NCT04789954

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

An Exploratory Study to Evaluate the Dose Response-Relationship of Pharmacodynamic Parameters of AryoSeven, in Patients With Hemophilia With Inhibitors

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: AryoGen Pharmed Co. · Industry
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

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 parameters in patients with hemophilia with inhibitors.

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 eptacog alfa, activated) and one dose of NovoSeven (30 µg/kg) on selected pharmacodynamic parameters (PD) \[Primary: Thrombin Generation Assay (TGA)\] in male adult and adolescent (\>12 years) patients with hemophilia A or B, with an inhibitors titer \>5 Bethesda Units \[BU\] and not in bleeding status. This will be an exploratory study to evaluate dose-response relationship of PD markers as surrogate efficacy endpoints.

Conditions

• Hemophilia A With Inhibitor
• Hemophilia B With Inhibitor

Interventions

Type	Name	Description
BIOLOGICAL	Eptacog alfa, activated	A dose sequence for each injection will be randomly selected from the following doses: 10 μg/kg, 30 μg/kg, 90 μg/kg, or 270 μg/kg, separated by a wash-out period of 3 days.

Timeline

• Start date: 2020-12-29
• Primary completion: 2021-08-13
• Completion: 2022-07-31
• First posted: 2021-03-10
• Last updated: 2022-09-10

Locations

1 site across 1 country: Iran