Summary

Eligibility
for people ages 12 years and up (full criteria)
Location
at UC Davis UCSF
Dates
study started
estimated completion

Description

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.

Official Title

Phase 1/2 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Marzeptacog Alfa (Activated) in Treatment of Episodic Bleeding in Subjects With Inherited Bleeding Disorders

Keywords

Factor VII Deficiency Glanzmann Thrombasthenia Hemophilia A With Inhibitor Hemostatic Disorders Hemophilia A Blood Coagulation Disorders Thrombasthenia Coagulation Factor VIIa variant

Eligibility

You can join if…

Open to people ages 12 years and up

  • 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

You CAN'T join if...

  • 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

Locations

  • UC Davis Medical Center not yet accepting patients
    Sacramento California 95817 United States
  • University of California -San Francisco not yet accepting patients
    San Francisco California 94143 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Catalyst Biosciences
ID
NCT04548791
Phase
Phase 1/2
Study Type
Interventional
Last Updated