Hemophilia A, SeverePhase 3NCT06738901
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 3 study testing SKP-0141 in people with Hemophilia A, Severe, run by SK Plasma Co., Ltd..
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Hemophilia A, Severe
- What is being tested?
- SKP-0141
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- No sites listed yet
- Study dates
- 31 Mar 2025to 31 Aug 2026
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
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
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
- Being tested
- SKP-0141
- Keywords
- Congenital hemophilia A, Prophylaxis, On-demand, FVIII concentrate, Hemophilia A
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SKP-0141Biological
Human plasma-derived coagulation factor VIII concentrate
| Study group | Role | What they receive |
|---|---|---|
| Prophylactic treatment | Experimental | Not described Receives: Biological: SKP-0141 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to 25 weeks
Secondary outcomes (18)
Hemostatic response
Efficacy of SKP-0141 for the treatment of breakthrough bleeding episodes using a 4-point scale in previously treated patients with severe hemophilia A
Measured: Up to 25 weeks
Consumption of SKP-0141 required for prophylaxis
Dose of SKP-0141 injections (IU/kg/year and IU/kg/month) required for prophylaxis in previously treated patients with severe hemophilia A
Measured: Up to 25 weeks
Consumption of SKP-0141 required for on-demand treatment
Dose/number of SKP-0141 injections (IU/kg/bleed) required for treatment of bleeding episodes in previously treated patients with severe hemophilia A
Measured: Up to 25 weeks
Peak plasma concentration (Cmax)
Maximum plasma concentration of SKP-0141 in previously treated patients with severe hemophilia A
Measured: At 1 week and 25 weeks
Time to reach peak plasma concentration (Tmax)
Time to reach peak plasma concentration of SKP-0141 in previously treated patients with severe hemophilia A
Measured: At 1 week and 25 weeks
Area under the plasma concentration versus time curve (AUC)
Area under the plasma concentration versus time curve in previously treated patients with severe hemophilia A
Measured: At 1 week and 25 weeks
Half-life (T1/2)
Half-life of SKP-0141 in previously treated patients with severe hemophilia A
Measured: At 1 week and 25 weeks
Total plasma clearance (CL)
Total plasma clearance of SKP-0141 in previously treated patients with severe hemophilia A
Measured: At 1 week and 25 weeks
Elimination constant (Kel)
Elimination rate constant of SKP-0141 in previously treated patients with severe hemophilia A
Measured: At 1 week and 25 weeks
Volume of distribution (Vd)
Volume of distribution of SKP-0141 in previously treated patients with severe hemophilia A
Measured: At 1 week and 25 weeks
Mean residence time (MRT)
Mean residence time in vivo of SKP-0141 in previously treated patients with severe hemophilia A
Measured: At 1 week and 25 weeks
Incremental in vivo recovery (IVR)
Incremental in vivo recovery (IVR) in previously treated patients with severe hemophilia A
Measured: At 1 week and 25 weeks
Incidence of treatment-emergent adverse events (TEAEs)
Incidence of treatment-emergent adverse events in previously treated patients with severe hemophilia A
Measured: Up to 26 weeks
Incidence of serious adverse events (SAEs)
Incidence of serious adverse events in previously treated patients with severe hemophilia A
Measured: Up to 26 weeks
Incidence of adverse events of special interest (AESIs)
Incidence of adverse events of special interest in previously treated patients with severe hemophilia A
Measured: Up to 26 weeks
Incidence of adverse events (AEs)
Incidence of adverse events in previously treated patients with severe hemophilia A
Measured: Up to 26 weeks
Incidence of clinically significant changes
Safety and tolerability of SKP-0141 in previously treated patients with severe hemophilia A based on the incidence of clinically significant changes from baseline in safety laboratory evaluations (hematology, serum chemistry, and urinalysis), vital signs (pre- and post-injection), physical examinations, and ECG
Measured: Up to 25 weeks
Incidence of FVIII inhibitor formation
Immunogenicity of SKP-0141 from incidence of FVIII inhibitor formation (≥0.6 Bethesda Units) calculated using the Nijmegen-modified Bethesda assay in previously treated patients with severe hemophilia A
Measured: Up to 25 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2024
- Study start31 Mar 2025 (estimated)
- Primary completion31 Jul 2026 (estimated)
- Study completion31 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A, SevereStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- SK Plasma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06738901
- Sponsor's protocol ID
- SKP-0141_HemA_I/III_2024
- Status last verified
- Dec 2024
- First submitted
- 8 Dec 2024
- Last updated
- 18 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No