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

NCT ID: NCT06738901

Last Updated: 2024-12-18

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 55 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-03-31
• Study Completion Date: 2026-08-31

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).

Conditions

Hemophilia A, Severe

Keywords

Congenital hemophilia A; Prophylaxis; On-demand; FVIII concentrate; Hemophilia A

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

Prophylactic treatment

Group Type: EXPERIMENTAL

SKP-0141

Intervention Type: BIOLOGICAL

Human plasma-derived coagulation factor VIII concentrate

Interventions

SKP-0141

Human plasma-derived coagulation factor VIII concentrate

Intervention Type: BIOLOGICAL

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
• 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

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

SK Plasma Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Byung Nam Chung

Role: CONTACT

Phone: 82-2-2008-2567

Email: byung-nam.chung@sk.com

Garam Kim, M.S.

Role: CONTACT

Phone: +82-2-2008-2062

Email: kgram@sk.com

Other Identifiers

SKP-0141_HemA_I/III_2024

Identifier Type: -

Identifier Source: org_study_id