A Randomized, Two-armed, Double-blind, Single-dose, Crossover, Two-sequence, Bioequivalence Clinical Trial to Compare PK Parameters and Safety of rFVIII-Fc (AryoGen Pharmed Co.) Versus Elocta® in PTPs With Severe Hemophilia A

AryoGen Pharmed Co.5 sites in 1 country50 target enrollmentJuly 22, 2023

ConditionsSevere Hemophilia A

InterventionsFactor VIII, recombinant human with Fc fusion (rFVIII-Fc)

DrugsFactor VIII, recombinant human with Fc fusion (rFVIII-Fc)

Overview

Phase: Phase 3
Intervention: Factor VIII, recombinant human with Fc fusion (rFVIII-Fc)
Conditions: Severe Hemophilia A
Sponsor: AryoGen Pharmed Co.
Enrollment: 50
Locations: 5
Primary Endpoint: dose-normalized Area Under the Curve (dnAUC last)
Status: Completed
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The study is designed as a randomized, two-armed, double-blind, single-dose, crossover, two-sequence, active-controlled, multi-center, bioequivalence clinical trial with a primary endpoint of dose-normalized area under the curve (dnAUC last)

Registry

clinicaltrials.gov

Start Date

July 22, 2023

End Date

September 27, 2023

Last Updated

2 years ago

Study Type

Interventional

Study Design

Crossover

Sex

Male

Investigators

Sponsor

AryoGen Pharmed Co.

Responsible Party

Sponsor

Eligibility 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
•Hb ≥ 8 mg/dL
•eGFR ≥ 30 mL/min
•ALT or AST ≤ 5×ULN
•Serum bilirubin ≤ 1.5×ULN

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 FVIII 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)

Arms & Interventions

AryoGen Pharmed Co. rFVIII-Fc/Elocta® (Sobi Co. rFVIII-Fc)

AryoGen Pharmed Co. rFVIII-Fc, IV, 50 units/kg, single dose, then Elocta® (Sobi Co. rFVIII-Fc), IV, 50 units/kg, single dose (cross-over)

Intervention: Factor VIII, recombinant human with Fc fusion (rFVIII-Fc)

Elocta® (Sobi Co. rFVIII-Fc)/AryoGen Pharmed Co. rFVIII-Fc

Elocta® (Sobi Co. rFVIII-Fc), IV, 50 units/kg, single dose, then AryoGen Pharmed Co. rFVIII-Fc, IV, 50 units/kg, single dose (cross-over)

Intervention: Factor VIII, recombinant human with Fc fusion (rFVIII-Fc)

Outcomes

Primary Outcomes

dose-normalized Area Under the Curve (dnAUC last)

Time Frame: pre-dose, 15 minutes, 30 minutes, 1 hour, 3 hours, 6 hours, 8 hours, 24 hours, 48 hours, 72 hours, 96 hours, 120 hours post-dose

Area under the concentration-time curve measured from the time of administration to the last measurable time point

Secondary Outcomes

Area Under the Curve to Infinity (AUC inf)(pre-dose, 15 minutes, 30 minutes, 1 hour, 3 hours, 6 hours, 8 hours, 24 hours, 48 hours, 72 hours, 96 hours, 120 hours post-dose)
Volume of distribution (Vd)(pre-dose, 15 minutes, 30 minutes, 1 hour, 3 hours, 6 hours, 8 hours, 24 hours, 48 hours, 72 hours, 96 hours, 120 hours post-dose)
Immunogenicity assessment(Immunogenicity sampling was done at screening visit and day 7, 12 and 28)
Incremental Recovery (IR)(pre-dose, 15 minutes, 30 minutes, 1 hour)
Maximum Plasma Activity (Cmax)(pre-dose, 15 minutes, 30 minutes, 1 hour, 3 hours, 6 hours, 8 hours, 24 hours, 48 hours, 72 hours, 96 hours, 120 hours post-dose)
Half-life (T ½)(pre-dose, 15 minutes, 30 minutes, 1 hour, 3 hours, 6 hours, 8 hours, 24 hours, 48 hours, 72 hours, 96 hours, 120 hours post-dose)
Clearance (Cl)(pre-dose, 15 minutes, 30 minutes, 1 hour, 3 hours, 6 hours, 8 hours, 24 hours, 48 hours, 72 hours, 96 hours, 120 hours post-dose)
Safety assessment by evaluation of adverse events (AEs) and abnormal laboratory results(Adverse events collection and documentation was done during the study (up to 28 days))