NCT00157157CompletedPhase 3
Recombinant Antihemophilic Factor Manufactured and Formulated Without Added Human or Animal Proteins (rAHF-PFM): Evaluation of Immunogenicity, Efficacy, and Safety in Previously Untreated Patients With Hemophilia A
Baxalta now part of Shire35 sites in 10 countries66 target enrollmentStarted: April 1, 2004Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Enrollment
- 66
- Locations
- 35
- Primary Endpoint
- Factor VIII Inhibitor Development
Study Overview
Brief Summary
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII (FVIII) before.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- — to 6 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •The subject has severe or moderately severe hemophilia A as defined by a baseline factor VIII level <= 2% of normal, as documented at screening
- •The subject is < 6 years of age
- •The subject's legally authorized representative has provided written informed consent
Exclusion Criteria
- •The subject has a history of exposure to factor VIII other than rAHF PFM or more than 3 infusions of commercially available rAHF PFM (i.e., ADVATE) within 28 days prior to screening, as determined by the subject's medical history. Any infusion of factor VIII replacement products prior to the 28-day period excludes the subject from participation
- •The subject has received more than 3 infusions of rAHF PFM (commercially available and/or study product) between screening and prior to the initial recovery infusion
- •The subject has a detectable inhibitor to factor VIII, as measured in the screening sample by the Nijmegen assay in the central laboratory
- •The subject has a history of inhibitor to factor VIII at any time prior to screening
- •The subject has a known hypersensitivity to rAHF PFM
- •The subject has any 1 of the following laboratory abnormalities at the time of screening:
- •Platelet count < 100,000/mm^3
- •Hemoglobin concentration < 10 g/dL (100 g/L)
- •Serum creatinine > 1.5 times the upper limit of normal (ULN) for age
- •Total bilirubin > 2 times the ULN for age
- •The subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g., qualitative platelet defect or von Willebrand's disease)
- •The subject is known to be seropositive for human immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV), as determined by the subject's medical history
- •At the time of enrollment, the subject has a clinically significant chronic disease other than hemophilia A
- •The subject is currently participating in another investigational drug study, or has participated in any clinical study involving an investigational drug within 120 days of the screening visit
- •The subject (or the subject's legally authorized representative) is identified by the investigator as being unable or unwilling to cooperate with study procedures
- •The subject has received any blood product, including packed red blood cells (RBC), platelets, plasma, or cryoprecipitate
Outcomes
Primary Outcomes
Factor VIII Inhibitor Development
Time Frame: Assessed during study period which was to be at least 75 exposure days or 3 years (whichever came first)
Percentage of treated participants who developed factor VIII inhibitors
Secondary Outcomes
- Assessment of Intra-operative Hemostasis(Assessed at the time of discharge from recovery room)
- Assessment of Postoperative Hemostasis(Assessed at the time of discharge from hospital or clinic)
- Development of Antibodies to Heterologous Proteins(Assessed at baseline, throughout the duration of the study, which was to be at least 75 exposure days or 3 years (whichever came first), and at the termination visit.)
- Assessment of Blood Loss During Surgical Procedures(Predicted volumes preoperatively estimated and actual volumes intraoperatively recorded)
- Bleeding Episodes Treated With 1 to ≥4 Infusions(Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first))
- Assessment of Hemostasis for Treatment of Bleeding Episodes(Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first))
- Annualized Rate of Bleeding Episodes(Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first))
- Weekly rAHF-PFM Utilization(Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first))
- In Vivo Incremental Recovery(30 minutes pre-infusion to 30 minutes post-infusion)
- Adverse Events Deemed Related to Treatment(Reported during the study period which was to be at least 75 exposure days or 3 years (whichever came first))
Investigators
Study Sites (35)
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