Factor IX as Adjunctive Therapy to Emicizumab (EMIX)

• Status: Not yet recruiting
• Trial end date: 2024-07-01
• Target enrollment: 0
• Participant gender: Male

Summary

This trial will enroll patients with severe hemophilia A. Experiments will be run in vitro by spiking patients' blood with different molecules (currently used and theoretically proposed as adjunctive therapy to emicizumab), therefore no more than minimal direct risk to patients is expected. This is a pilot preclinical study.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Tulane University
• Collaborator: Genentech, Inc.

Criteria

Inclusion Criteria:

• Signed Informed Consent/Assent Form

• Male patients, Age 12 years and older at time of signing Informed Consent Form

• Ability to comply with the study protocol, in the investigator's judgment

• Diagnosis of severe hemophilia A and are receiving emicizumab prophylaxis, on a weekly maintenance regimen

Exclusion Criteria:

• Not able to sign consent

• Not willing to provide extra blood for the experiments.

• Patients carrying the diagnoses of other coagulopathies in addition to hemophilia A

• Patients that have received any hemostatic agent within 5 half-lives of the blood draw. This applies to any agent that might have been used for any reason prior to the blood draw and might affect the global hemostasis.