Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia

Catalyst Biosciences

Status and phase: Completed; Phase 1

Conditions: Hemophilia A Without Inhibitor; Hemophilia B Without Inhibitor; Hemophilia B; Hemophilia A; Hemophilia B With Inhibitor; Hemophilia A With Inhibitor

Treatments: Biological: MarzAA (marzeptacog alfa [activated])

Study type: Interventional

Funder types: Industry

Identifiers

NCT04072237

MAA-102

Details and patient eligibility

About

This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe Hemophilia A or B, with or without an inhibitor.

Full description

This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe Hemophilia A or B, with or without an inhibitor. The study will enroll at least 8 adult male subjects with moderate or severe Hemophilia A or B with or without an inhibitor, in each dosing stage. Each subject will receive escalating doses of MarzAA for each stage of the study (except for Stage 5, where subjects receive the same dose as in Stage 4 split between two anatomical sites).

Enrollment: 11 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Moderate or severe congenital Hemophilia A or B, with or without an inhibitor
• Male, age 18 or older
• Affirmation of informed consent with signature confirmation before any trial related activities

Exclusion criteria

• Inability to discontinue and washout prophylaxis treatment 72 hours prior to dosing.
• Previous participation in a trial involving SC Administration of rFVIIa or any trial using a modified amino-acid sequence FVIIa
• Known positive antibody to FVII or FVIIa detected by central laboratory at screening
• Have a coagulation disorder other than hemophilia A or B, with or without an inhibitor
• Significant contraindication to participate

Trial design

Primary purpose: Diagnostic

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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11 participants in 1 patient group

Study Population

Experimental group

Description:

MarzAA (Coagulation Factor VIIa variant) 18 µg/kg intravenously (Stage 1) followed by MarzAA 30 µg/kg subcutaneously (SC) (Stage 2), MarzAA 45 µg/kg SC (Stage 3), MarzAA 60 µg/kg SC (Stage 4), MarzAA 2x30 µg/kg SC (Stage 5), MarzAA 90 µg/kg SC (Stage 6), MarzAA 120 µg/kg SC (Stage 7), MarzAA 2×60 µg/kg SC (Stage 8), MarzAA 3x60 µg/kg SC (Stage 9)

Treatment:

Biological: MarzAA (marzeptacog alfa [activated])