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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06738901
- Acronym / sponsor ID
- SKP-0141_HemA_I/III_2024
- Conditions
- Hemophilia A, Severe
- Keywords
- Congenital hemophilia A, Prophylaxis, On-demand, FVIII concentrate, Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- SK Plasma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 31 Mar 2025 (estimated)
- Primary completion
- 31 Jul 2026 (estimated)
- Study completion
- 31 Aug 2026 (estimated)
- First posted
- 18 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Prophylactic treatment | Experimental | Biological: SKP-0141 |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SKP-0141Human plasma-derived coagulation factor VIII concentrate | Biological | Prophylactic treatment |
Primary outcome
Annualized bleeding rate
Time frame: Up to 25 weeks
Efficacy of SKP-0141 in bleeding prophylaxis in previously treated patients with severe hemophilia A based on the number of bleeding episodes per year
18 secondary outcomes
Hemostatic response
Time frame: Up to 25 weeks
Consumption of SKP-0141 required for prophylaxis
Time frame: Up to 25 weeks
Consumption of SKP-0141 required for on-demand treatment
Time frame: Up to 25 weeks
Peak plasma concentration (Cmax)
Time frame: At 1 week and 25 weeks
Time to reach peak plasma concentration (Tmax)
Time frame: At 1 week and 25 weeks
Area under the plasma concentration versus time curve (AUC)
Time frame: At 1 week and 25 weeks
Half-life (T1/2)
Time frame: At 1 week and 25 weeks
Total plasma clearance (CL)
Time frame: At 1 week and 25 weeks
Elimination constant (Kel)
Time frame: At 1 week and 25 weeks
Volume of distribution (Vd)
Time frame: At 1 week and 25 weeks
Mean residence time (MRT)
Time frame: At 1 week and 25 weeks
Incremental in vivo recovery (IVR)
Time frame: At 1 week and 25 weeks
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: Up to 26 weeks
Incidence of serious adverse events (SAEs)
Time frame: Up to 26 weeks
Incidence of adverse events of special interest (AESIs)
Time frame: Up to 26 weeks
Incidence of adverse events (AEs)
Time frame: Up to 26 weeks
Incidence of clinically significant changes
Time frame: Up to 25 weeks
Incidence of FVIII inhibitor formation
Time frame: Up to 25 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing SKP-0141 in people with Hemophilia A, Severe, run by SK Plasma Co., Ltd..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 18 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Byung Nam Chung (82-2-2008-2567, byung-nam.chung@sk.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized bleeding rate (time frame: Up to 25 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2024.