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

SK Plasma Co., Ltd.0 sites55 target enrollmentMarch 31, 2025

ConditionsHemophilia A, Severe

InterventionsSKP-0141

DrugsSKP-0141

Overview

Phase: Phase 3
Intervention: SKP-0141
Conditions: Hemophilia A, Severe
Sponsor: SK Plasma Co., Ltd.
Enrollment: 55
Primary Endpoint: Annualized bleeding rate
Status: Not yet recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Similar Trials

Overview

Brief Summary

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).

Registry

clinicaltrials.gov

Start Date

March 31, 2025

End Date

August 31, 2026

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

Male

Investigators

Sponsor

SK Plasma Co., Ltd.

Responsible Party

Sponsor

Eligibility Criteria

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

Arms & Interventions

Prophylactic treatment

Intervention: SKP-0141

Outcomes

Primary Outcomes

Annualized bleeding rate

Time Frame: Up to 25 weeks

Efficacy of SKP-0141 in bleeding prophylaxis in previously treated patients with severe hemophilia A based on the number of bleeding episodes per year

Secondary Outcomes

Hemostatic response(Up to 25 weeks)
Consumption of SKP-0141 required for prophylaxis(Up to 25 weeks)
Consumption of SKP-0141 required for on-demand treatment(Up to 25 weeks)
Peak plasma concentration (Cmax)(At 1 week and 25 weeks)
Time to reach peak plasma concentration (Tmax)(At 1 week and 25 weeks)
Area under the plasma concentration versus time curve (AUC)(At 1 week and 25 weeks)
Half-life (T1/2)(At 1 week and 25 weeks)
Total plasma clearance (CL)(At 1 week and 25 weeks)
Elimination constant (Kel)(At 1 week and 25 weeks)
Volume of distribution (Vd)(At 1 week and 25 weeks)
Mean residence time (MRT)(At 1 week and 25 weeks)
Incremental in vivo recovery (IVR)(At 1 week and 25 weeks)
Incidence of treatment-emergent adverse events (TEAEs)(Up to 26 weeks)
Incidence of serious adverse events (SAEs)(Up to 26 weeks)
Incidence of adverse events of special interest (AESIs)(Up to 26 weeks)
Incidence of adverse events (AEs)(Up to 26 weeks)
Incidence of clinically significant changes(Up to 25 weeks)
Incidence of FVIII inhibitor formation(Up to 25 weeks)