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

Catalyst Biosciences

Status and phase: Terminated; Phase 3

Conditions: Hemophilia B With Inhibitor; Hemophilia A With Inhibitor

Treatments: Biological: MarzAA

Study type: Interventional

Funder types: Industry

Identifiers

NCT04489537

MAA-304

Details and patient eligibility

About

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).

Enrollment: 18 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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18 participants in 2 patient groups

MarzAA

Experimental group

Description:

Coagulation Factor VIIa variant, 60 µg/kg by subcutaneous route, administered on-demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis

Treatment:

Biological: MarzAA

Standard of Care

Active Comparator group

Description:

Standard of care administered on-demand during bleeding episodes

Treatment:

Biological: MarzAA