Clinical trial NCT01027377: Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Severe Hemophilia A
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2009-12-07
Enrollment: 16
Sex: MALE
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Safety will be assessed by physical examination, vital signs, laboratory changes overtime, adverse events, and assessment of inhibitor (Bethesda) and antibody development against rFVIIIFc.
timeFrame: 78 weeks
Secondary outcomes:
measure: Pharmacokinetic parameters will be measured such as, but not limited to Tmax, Cmax, t1/2, CL, Vd, AUC, MRT, and incremental recovery.
timeFrame: 78 weeks
measure: To determine the pharmacokinetics parameters after single administration of 25 and 65 IU/kg of rFVIIFc compared to advate
timeFrame: 78 weeks
measure: To determine the pharmacodynamic activity of FVIII over time for both doses of rFVIIIFc
timeFrame: 78 weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Research Site
state: California
city: Sacramento
country: United States
facility: Research Site
state: California
city: Waltham
country: United States
facility: Research Site
state: Massachusetts
city: Pittsburgh
country: United States
facility: Research Site
state: Pennsylvania
city: Seattle
country: United States
facility: Research Site
state: Washington
city: Hong Kong
country: Hong Kong
facility: Research Site
city: Tel Aviv
country: Israel
facility: Research Site
Study officials:
References:
citation: Katragadda S, Neelakantan S, Diao L, Wong N. Population Pharmacokinetic Analysis of Recombinant Factor VIII Fc Fusion Protein in Subjects With Severe Hemophilia A: Expanded to Include Pediatric Subjects. J Clin Pharmacol. 2021 Jul;61(7):889-900. doi: 10.1002/jcph.1854. Epub 2021 Apr 14.
citation: Powell JS, Josephson NC, Quon D, Ragni MV, Cheng G, Li E, Jiang H, Li L, Dumont JA, Goyal J, Zhang X, Sommer J, McCue J, Barbetti M, Luk A, Pierce GF. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood. 2012 Mar 29;119(13):3031-7. doi: 10.1182/blood-2011-09-382846. Epub 2012 Jan 5.