An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: SKP-0141.
- Who it may be relevant to
- Registry conditions: Hemophilia A, Severe. Basic parameters: 12 years — 65 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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).
Interventions
- Biological SKP-0141
Human plasma-derived coagulation factor VIII concentrate
Primary outcome measures
- Annualized bleeding rate [Time frame: Up to 25 weeks]
Secondary outcome measures (12)
- 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]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Prevention
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT06738901 · SKP-0141_HemA_I/III_2024