Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Conditions
Brief summary
The purpose of the trial is to evaluate the safety and efficacy of MarzAA for on-demand treatment and control of bleeding episodes in hemophilia A or B patients with inhibitors compared with their standard of care (SOC).
Interventions
BIOLOGICALMarzAA
A cross over design to assess the efficacy of a dosing regimen of 60 µg/kg of MarzAA compared with standard of care for the treatment of bleeding episodes.
Sponsors
Catalyst Biosciences
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosis of congenital hemophilia A or B with inhibitors * Male or Female, age 12 or older * History of frequent bleeding episodes * Affirmation of informed consent with signature confirmation and assent for children between ages 12 to 17 before any study related activities
Exclusion criteria
* Previous participation in a clinical trial evaluating a modified rFVIIa agent * Received an investigational drug within 30 days or 5 half-lives or absence of clinical effect * Known hypersensitivity to trial or related product * Known positive antibody to FVII or FVIIa detected by central lab at screening * Have a coagulation disorder other than hemophilia A or B * Be immunosuppressed * Significant contraindication to participate
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Bleeding episode treatment success | 24 hours after the first administration of study drug | Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment compared with standard of care. |
Countries
Armenia, Georgia, Hungary, India, Italy, Malaysia, Mexico, Poland, Russia, South Africa, Spain, Taiwan, Turkey (Türkiye), Ukraine, United Kingdom, United States
Outcome results
None listed