NCT06738901Not yet recruitingPhase 3
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
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Not yet recruiting
- Sponsor
- SK Plasma Co., Ltd.
- Enrollment
- 55
- Primary Endpoint
- Annualized bleeding rate
Study 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).
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Prevention
- Masking
- None
Eligibility Criteria
- Ages
- 12 Years to 65 Years (Child, Adult, Older Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Prophylactic treatment
Experimental
Intervention: SKP-0141 (Biological)
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
- 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)
- Half-life (T1/2)(At 1 week and 25 weeks)
- 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)
- 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)
Investigators
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