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).
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
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
Single group
SKP-0141
Biological/VaccineHuman plasma-derived coagulation factor VIII concentrate
Annualized bleeding rate
Efficacy of SKP-0141 in bleeding prophylaxis in previously treated patients with severe hemophilia A based on the number of bleeding episodes per year
Hemostatic response
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
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
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)
Maximum plasma concentration of SKP-0141 in previously treated patients with severe hemophilia A
Hemostatic response in surgical prophylaxis
Hemostatic response (efficacy) of SKP-0141 in surgical prophylaxis in previously treated patients with severe hemophilia A
Click on the lead sponsor to view all of their trials.
This trial is not recruiting at the moment. You can still explore other options:
