Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A

Study Overview
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Brief Summary
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The study is to investigate the safety, tolerability, and pharmacokinetics (the determination of the concentration of the administered drug in blood over time) of recombinant Factor VIII Fc fusion protein (rFVIIIFc) in previously-treated subjects with severe hemophilia A.

Official Title
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An Open-Label, Crossover, Dose-Escalation, and Multi-Center Study to Determine the Safety, Tolerability, and Pharmacokinetic of a Single Intravenous Injection of rFVIIIFc in Previously Treated Patients With Severe Hemophilia A

Conditions
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Severe Hemophilia A

Intervention / Treatment
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* Drug: rFVIIIFc
* Drug: rFVIIIFc


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and 12 years of age and older and weigh at least 40 kg Diagnosed with severe hemophilia A (baseline Factor VIII level less than 1%) History of at least 100 exposure days to any Factor VIII product Exclusion Criteria: History of Factor VIII inhibitors Kidney or liver dysfunction Diagnosed with another coagulation defect other than hemophilia A

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: A<br>Cohort to receive a single low dose intravenous injection of commercially available rFVIII with safety and PK assessments followed by a single low dose intravenous injection of rFVIIIFc with safety and PK assessments | Drug: rFVIIIFc<br>* Single dose<br>* Other names: Recombinant Factor VIII fusion protein;|
| Experimental: B<br>Cohort to receive a single high dose intravenous injection of commercially available rFVIII with safety and PK assessments followed by a single high dose intravenous injection of rFVIIIFc with safety and PK assessments | Drug: rFVIIIFc<br>* Single dose<br>* Other names: Recombinant Factor VIII fusion protein;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety will be assessed by physical examination, vital signs, laboratory changes overtime, adverse events, and assessment of inhibitor (Bethesda) and antibody development against rFVIIIFc. |  | 78 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetic parameters will be measured such as, but not limited to Tmax, Cmax, t1/2, CL, Vd, AUC, MRT, and incremental recovery. |  | 78 weeks | 
| To determine the pharmacokinetics parameters after single administration of 25 and 65 IU/kg of rFVIIFc compared to advate |  | 78 weeks | 
| To determine the pharmacodynamic activity of FVIII over time for both doses of rFVIIIFc |  | 78 weeks |