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

Catalyst Biosciences

Status and phase: Completed; Phase 2

Conditions: Hemophilia B With Inhibitor; Hemophilia A With Inhibitor

Treatments: Biological: Coagulation Factor VIIa variant

Study type: Interventional

Funder types: Industry

Identifiers

NCT03407651

MAA-201

Details and patient eligibility

About

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.

Full 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.

Enrollment: 11 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Severe congenital hemophilia A or B with an inhibitor.
• History of frequent spontaneous bleeding episodes.
• Male, age 18 or older.
• Affirmation of informed consent with signature confirmation before any trial-related activities.

Exclusion criteria

• Receiving prophylaxis treatment.
• Previous participation in a clinical trial evaluating a modified rFVIIa agent.
• Known positive antibody to FVII or FVIIa detected by central laboratory at screening.
• Have a coagulation disorder other than hemophilia A or B.
• Significant contraindication to participation.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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11 participants in 3 patient groups

Part 1a

Experimental group

Description:

Coagulation Factor VIIa variant, 18 µg/kg by intravenous route

Treatment:

Biological: Coagulation Factor VIIa variant

Part 1b

Experimental group

Description:

Coagulation Factor VIIa variant, 30 µg/kg by subcutaneous route

Treatment:

Biological: Coagulation Factor VIIa variant

Part 2

Experimental group

Description:

Coagulation Factor VIIa variant, 30, 60, 90, 120 µg/kg by subcutaneous route

Treatment:

Biological: Coagulation Factor VIIa variant