Phase 1, Open-label, Multi-Center, to investigate Safety, Efficacy, and Pharmacokinetics (PK) of PGA40 in Previously Treated Subjects with Severe Hemophilia A
- Conditions
- Diseases of the blood and blood -forming organs and certain disorders involving the immune mechanism
- Registration Number
- KCT0006060
- Lead Sponsor
- PanGen Biotech
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Male
- Target Recruitment
- 12
To be eligible for study entry, subjects must satisfy all of the following criteria:
1.Male aged = 12 to = 65 years.
2.Male subjects with the diagnosis of severe (FVIII:C < 1%)hemophilia documented in medical records.
3.Previously treated with plasma-derived and/or recombinant FVIII products for a minimum of 150 EDs (based on the subject’s medical records). Cryoprecipitate and frozen plasma treatment are not considered as a treatment.
4.No history of FVIII inhibitors based on the subject’s previous medical records.
5.No detectable FVIII inhibitors (=0.6) Bethesda units [BU]) as assessed by a central laboratory at the time of screening.
6.Subject who are HIV negative. If, HIV positive viral load < 400.000 copies/mL and CD4+ lymphocyte count ?200/µL at screening
7.Subjects who are willing to undergo a prophylactic treatment for at least 50 EDs.
8.Non-bleeding state (i.e., no clinical signs of active bleeding) at the time of administration of Investigational product for measurement of recovery in relation to the administration of the first dose and/or at the pharmacokinetic session.
9.Subject and/or legal representative has provided signed ICF.
Subjects will be excluded from the study if one or more of the following criteria are applicable:
1.Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII products or hamster protein.
2.Previous defined medical record of arterial thrombotic events (i.e. deep vein thrombosis, stroke, pulmonary embolism, myocardial infraction and arterial embolus)
3.Abnormal renal function (serum creatinine >2 times the upper limit of normal at screening).
4.Active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level > 5 times the upper limit of normal at screening.
5.Diagnosis of an inherited or acquired hemostatic defect other than hemophilia A.
6.Platelet count < 100,000/µL based on medical records and/or based on local laboratory values at screening.
7.A clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject’s safety or compliance.
8.Patients who are receiving immunotherapy drugs other than chemotherapy, anti-RNA tumor virus chemotherapy, or who use oral or intravenous corticosteroids chronically within 3 months of screening. However, short-term (within 14 days) prednisone / methylprednisolone is allowed for the treatment of diseases such as synovitis and asthma, according to the physician's judgment.
9.Use of an investigational medicinal product within 30 days prior to the first PGA40 administration.
10.Subjects who received blood transfusions within 30 days of screening.
Study & Design
- Study Type
- Interventional Study
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Evaluate PK characteristics of PGA40
- Secondary Outcome Measures
Name Time Method Demonstrate safety of PGA40;Demonstrate the hemostatic efficacy of PGA40 severe hemophilia A subjects under prophylactic treatment. Demonstrate the hemostatic efficacy of bleeding episode (BE) treatment by PGA40 in severe hemophilia A subjects;Evaluate the incidence of FVIII inhibitor development during 6 months and 50 EDs PGA40 treatment period.