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

Phase 3

Not yet recruiting

• Conditions: Hemophilia A, Severe
• Interventions: Biological: SKP-0141

• Registration Number: NCT06738901
• Lead Sponsor: SK Plasma Co., Ltd.

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).

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-12
• Study First Submitted: 2024-12-08
• Study First Submitted QC: 2024-12-13
• Last Update Submitted: 2024-12-13
• Study First Posted: 2024-12-18
• Last Update Posted: 2024-12-18
• Study Start: 2025-03-31
• Primary Completion: 2026-07-31
• Study Completion: 2026-08-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: Male
• Target Recruitment: 55

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Prophylactic treatment	SKP-0141	-

Primary Outcome Measures

Name	Time	Method
Annualized bleeding rate	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 Outcome Measures

Name	Time	Method
Hemostatic response	Up to 25 weeks	Efficacy of SKP-0141 for the treatment of breakthrough bleeding episodes using a 4-point scale in previously treated patients with severe hemophilia A
Consumption of SKP-0141 required for prophylaxis	Up to 25 weeks	Dose of SKP-0141 injections (IU/kg/year and IU/kg/month) required for prophylaxis in previously treated patients with severe hemophilia A
Consumption of SKP-0141 required for on-demand treatment	Up to 25 weeks	Dose/number of SKP-0141 injections (IU/kg/bleed) required for treatment of bleeding episodes in previously treated patients with severe hemophilia A
Peak plasma concentration (Cmax)	At 1 week and 25 weeks	Maximum plasma concentration of SKP-0141 in previously treated patients with severe hemophilia A
Time to reach peak plasma concentration (Tmax)	At 1 week and 25 weeks	Time to reach peak plasma concentration of SKP-0141 in previously treated patients with severe hemophilia A
Area under the plasma concentration versus time curve (AUC)	At 1 week and 25 weeks	Area under the plasma concentration versus time curve in previously treated patients with severe hemophilia A
Half-life (T1/2)	At 1 week and 25 weeks	Half-life of SKP-0141 in previously treated patients with severe hemophilia A
Total plasma clearance (CL)	At 1 week and 25 weeks	Total plasma clearance of SKP-0141 in previously treated patients with severe hemophilia A
Elimination constant (Kel)	At 1 week and 25 weeks	Elimination rate constant of SKP-0141 in previously treated patients with severe hemophilia A
Volume of distribution (Vd)	At 1 week and 25 weeks	Volume of distribution of SKP-0141 in previously treated patients with severe hemophilia A
Mean residence time (MRT)	At 1 week and 25 weeks	Mean residence time in vivo of SKP-0141 in previously treated patients with severe hemophilia A
Incremental in vivo recovery (IVR)	At 1 week and 25 weeks	Incremental in vivo recovery (IVR) in previously treated patients with severe hemophilia A
Incidence of treatment-emergent adverse events (TEAEs)	Up to 26 weeks	Incidence of treatment-emergent adverse events in previously treated patients with severe hemophilia A
Incidence of serious adverse events (SAEs)	Up to 26 weeks	Incidence of serious adverse events in previously treated patients with severe hemophilia A
Incidence of adverse events of special interest (AESIs)	Up to 26 weeks	Incidence of adverse events of special interest in previously treated patients with severe hemophilia A
Incidence of adverse events (AEs)	Up to 26 weeks	Incidence of adverse events in previously treated patients with severe hemophilia A
Incidence of clinically significant changes	Up to 25 weeks	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
Incidence of FVIII inhibitor formation	Up to 25 weeks	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