Severe hemophilia A. Symptomatic hemophilia A carrier
Conditions
Interventions
Intervention 1: Intervention group: rFVIII-Fc (Aryogen Pharmed Co.), IV, 50 units/kg, Single dose, then rFVIII-Fc (Elocta®, Sobi Co.), Cross-over. Intervention 2: Intervention group: rFVIII-Fc (Elocta
Sponsors
AryoGen Company
Eligibility
Sex/Gender
Male
Age
12 Years to No maximum
Inclusion criteria
Inclusion criteria: Male patients = 12 years, with signed informed consent by the patient, or the patient's legally authorized representative for patients under the legal age Diagnosed with severe hemophilia A (endogenous FVIII <1% [1 IU/dL]) History of at least 150 documented prior exposure days to any FVIII product Having adequate bone marrow and organ function:• Plt = 80,000 cells/µL • Hgb = 8 mg/dL• eGFR = 30 mL/min• ALT or AST = 5×ULN• Serum bilirubin = 1.5×ULN
Exclusion criteria
Exclusion criteria: Measurable anti-drug antibody activity against FVIII (= 0.6 BU/mL) at screening or a history of developing anti FVIII antibody History of other coagulation disorders except for hemophilia A Acute hemorrhagic state Infection with HCV or HBV HIV-positive patients Infusion of any products containing FVIII within 7 days prior to first administration Previous treatment with commercially available extended half-life products Receiving drugs which increase bleeding tendency (e.g: Anti-coagulants, antiplatelets, omega 3, Vit E, etc.) within 2 weeks of screening. NSAIDs are permitted. Current systemic treatment with immunosuppressive drugs Hypersensitivity or anaphylaxis associated with any FVIII concentrate or intravenous immunoglobulin (IVIg) Planned elective surgery Current enrolment or willing to enroll in any other experimental study during the time of current trial Subjects assessed by the investigator to be unable or unwilling to comply with the requirements of the protocol (e.g.: physical, psychological and mental problems)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| DnAUC last (dose-normalized area under the curve). Timepoint: 12 days after the first intervention. Method of measurement: One-Stage Assay (OSA) and Chromogenic Substrate Assay (CSA). | — |
Secondary
| Measure | Time frame |
|---|---|
| AUC inf. Timepoint: pre-dose and 0.25, 0.5, 1, 3, 6, 8, 24, 48, 72, 96 and 120 h after each infusion. Method of measurement: One-Stage Assay (OSA) and Chromogenic Substrate Assay (CSA).;Cmax. Timepoint: pre-dose and 0.25, 0.5, 1, 3, 6, 8, 24, 48, 72, 96 and 120 h after each infusion. Method of measurement: One-Stage Assay (OSA) and Chromogenic Substrate Assay (CSA).;Incremental recovery (IR). Timepoint: pre-dose and 0.25, 0.5, 1, 3, 6, 8, 24, 48, 72, 96 and 120 h after each infusion. Method of measurement: One-Stage Assay (OSA) and Chromogenic Substrate Assay (CSA).;Half-life (T ½). Timepoint: pre-dose and 0.25, 0.5, 1, 3, 6, 8, 24, 48, 72, 96 and 120 h after each infusion. Method of measurement: One-Stage Assay (OSA) and Chromogenic Substrate Assay (CSA).;Vd. Timepoint: pre-dose and 0.25, 0.5, 1, 3, 6, 8, 24, 48, 72, 96 and 120 h after each infusion. Method of measurement: One-Stage Assay (OSA) and Chromogenic Substrate Assay (CSA).;Clearance. Timepoint: pre-dose and 0.25, 0.5, 1, 3, 6, 8, 24, 48, 72, 96 and 120 h after each infusion. Method of measurement: One-Stage Assay (OSA) and Chromogenic Substrate Assay (CSA).;Assessment of Adverse events. Timepoint: At screening and on days 0,1, 2, 3, 4, 5, 7, 8, 9, 10, 11, 12 and 28. Method of measurement: Clinical monitoring.;Immunogenicity: Anti-drug antibody. Timepoint: At screening and on days 7, 12 and 28. Method of measurement: Nijmegen Bethesda assay. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactDr. Nasim Anjidani
Orchid Pharmed Co.
Outcome results
None listed