Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor
Conditions
Brief summary
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
Interventions
BIOLOGICALCoagulation Factor VIIa variant
Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes
Sponsors
Catalyst Biosciences
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosis of cohort: FVII deficiency, Glanzmann Thrombasthenia, or hemophilia A 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 * Agreement to use highly effective birth control throughout the study if the subject has childbearing potential
Exclusion criteria
* Genotype of FVIID subjects with identified mutations by central lab at screening * 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, whichever is longer * Known hypersensitivity to trial or related product * Known positive antibody to FVII or FVIIa detected by central lab at screening * Be immunosuppressed * Significant contraindication to participate
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose | Dosing period for each stage in a cohort will be approximately 5 to 11 days | Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort. |
| 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 |
Countries
India, Italy, Russia, Ukraine, United States
Outcome results
None listed