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

Hemophilia A With Inhibitor Clinical Trial

Official title:

Phase 3 Study to Evaluate the Efficacy and Safety of Subcutaneous Marzeptacog Alfa (Activated) For On Demand Treatment and Control of Bleeding Episodes in Subjects With Hemophilia A or Hemophilia B, With Inhibitors: The Crimson 1 Study

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04489537
• Other study ID #: MAA-304
• Status: Terminated
• Phase: Phase 3
• Start date: May 4, 2021
• Est. completion date: December 1, 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 safety and efficacy of MarzAA for on-demand treatment and control of bleeding episodes in hemophilia A or B patients with inhibitors compared with their standard of care (SOC).

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 18
• Est. completion date: December 1, 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 congenital hemophilia A or B 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

Exclusion Criteria:

• 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
• Known hypersensitivity to trial or related product
• Known positive antibody to FVII or FVIIa detected by central lab at screening
• Have a coagulation disorder other than hemophilia A or B
• Be immunosuppressed
• Significant contraindication to participate

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia A With Inhibitor
• Hemophilia B
• Hemophilia B With Inhibitor

Intervention

Biological:
• MarzAA
A cross over design to assess the efficacy of a dosing regimen of 60 µg/kg of MarzAA compared with standard of care for the treatment of bleeding episodes.

Locations

Country	Name	City	State
Armenia	Hematology Center after Prof. R. Yeolyan	Yerevan
Georgia	JSC "K.Eristavi National Center of Experimental and Clinical Surgery"	Tbilisi
Hungary	Magyar Honvédség Egészségügyi Központ, Országos Haemophilia Központ	Budapest
India	Nirmal Hospital	Gujrat
India	K.L.E.S Dr. Prabhakar Kore Hospital and Medical Research Centre	Karnad
India	Sanjay Gandhi Postgraduate Institute of Medical Sciences	Lucknow
India	K.J Somaiya Hospital and Research Centre	Mumbai
India	MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre	Mumbai
India	Sahyadri Specialty Hospital	Mumbai
India	Grant Medical Foundation, Ruby Hall Clinic	Pune
India	All India Institute of Medical Sciences, Rishikesh	Rishikesh
Italy	Azienda Ospedaliero-Universitaria Careggi	Firenze
Italy	Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano	Milan
Italy	Azienda Ospedaliera Universitaria Federico II	Napoli
Italy	Citta della Salute e della Scienza di Torino - Ospedale le Molinette	Torino
Malaysia	Hospital Ampang	Ampang
Malaysia	Raja Perempuan Zainab II Hospital	Kota Bharu
Malaysia	Queen Elizabeth Hospital Malaysia	Kota Kinabalu
Malaysia	Hospital Tunku Azizah Kuala Lumpur	Kuala Lumpur
Malaysia	Tuanku Ja'afar Hospital, Seremban	Sungai Petani
Mexico	Instituto Nacional de Pediatria	Ciudad de mexico
Mexico	Instituto de Investigaciones Aplicada a la Neurociencia A.C.	Durango
Mexico	Investigación e Innovación en Medicina Traslacional S.A.P.I. de C.V.	Mexico City
Poland	Korczowski Bartosz, Gabinet Lekarski	Rzeszów
Russian Federation	Kirov Research Institute of Hematology and Blood Transfusion	Kirov
Russian Federation	Medis, Llc	Nizhny Novgorod
South Africa	Haemophilia Comprehensive Care Centre	Johannesburg
Spain	Hospital Universitario Vall d'Hebron	Barcelona
Spain	Hospital de Malaga	Málaga
Taiwan	Changhua Christian Hospital (CCH)	Changhua City
Taiwan	Taichung Veterans General Hospital	Taichung
Turkey	Akdeniz University Medical Faculty Hospital	Antalya
Turkey	Istanbul University Faculty of Medicine	Istanbul
Turkey	Ege University Medical Faculty	Izmir
Turkey	Özel Acibadem Adana Hastanesi	Seyhan
Ukraine	"Cherkasy Regional Oncological Dispensary of Cherkasy Regional Council", Regional Treatment and Diagnostic Center of Hematology	Cherkasy
Ukraine	"Kyiv City Clinical Hospital #9" of Executive Body of Kyiv City Council (Kyiv City State Administration), City Scientific-Practical Centre of Diagnostics and Treatment	Kyiv
Ukraine	"Institute of Blood Pathology and Transfusion Medicine of National Academy of Medical Sciences of Ukraine", Haematology Department	Lviv
United Kingdom	Queen Elizabeth Hospital Birmingham	Birmingham
United Kingdom	Royal Liverpool University Hospital	Liverpool
United Kingdom	University Hospital Southampton NHS Foundation Trust	Southampton
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Lombardi Comprehensive Cancer Center	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Catalyst Biosciences

Countries where clinical trial is conducted

United States,  Armenia,  Georgia,  Hungary,  India,  Italy,  Malaysia,  Mexico,  Poland,  Russian Federation,  South Africa,  Spain,  Taiwan,  Turkey,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
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 compared with standard of care.	24 hours after the first administration of study drug