NCT06738901

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

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 55 (estimated)
• Sponsor: SK Plasma Co., Ltd. · Industry
• Sex: Male
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Hemophilia A, Severe

Interventions

Type	Name	Description
BIOLOGICAL	SKP-0141	Human plasma-derived coagulation factor VIII concentrate

Timeline

• Start date: 2025-03-31
• Primary completion: 2026-07-31
• Completion: 2026-08-31
• First posted: 2024-12-18
• Last updated: 2024-12-18