A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients With Severe Hemophilia A
Phase 2/Phase 3
12 Years
65 Years
12 Years
65 Years
Open (Enrolling)
Male
Hemophilia A
Male
Hemophilia A
Trial Information
A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients With Severe Hemophilia A
Main
Inclusion Criteria:
- Participant and/or legal representative has/have voluntarily provided signed informed
consent
- Participant is 12 to 65 years old at the time of screening
- Participant is male with severe hemophilia A (Factor VIII (FVIII) clotting activity <
1%) as confirmed by central laboratory at screening after the appropriate washout
period or a documented FVIII clotting activity <1%
- Participant has been previously treated with plasma-derived FVIII concentrates or
recombinant FVIII for ≥150 documented exposure days (EDs)
- Participant is currently receiving prophylaxis or on-demand therapy with FVIII
- Participant is willing and able to comply with the requirements of the protocol
Main Exclusion Criteria:
- Participant has detectable FVIII inhibitory antibodies (≥ 0.6 Bethesda Units (BU)
using the Nijmegen modification of the Bethesda assay) as confirmed by central
laboratory at screening
- Participant has history of FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen
modification of the Bethesda assay or ≥ 0.6 BU using the Bethesda assay) at any time
prior to screening
- Participant has been diagnosed with an inherited or acquired hemostatic defect other
than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Factorial Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Annualized Bleeding Rate (ABR)
Outcome Time Frame:
9 months
Safety Issue:
No
Principal Investigator
Benny Sorensen, MD, PhD
Investigator Role:
Study Director
Investigator Affiliation:
Baxter Healthcare Corporation
Authority:
United States: Food and Drug Administration
Study ID:
261201
NCT ID:
NCT01736475
Start Date:
January 2013
Completion Date:
September 2014
Related Keywords:
- Hemophilia A
- Hemophilia A
Name | Location |
|---|---|
| University of Oklahoma Health Sciences Center | Oklahoma City, Oklahoma 73104 |
| Arkansas Children's Hospital | Little Rock, Arkansas 72202-3591 |
| Cincinnati Children's Hospital Medical Center | Cincinnati, Ohio 45229-3039 |
| University of Louisville Hospital | Louisville, Kentucky 40202 |
| Duke University Medical Center | Durham, North Carolina 27710 |
| Palmetto Health | Columbia, South Carolina 29203 |
| University of Florida, Gainesville | Gainesville, Florida 32611 |
| Penn State Hershey Cancer Center | Hershey, Pennsylvania 17033 |
| University of North Carolina Chapel Hill | Chapel Hill, North Carolina |
| Tulane University Medical School | New Orleans, Louisiana 70112 |
| Penn Hemophilia Program | Philadelphia, Pennsylvania 19104 |
| Puget Sound Blood Group | Seattle, Washington 98104 |
