Severe Hemophilia A
Conditions
Brief summary
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.
Interventions
DRUGrFVIIIFc
Single dose
Sponsors
Bioverativ Therapeutics Inc.
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male and 12 years of age and older and weigh at least 40 kg 2. Diagnosed with severe hemophilia A (baseline Factor VIII level less than 1%) 3. History of at least 100 exposure days to any Factor VIII product
Exclusion criteria
1. History of Factor VIII inhibitors 2. Kidney or liver dysfunction 3. Diagnosed with another coagulation defect other than hemophilia A
Design outcomes
Primary
| Measure | 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
| Measure | 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 |
Countries
Hong Kong, Israel, United States
Outcome results
None listed