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NCT06738901

PHASE3

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

Sponsor: SK Plasma Co., Ltd.

View on ClinicalTrials.gov

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).

Official title: 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

Key Details

Gender

MALE

Age Range

12 Years - 65 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2025-03-31

Completion Date

2026-08-31

Last Updated

2024-12-18

Healthy Volunteers

Conditions

Hemophilia A, Severe

Interventions

BIOLOGICAL

SKP-0141

Human plasma-derived coagulation factor VIII concentrate

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