Phase 2
N=11
Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B
Hemophilia A With Inhibitor · Hemophilia B With Inhibitor
Bottom Line
View on ClinicalTrials.gov: NCT03407651 ↗Enrolled (actual)
11
Serious AEs
3.2%
Results posted
Sep 2021
Primary outcome: Primary: Bleeding Episode Prevention Success — 1.4640 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Coagulation Factor VIIa variant (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Catalyst Biosciences
- Primary completion
- Mar 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Bleeding Episode Prevention Success |
1.4640 | — |
| SECONDARY Occurrence of Breakthrough Bleeding |
0; 0; 5; 0 | — |
| SECONDARY Occurrence of Clinical Thrombotic Event |
0; 0; 0; 0 | — |
| SECONDARY Coagulation Assessment - Prothrombin Time |
-3.70; -2.80; -3.0; -4.80 | — |
| SECONDARY Coagulation Assessment - Activated Partial Thromboplastin Time |
-10.60; 1.30; -8.30; -15.00 | — |
| SECONDARY Coagulation Assessment - Fibrinogen |
-10.0; -15.00; -4.0; -4.0 | — |
| SECONDARY Number of Events of Antibody Formation |
0; 0; 0; 0 | — |
| SECONDARY Number of Events of an Antibody Response |
0; 0; 0; 0 | — |
| SECONDARY Thrombogenicity Assessment |
0; 0 | — |
Summary
Phase 2, multi-center, open-label study designed to evaluate the PK, bioavailability, PD, efficacy and safety of a daily subcutaneous [SC] treatment regimen with MarzAA for bleeding prophylaxis in 12 adult subjects with hemophilia A or B with an inhibitor and history of frequent spontaneous bleeding episodes.
Eligibility Criteria
Inclusion Criteria
- Severe congenital hemophilia A or B with an inhibitor.
- History of frequent spontaneous bleeding episodes.
- Male, age 18 or older.
- Affirmation of informed consent with signature confirmation before any trial-related activities.
Exclusion Criteria
- Receiving prophylaxis treatment.
- Previous participation in a clinical trial evaluating a modified rFVIIa agent.
- Known positive antibody to FVII or FVIIa detected by central laboratory at screening.
- Have a coagulation disorder other than hemophilia A or B.
- Significant contraindication to participation.
Data sourced from ClinicalTrials.gov (NCT03407651). 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.