Status:

COMPLETED

Study on the Dose-response Relationship of Pharmacodynamic Parameters in Patients With Hemophilia With Inhibitors

Lead Sponsor:

AryoGen Pharmed Co.

Conditions:

Hemophilia A With Inhibitor

Hemophilia B With Inhibitor

Eligibility:

MALE

12+ years

Phase:

EARLY_PHASE1

Brief Summary

Randomized, double-blind, single-dose, 5 ways crossover, exploratory clinical trial evaluating four different doses of AryoSeven (eptacog alfa, activated) and NovoSeven on selected pharmacodynamic par...

Detailed Description

Randomized, double-blind, single dose, 5 ways crossover, clinical trial evaluating four different doses (10 µg/kg, 30 µg/kg, 90 µg/kg, and 270 µg/kg) of AryoSeven (recombinant human FVII activated or ...

Eligibility Criteria

Inclusion

  • 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, not in bleeding episode
  • Male adults and adolescents (\>12 years)
  • Patient informed consent has been obtained \[Patients to be enrolled must also provide voluntary written informed consent to the protocol prior to screening to be eligible for the study. For adolescents, parent/legal guardian must provide consent and, wherever possible, patient assent will also be obtained. For compromised patients, their designated proxy must provide informed consent\].
  • Patients willing and able to be hospitalized prior to time of study medication administration for plasma sampling (5 times during the study).

Exclusion

  • 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 AryoSeven/NovoSeven or planned to occur during the trial
  • 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.
  • Concomitant diseases and/or medications, or any other conditions, that render the patient unsuitable for inclusion into the study in the judgement of the investigator.

Key Trial Info

Start Date :

December 29 2020

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

July 31 2022

Estimated Enrollment :

14 Patients enrolled

Trial Details

Trial ID

NCT04789954

Start Date

December 29 2020

End Date

July 31 2022

Last Update

September 10 2022

Active Locations (1)

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Page 1 of 1 (1 locations)

1

Comprehensive Hemophilia Care Center

Tehran, Iran