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

NCT06738901 | Not Yet Recruiting Phase 3 DMC

• 1 other identifier: SKP-0141_HemA_I/III_2024
• Study Type: interventional
• Target: 55
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Hemophilia A, Severe

Keywords

Congenital hemophilia A; Prophylaxis; On-demand; FVIII concentrate; Hemophilia A

Outcome Measures

Primary Outcomes (1)

• 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

  Up to 25 weeks

Secondary Outcomes (18)

• 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

Other Outcomes (1)

• Hemostatic response in surgical prophylaxis

  Perioperatively/Periprocedurally

Study Arms (1)

Prophylactic treatment

EXPERIMENTAL

Biological: SKP-0141

Interventions

SKP-0141 BIOLOGICAL

Human plasma-derived coagulation factor VIII concentrate

Prophylactic treatment

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• SK Plasma Co., Ltd.: lead

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Byung Nam Chung

CONTACT

82-2-2008-2567; byung-nam.chung@sk.com

Garam Kim, M.S.

CONTACT

+82-2-2008-2062; kgram@sk.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 8, 2024
• First Posted: December 18, 2024
• Study Start: March 31, 2025
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): August 31, 2026
• Last Updated: December 18, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share