hepatitis B virus deoxyribonucleic acid/HCV ribonucleic acid at Screening
• Platelet count <100 000/μL at Screening
• Patients with serum aspartate aminotransferase or serum alanine aminotransferase values >5 × the upper limit of normal or serum creatinine values >2 × ULN at Screening
• Patients who are currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment within 3 months prior to Visit 1
• Use of any other investigational medicinal product, cryoprecipitate, whole blood, or plasma within 30 days or 5 half-lives prior to Visit 1
• Known or suspected hypersensitivity to any FVIII product or their excipients
• Has a physical, medical, or psychological condition, that in the opinion of the PI, may interfere with the evaluation of the study.
• Are study site personnel directly affiliated with this study and their immediate families

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Prophylactic treatment	Biological: SKP-0141; Human plasma-derived coagulation factor VIII concentrate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized bleeding rate	Efficacy of SKP-0141 in bleeding prophylaxis in previously treated patients with severe hemophilia A based on the number of bleeding episodes per year	Up to 25 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hemostatic response	Efficacy of SKP-0141 for the treatment of breakthrough bleeding episodes using a 4-point scale in previously treated patients with severe hemophilia A	Up to 25 weeks
Consumption of SKP-0141 required for prophylaxis	Dose of SKP-0141 injections (IU/kg/year and IU/kg/month) required for prophylaxis in previously treated patients with severe hemophilia A	Up to 25 weeks
Consumption of SKP-0141 required for on-demand treatment	Dose/number of SKP-0141 injections (IU/kg/bleed) required for treatment of bleeding episodes in previously treated patients with severe hemophilia A	Up to 25 weeks
Peak plasma concentration (Cmax)	Maximum plasma concentration of SKP-0141 in previously treated patients with severe hemophilia A	At 1 week and 25 weeks
Time to reach peak plasma concentration (Tmax)	Time to reach peak plasma concentration of SKP-0141 in previously treated patients with severe hemophilia A	At 1 week and 25 weeks
Area under the plasma concentration versus time curve (AUC)	Area under the plasma concentration versus time curve in previously treated patients with severe hemophilia A	At 1 week and 25 weeks
Half-life (T1/2)	Half-life of SKP-0141 in previously treated patients with severe hemophilia A	At 1 week and 25 weeks
Total plasma clearance (CL)	Total plasma clearance of SKP-0141 in previously treated patients with severe hemophilia A	At 1 week and 25 weeks
Elimination constant (Kel)	Elimination rate constant of SKP-0141 in previously treated patients with severe hemophilia A	At 1 week and 25 weeks
Volume of distribution (Vd)	Volume of distribution of SKP-0141 in previously treated patients with severe hemophilia A	At 1 week and 25 weeks
Mean residence time (MRT)	Mean residence time in vivo of SKP-0141 in previously treated patients with severe hemophilia A	At 1 week and 25 weeks
Incremental in vivo recovery (IVR)	Incremental in vivo recovery (IVR) in previously treated patients with severe hemophilia A	At 1 week and 25 weeks
Incidence of treatment-emergent adverse events (TEAEs)	Incidence of treatment-emergent adverse events in previously treated patients with severe hemophilia A	Up to 26 weeks
Incidence of serious adverse events (SAEs)	Incidence of serious adverse events in previously treated patients with severe hemophilia A	Up to 26 weeks
Incidence of adverse events of special interest (AESIs)	Incidence of adverse events of special interest in previously treated patients with severe hemophilia A	Up to 26 weeks
Incidence of adverse events (AEs)	Incidence of adverse events in previously treated patients with severe hemophilia A	Up to 26 weeks
Incidence of clinically significant changes	Safety and tolerability of SKP-0141 in previously treated patients with severe hemophilia A based on the incidence of clinically significant changes from baseline in safety laboratory evaluations (hematology, serum chemistry, and urinalysis), vital signs (pre- and post-injection), physical examinations, and ECG	Up to 25 weeks
Incidence of FVIII inhibitor formation	Immunogenicity of SKP-0141 from incidence of FVIII inhibitor formation (≥0.6 Bethesda Units) calculated using the Nijmegen-modified Bethesda assay in previously treated patients with severe hemophilia A	Up to 25 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hemostatic response in surgical prophylaxis	Hemostatic response (efficacy) of SKP-0141 in surgical prophylaxis in previously treated patients with severe hemophilia A	Perioperatively/Periprocedurally

Collaborators and Investigators

• Sponsor: SK Plasma Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): March 31, 2025
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): August 31, 2026

Study Registration Dates

• First Submitted: December 8, 2024
• First Submitted That Met QC Criteria: December 13, 2024
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: December 13, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Keywords: Prophylaxis; Hemophilia A; On-demand; Congenital hemophilia A; FVIII concentrate
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A
• Other Study ID Numbers: SKP-0141_HemA_I/III_2024

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