an open-label study evaluating the efficacy, safety, pharmacokinetics, and immunogenicity of skp-0141 for the treatment and prophylaxis in severe hemophilia a patients

Conditions: Hemophilia A, Severe

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).


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

Keywords: congenital hemophilia a prophylaxis on-demand fviii concentrate hemophilia a