Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders

Hemophilia A With Inhibitor Clinical Trial

Official title:

Phase 1/2 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Marzeptacog Alfa (Activated) in Treatment of Episodic Bleeding in Subjects With Inherited Bleeding Disorders

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04548791
• Other study ID #: MAA-202
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: May 17, 2021
• Est. completion date: December 3, 2021

Study information

• Verified date: December 2021
• Source: Catalyst Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 19
• Est. completion date: December 3, 2021
• Est. primary completion date: November 15, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Diagnosis of cohort: FVII deficiency, Glanzmann Thrombasthenia, or hemophilia A 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

• Agreement to use highly effective birth control throughout the study if the subject has childbearing potential

Exclusion Criteria:

• Genotype of FVIID subjects with identified mutations by central lab at screening

• 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, whichever is longer

• Known hypersensitivity to trial or related product

• Known positive antibody to FVII or FVIIa detected by central lab at screening

• Be immunosuppressed

• Significant contraindication to participate

Related Conditions & MeSH terms

• Blood Coagulation Disorders
• Factor VII Deficiency
• Glanzmann Thrombasthenia
• Hemophilia A
• Hemophilia A With Inhibitor
• Hemostatic Disorders

Intervention

Biological:
• Coagulation Factor VIIa variant
Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes

Locations

Country	Name	City	State
India	Mazumdar Shaw Medical Centre	Bengaluru
India	St. John's Medical College Hospital	Bengaluru
India	Amrita Institute of Medical Sciences and Research Centre	Kochi
India	K. J. Somaiya Hospital and Research Centre	Mumbai
India	Sahyadri Super Speciality Hospital	Pune
Italy	Careggi University Hospital	Florence
Italy	Center for Thrombosis and Haemorrhagic Diseases	Milan
Italy	Maggiore Polyclinic Hospital, IRCCS Ca' Granda	Milan
Italy	Children's Hospital BambiNo Gesù, IRCCS (PEDS)	Roma
Italy	City of Health and Science of Turin	Turin
Russian Federation	Territorial Clinical Hospital	Barnaul
Russian Federation	National Medical Hematology Research Center under the Ministry of Healthcare of the Russian Federation	Moscow
Ukraine	Institute of Blood Pathology and Transfusion Medicine, Department of Surgery and Clinical Transfusiology	Lviv
United States	University of Colorado Hemophilia and Thrombosis Center	Aurora	Colorado
United States	Rush University	Chicago	Illinois
United States	Children's Hospital of Michigan	Detroit	Michigan
United States	Michigan State University Center for Bleeding Disorders & Clotting Disorders	East Lansing	Michigan
United States	East Carolina University	Greenville	North Carolina
United States	UC Davis Medical Center	Sacramento	California
United States	University of California -San Francisco	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Catalyst Biosciences

Countries where clinical trial is conducted

United States,  India,  Italy,  Russian Federation,  Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose	Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.	Dosing period for each stage in a cohort will be approximately 5 to 11 days
Primary	Bleeding episode treatment success	Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment	24 hours after the first administration of study drug