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rFVIII-Fc (Produced by AryoGen Pharmed Co.) Pharmacokinetic Study

Phase 3
Completed
Conditions
Severe Hemophilia A
Interventions
Drug: Factor VIII, recombinant human with Fc fusion (rFVIII-Fc)
Registration Number
NCT06137092
Lead Sponsor
AryoGen Pharmed Co.
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)

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
Male
Target Recruitment
50
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)
  • 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)

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
AryoGen Pharmed Co. rFVIII-Fc/Elocta® (Sobi Co. rFVIII-Fc)Factor VIII, recombinant human with Fc fusion (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)
Elocta® (Sobi Co. rFVIII-Fc)/AryoGen Pharmed Co. rFVIII-FcFactor VIII, recombinant human with Fc fusion (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)
Primary Outcome Measures
NameTimeMethod
dose-normalized Area Under the Curve (dnAUC last)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 Outcome Measures
NameTimeMethod
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

Area under the concentration-time curve measured from the time of administration to the infinity.

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

Volume of distribution estimated from the terminal phase

Immunogenicity assessmentImmunogenicity sampling was done at screening visit and day 7, 12 and 28

Immunogenicity of factor viii was evaluated at scheduled visits by blood sampling to determine the production of inhibitor against factor viii.

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

Maximum plasma activity during a dosing interval

Incremental Recovery (IR)pre-dose, 15 minutes, 30 minutes, 1 hour

The rise in FVIII activity in IU/dL per unit dose administered in IU/kg

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

Time required for the activity of the drug to reach half of its original value

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

Rate at which the body removes the drug, measured as the volume of the plasma cleared of drug per unit time per unit weight

Safety assessment by evaluation of adverse events (AEs) and abnormal laboratory resultsAdverse events collection and documentation was done during the study (up to 28 days)

Safety assessment, including the incidence of all reported AEs and abnormal laboratory results was done. All AEs were classified based on the Medical Dictionary for Regulatory Activities (MedDRA Desktop Browser 4.0 Beta) terms as System Organ Class (SOC) and Preferred Term (PT). All the reported events were graded according to the Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0). Moreover, seriousness of AEs was assessed according to International Council for Harmonization (ICH-E2B) guidelines. The causality relation was assessed based on the World Health Organization (WHO) criteria.

Trial Locations

Locations (5)

Dastqeib Hospital

🇮🇷

Shiraz, Iran, Islamic Republic of

Mofid Hospital

🇮🇷

Tehran, Iran, Islamic Republic of

Seyed-Al-Shohada Hospital

🇮🇷

Isfahan, Iran, Islamic Republic of

Sarvar Clinic

🇮🇷

Mashhad, Iran, Islamic Republic of

Imam Khomeini

🇮🇷

Tehran, Iran, Islamic Republic of

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