Health Condition 1: D66- Hereditary factor VIII deficiency
Conditions
Interventions
Intervention1: Study Drug Name: SKP-0141: Route: IV injection
Dose: Lyophilized powder and solvent for solution for injection
Dose Range: 25 to 50 IU/kg
Treatment period: 25 weeks
Control Interventio
Sponsors
SK Plasma Co Ltd
IQVIA RDSIndia Pvt Ltd
Eligibility
Inclusion criteria
Inclusion criteria: Patients are eligible to be included in the study only if all of the following criteria apply: 1. A patient or parent/legal guardian (as appropriate) who is capable of giving signed informed consent, and where required, the patient is capable of providing assent), which includes compliance with the requirements and restrictions listed in the Informed Consent Form (ICF), Assent Form, and in this protocol. 2. Patients assigned male at birth and must be 12 to 65 years old (inclusive) at the time of Screening (signing the ICF by the patient or their parent/legal guardian or provision of assent, as appropriate). 3. Diagnosis of severe congenital hemophilia A, defined as an FVIII level of greater than 1 percent as documented in the patient s medical records. Note, if available documented evidence of FVIII level is not within 4 weeks of Screening visit, a baseline FVIII level assessments will be done at Screening. 4. 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. 5. 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 (EOS) visit and also refrain from donating sperm during this period. Patients with a partner or partners who is (are) not of childbearing potential are exempt from these requirements.
Exclusion criteria
Exclusion criteria: Patients are excluded from the study if any of the following criteria apply: 1. Any history of or current FVIII inhibitors or any first order family history of FVIII inhibitors in terms of detectable FVIII inhibitors (ie, less than or equal to 0.6 Bethesda Units [BU]) using the Nijmegen-modification of the Bethesda assay 2. Any known congenital or acquired coagulation disorder other than the congenital hemophilia A. 3. Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction, and arterial embolus within 3 months prior to Visit 1.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annualized bleeding rate (number of bleeding episodes per year)Timepoint: Visit 10 | — |
Secondary
| Measure | Time frame |
|---|---|
| Patient assessment of hemostatic response for any breakthrough bleeding episode, using a 4-point scale from start of treatment and until Visit 10 Dose of SKP-0141 injections (IU/kg/year and IU/kg/month) required for prophylaxis from start of treatment and until Visit 10 Timepoint: Visit 10 | — |
Countries
India, Indonesia, Malaysia, Philippines, Republic of Korea, Thailand, Turkey
Contacts
Public ContactShweta Pradhan
IQVIA RDS (India) Private Limited
Outcome results
None listed