Clinical trial NCT06738901: An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients
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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Hemophilia A, Severe
Keywords: Congenital hemophilia A
Prophylaxis
On-demand
FVIII concentrate
Hemophilia A
Lead sponsor: SK Plasma Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2024-12-18
Enrollment: 55
Sex: MALE
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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).


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

Primary outcomes:
measure: Annualized bleeding rate
timeFrame: Up to 25 weeks
Secondary outcomes:
measure: Hemostatic response
timeFrame: Up to 25 weeks
measure: Consumption of SKP-0141 required for prophylaxis
timeFrame: Up to 25 weeks
measure: Consumption of SKP-0141 required for on-demand treatment
timeFrame: Up to 25 weeks
measure: Peak plasma concentration (Cmax)
timeFrame: At 1 week and 25 weeks
measure: Time to reach peak plasma concentration (Tmax)
timeFrame: At 1 week and 25 weeks
measure: Area under the plasma concentration versus time curve (AUC)
timeFrame: At 1 week and 25 weeks
measure: Half-life (T1/2)
timeFrame: At 1 week and 25 weeks
measure: Total plasma clearance (CL)
timeFrame: At 1 week and 25 weeks
measure: Elimination constant (Kel)
timeFrame: At 1 week and 25 weeks
measure: Volume of distribution (Vd)
timeFrame: At 1 week and 25 weeks
measure: Mean residence time (MRT)
timeFrame: At 1 week and 25 weeks
measure: Incremental in vivo recovery (IVR)
timeFrame: At 1 week and 25 weeks
measure: Incidence of treatment-emergent adverse events (TEAEs)
timeFrame: Up to 26 weeks
measure: Incidence of serious adverse events (SAEs)
timeFrame: Up to 26 weeks
measure: Incidence of adverse events of special interest (AESIs)
timeFrame: Up to 26 weeks
measure: Incidence of adverse events (AEs)
timeFrame: Up to 26 weeks
measure: Incidence of clinically significant changes
timeFrame: Up to 25 weeks
measure: Incidence of FVIII inhibitor formation
timeFrame: Up to 25 weeks
Other outcomes:
measure: Hemostatic response in surgical prophylaxis
timeFrame: Perioperatively/Periprocedurally
Locations:
Study officials:
References: