NCT06738901Not yet recruitingPhase 3
An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients
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
Source: ClinicalTrials.gov · Last updated 18 Dec 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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).
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 55 (estimated)
- First submitted
- 8 Dec 2024
- Start date
- 31 Mar 2025
- Primary completion date
- 31 Jul 2026
- Lead sponsor
- SK Plasma Co., Ltd.
- Diseases
- Haemophilia
- NCT number
- NCT06738901
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 55
- From 31 Mar 2025 to the primary completion date, 31 Jul 2026
- No sites listed in no country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
- 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
Other illness
- 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
- 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.
Lab values
- 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
Other
- Are study site personnel directly affiliated with this study and their immediate families
Who the study is looking for
- 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
Age in the registry: 12 years to 65 years · Sex: Male · Does not accept healthy volunteers
This page is information, not medical advice. Discuss any trial with your doctor.