Back to resultsStudy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A
Primary Purpose
Severe Hemophilia A
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
rFVIIIFc
rFVIIIFc
Sponsored by
About this trial
This is an interventional treatment trial for Severe Hemophilia A
Eligibility Criteria
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
Sites / Locations
- Research Site
- Research Site
- Research Site
- Research Site
- Research Site
- Research Site
- Research Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
A
B
Arm Description
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
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
Outcomes
Primary Outcome Measures
Safety will be assessed by physical examination, vital signs, laboratory changes overtime, adverse events, and assessment of inhibitor (Bethesda) and antibody development against rFVIIIFc.
Secondary Outcome Measures
Pharmacokinetic parameters will be measured such as, but not limited to Tmax, Cmax, t1/2, CL, Vd, AUC, MRT, and incremental recovery.
To determine the pharmacokinetics parameters after single administration of 25 and 65 IU/kg of rFVIIFc compared to advate
To determine the pharmacodynamic activity of FVIII over time for both doses of rFVIIIFc
Full Information
NCT ID
NCT01027377
First Posted
December 4, 2009
Last Updated
December 16, 2020
Sponsor
Bioverativ Therapeutics Inc.
1. Study Identification
Unique Protocol Identification Number
NCT01027377
Brief Title
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
2. Study Status
Record Verification Date
August 2018
Overall Recruitment Status
Completed
Study Start Date
December 2009 (undefined)
Primary Completion Date
July 2010 (Actual)
Study Completion Date
May 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bioverativ Therapeutics Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Hemophilia A
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
16 (Actual)
8. Arms, Groups, and Interventions
Arm Title
A
Arm Type
Experimental
Arm Description
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
Arm Title
B
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
rFVIIIFc
Other Intervention Name(s)
Recombinant Factor VIII fusion protein
Intervention Description
Single dose
Intervention Type
Drug
Intervention Name(s)
rFVIIIFc
Other Intervention Name(s)
Recombinant Factor VIII fusion protein
Intervention Description
Single dose
Primary Outcome Measure Information:
Title
Safety will be assessed by physical examination, vital signs, laboratory changes overtime, adverse events, and assessment of inhibitor (Bethesda) and antibody development against rFVIIIFc.
Time Frame
78 weeks
Secondary Outcome Measure Information:
Title
Pharmacokinetic parameters will be measured such as, but not limited to Tmax, Cmax, t1/2, CL, Vd, AUC, MRT, and incremental recovery.
Time Frame
78 weeks
Title
To determine the pharmacokinetics parameters after single administration of 25 and 65 IU/kg of rFVIIFc compared to advate
Time Frame
78 weeks
Title
To determine the pharmacodynamic activity of FVIII over time for both doses of rFVIIIFc
Time Frame
78 weeks
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Research Site
City
Los Angeles
State/Province
California
Country
United States
Facility Name
Research Site
City
Sacramento
State/Province
California
Country
United States
Facility Name
Research Site
City
Waltham
State/Province
Massachusetts
Country
United States
Facility Name
Research Site
City
Pittsburgh
State/Province
Pennsylvania
Country
United States
Facility Name
Research Site
City
Seattle
State/Province
Washington
Country
United States
Facility Name
Research Site
City
Hong Kong
Country
Hong Kong
Facility Name
Research Site
City
Tel Aviv
Country
Israel
12. IPD Sharing Statement
Citations:
PubMed Identifier
33719084
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.
Results Reference
derived
PubMed Identifier
22223821
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.
Results Reference
derived
Learn more about this trial
Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A
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