NCT03407651

Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B

Phase 2 Study to Evaluate the Pharmacokinetics, Efficacy and Safety of a Daily Subcutaneous Treatment Regimen With Marzeptacog Alfa (Activated) for Bleeding Prophylaxis in Adult Subjects With Hemophilia A and B Subjects With an Inhibitor

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Catalyst Biosciences · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Multi-center, open-label Phase 2 study to evaluate the PK, bioavailability, PD, efficacy and safety of a daily SC treatment regimen with MarzAA for bleeding prophylaxis in adult subjects with hemophilia A or B with an inhibitor. The study will enroll and dose, both intravenously and subcutaneously, a total of 12 adult male subjects with severe congenital hemophilia A or B with an inhibitor, and history of frequent bleeding episodes during the 6 months prior to enrollment, as per the individual's bleeding and treatment records. Once a subject is enrolled into the trial, the study will be conducted in three parts (occurring consecutively): Part 1a (24 hours): Single IV administration of MarzAA; Part 1b (48 hours): Single SC administration of MarzAA; Part 2: Daily SC administration. Dose escalation in Part 2 will occur if breakthrough bleeding occurs. Subjects are treated for 50 days at the final dose level required.

Conditions

• Hemophilia A With Inhibitor
• Hemophilia B With Inhibitor

Interventions

Type	Name	Description
BIOLOGICAL	Coagulation Factor VIIa variant	Single intravenous injection of MarzAA, followed by single subcutaneous injection of MarzAA, followed by daily subcutaneous injection of MarzAA for 50 days at final dose level required.

Timeline

• Start date: 2017-12-18
• Primary completion: 2019-03-15
• Completion: 2019-04-13
• First posted: 2018-01-23
• Last updated: 2021-09-23
• Results posted: 2021-09-23

Locations

9 sites across 5 countries: Armenia, Georgia, Poland, Russia, South Africa

Regulatory

• FDA-regulated drug study