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

Официальное название

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

Обзор

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).

Вмешательства

• Биопрепарат SKP-0141
  Human plasma-derived coagulation factor VIII concentrate

Первичные конечные точки

• Annualized bleeding rate [Срок оценки: Up to 25 weeks]

Вторичные конечные точки (12)

• Hemostatic response [Срок оценки: Up to 25 weeks]
• Consumption of SKP-0141 required for prophylaxis [Срок оценки: Up to 25 weeks]
• Consumption of SKP-0141 required for on-demand treatment [Срок оценки: Up to 25 weeks]
• Peak plasma concentration (Cmax) [Срок оценки: At 1 week and 25 weeks]
• Time to reach peak plasma concentration (Tmax) [Срок оценки: At 1 week and 25 weeks]
• Area under the plasma concentration versus time curve (AUC) [Срок оценки: At 1 week and 25 weeks]
• Half-life (T1/2) [Срок оценки: At 1 week and 25 weeks]
• Total plasma clearance (CL) [Срок оценки: At 1 week and 25 weeks]
• Elimination constant (Kel) [Срок оценки: At 1 week and 25 weeks]
• Volume of distribution (Vd) [Срок оценки: At 1 week and 25 weeks]
• Mean residence time (MRT) [Срок оценки: At 1 week and 25 weeks]
• Incremental in vivo recovery (IVR) [Срок оценки: At 1 week and 25 weeks]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Профилактика

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT06738901 · SKP-0141_HemA_I/III_2024

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗