Phase 3
N=55
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT00157157 ↗Enrolled (actual)
55
Serious AEs
50.9%
Results posted
Jul 2011
Primary outcome: Primary: Factor VIII Inhibitor Development — 29.1 percentage
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM) (Biological)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Baxalta now part of Shire
- Primary completion
- Sep 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Factor VIII Inhibitor Development |
29.1 | — |
| SECONDARY Bleeding Episodes Treated With 1 to ≥4 Infusions |
356; 107; 35; 19 | — |
| SECONDARY Assessment of Hemostasis for Treatment of Bleeding Episodes |
258; 177; 30; 1; 51 | — |
| SECONDARY Annualized Rate of Bleeding Episodes |
4.95 | — |
| SECONDARY Weekly rAHF-PFM Utilization |
87.1; 12.5; 606.4 | — |
| SECONDARY In Vivo Incremental Recovery |
1.81; 1.71 | — |
| SECONDARY Assessment of Intra-operative Hemostasis |
18; 4; 0; 0 | — |
| SECONDARY Assessment of Postoperative Hemostasis |
23; 2; 1; 1 | — |
| SECONDARY Assessment of Blood Loss During Surgical Procedures |
50.0; 20.0 | — |
| SECONDARY Adverse Events Deemed Related to Treatment |
36.4 | — |
| SECONDARY Development of Antibodies to Heterologous Proteins |
0.00; 0.00; 0.00 | — |
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.
Eligibility Criteria
Inclusion Criteria
- The subject has severe or moderately severe hemophilia A as defined by a baseline factor VIII level 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
Data sourced from ClinicalTrials.gov (NCT00157157). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.