Pharmacodynamic, Pharmacokinetic Study ofAryoSeven with Novoseven®, in Patient with Hemophilia A or B with Inhibitors

Phase 3

Conditions: Condition 1: Hemophilia A with inhibitor. Condition 2: Hemophilia B with inhibitor.
Deficiency factor VIII (with functional defect)
Hereditary factor IX deficiency (with functional defect)

Registration Number: IRCT2017021831193N2

Lead Sponsor: AryoGen Pharmed

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Complete

Sex: Male

Target Recruitment: 48

Inclusion Criteria

Confirmed diagnosis of congenital haemophilia A or B with inhibitors to FVIII or FIX titer >5 Bethesda Units [BU]
-With > 2 episodes of bleeding/year requiring treatment with FVII infusions, non in bleeding episode
-Male subjects
-Adult and children (>12 years)
-Patients to be enrolled must also provide voluntary written informed consent to the protocol to be eligible for the study. For minor patients, parent/legal guardian will provide consent and, when possible, patient assent will also be obtained. For compromised patients, their designated proxy must provide informed consent.
-For the PK/PD phase, patients will be hospitalized at time of study medication administration for plasma sampling (2 times during the study).

Exclusion Criteria

-Any other type of congenital or acquired coagulopathy, such as: liver disease (hepatitis), vitamin k deficiency, uremia, malignancy.
-Antibodies against Factor VII.
-Ongoing bleeding prophylaxis regimens with Novoseven or planned to occur during the trial.
-Patients who have received routine (prophylactic) treatment with rFVIIa in the period between screening visit (visit 1) and visit 2 of this study (first dose administration).
-Platelet count less than 100.000 platelets/mcL (at screening visit).
-Any clinical sign or known history of arterial thrombotic event or deep venous- thrombosis or pulmonary embolism.
-HIV positive with current CD4+ count of less than 200/µL.
-Liver cirrhosis.
-Factor VIII/IX immune tolerance induction regimen planned to occur during the trial.
-Known hypersensitivity to the study medication.
-Parallel participation in another experimental drug trial.
-Parallel participation in another marketed drug trial that may affect the primary end point of the study.