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

NCT ID: NCT04072237

Last Updated: 2021-09-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-09-24
• Study Completion Date: 2020-06-17

Brief Summary

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.

Detailed 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).

Conditions

Hemophilia A; Hemophilia B; Hemophilia A With Inhibitor; Hemophilia B With Inhibitor; Hemophilia A Without Inhibitor; Hemophilia B Without Inhibitor

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: DIAGNOSTIC
• Blinding Strategy: NONE

Study Groups

Study Population

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)

Group Type: EXPERIMENTAL

MarzAA (marzeptacog alfa [activated])

Intervention Type: BIOLOGICAL

Single intravenous dose and ascending doses of subcutaneous injection of MarzAA (Coagulation Faction VIIa Variant)

Interventions

MarzAA (marzeptacog alfa [activated])

Single intravenous dose and ascending doses of subcutaneous injection of MarzAA (Coagulation Faction VIIa Variant)

Intervention Type: BIOLOGICAL

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Catalyst Biosciences

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Howard Levy, MD, PhD, MMM

Role: STUDY_DIRECTOR

Sponsor GmbH

Locations

Medical Center "Hippocrates - N"

Plovdiv, , Bulgaria

Specialized Hospital for Active Treatment of Hematological Diseases

Sofia, , Bulgaria

Kirov Research Institute of Hematology and Blood Transfusion

Kirov, , Russia

National Medical Hematology Research Center

Moscow, , Russia

Municipal Policlinic # 37, City Center for Hemophilia Treatment

Saint Petersburg, , Russia

Countries

Bulgaria; Russia

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

MAA-102

Identifier Type: -

Identifier Source: org_study_id