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

NCT04072237 | Completed Phase 1 Results FDA Drug

• 1 other identifier: MAA-102
• Study Type: interventional
• Target: 11
• Locations: 2 countries
• Sites: 5

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.

Conditions

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

Outcome Measures

Primary Outcomes (2)

• Comparative MarzAA Activity by Dose Level/Stage - AUC0-∞ and AUC0-last

  Comparative pharmacokinetics (PK) by dose level/stage based on examination of AUC frequencies of these for each of the dose groups

  Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.

• Comparative MarzAA Activity by Dose Level/Stage - AUCT1-T2 Normalized by Dose = AUC0-last/Dose

  Comparative pharmacokinetics by dose level/stage based on examination of AUC frequency of these for each of the dose groups

  Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.

Secondary Outcomes (21)

• Comparative MarzAA Activity of Intravenous and Subcutaneous - Cmax

  Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.

• Comparative MarzAA Activity of Intravenous and Subcutaneous - Tmax

  Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.

• Comparative MarzAA Activity of Intravenous and Subcutaneous - T1/2eqα

  Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.

• Comparative MarzAA Activity of Intravenous and Subcutaneous - T1/2λ-z

  Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.

• Comparative MarzAA Activity of Intravenous and Subcutaneous - CL

  Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.

Study Arms (1)

Study Population

EXPERIMENTAL

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)

Biological: MarzAA (marzeptacog alfa [activated])

Interventions

MarzAA (marzeptacog alfa [activated]) BIOLOGICAL

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

Study Population

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Catalyst Biosciences: lead

Study Sites (5)

Medical Center "Hippocrates - N"

Plovdiv, Bulgaria

Location

Specialized Hospital for Active Treatment of Hematological Diseases

Sofia, Bulgaria

Location

Kirov Research Institute of Hematology and Blood Transfusion

Kirov, Russia

Location

National Medical Hematology Research Center

Moscow, Russia

Location

Municipal Policlinic # 37, City Center for Hemophilia Treatment

Saint Petersburg, Russia

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Interventions

marzeptacog alfa (activated)

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Results Point of Contact

• Title: Howard Levy, Chief Medical Officer
• Organization: Catalyst Biosciences

hlevy@catbio.com

+1.650.266.6871

Study Officials

• Howard Levy, MD, PhD, MMM

  Sponsor GmbH

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2019
• First Posted: August 28, 2019
• Study Start: September 24, 2019
• Primary Completion: April 30, 2020
• Study Completion: June 17, 2020
• Last Updated: September 13, 2021
• Results First Posted: July 16, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

RU (3); BU (2)