Phase 3
N=25
A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa
Hemophilia A With Inhibitors · Hemophilia B With Inhibitors
Bottom Line
View on ClinicalTrials.gov: NCT02448680 ↗Enrolled (actual)
25
Serious AEs
4.2%
Results posted
Aug 2020
Primary outcome: Primary: Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement. — 0.654; 0.603 Proportion of successfully treated BEs
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Coagulation FVIIa (Recombinant) (Biological)
- Age
- Pediatric
- Sex
- Male
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
- Primary completion
- Jun 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement. |
0.654; 0.603 | — |
| PRIMARY Proportion of Successfully Treated Bleeding Episodes (Mild/Moderate/Severe) Per EMA Definition |
0.667; 0.625 | — |
| SECONDARY Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes |
0.667; 0.629 | — |
| SECONDARY Time to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes |
9.00; 12.00 | — |
| SECONDARY Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode |
3.6; 2.6 | — |
| SECONDARY Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode |
6.733; 8.287 | — |
Summary
The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or IX in 12 patients ( birth to <6 years old), and 12 patients (≥6 years old to <12 years old).
Eligibility Criteria
Inclusion Criteria
- be male with a diagnosis of congenital hemophilia A or B of any severity
- have one of the following:
- a positive inhibitor test BU ≥5, OR
- a Bethesda Unit (BU) 200/µL)
- have a known allergy or hypersensitivity to rabbits
- have platelet count <100,000/mL
- have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of study drug
- have received an investigational drug within 30 days of first study drug administration, or be expected to receive such drug during participation in this study
Data sourced from ClinicalTrials.gov (NCT02448680). 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.