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

Phase 3

Terminated

• Conditions: Hemophilia B With Inhibitor; Hemophilia A With Inhibitor
• Interventions: Biological: MarzAA

• Registration Number: NCT04489537
• Lead Sponsor: Catalyst Biosciences

Brief 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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-07-24
• Study First Submitted QC: 2020-07-24
• Study First Posted: 2020-07-28
• Study Start: 2021-05-04
• Primary Completion: 2021-11-15
• Status Verified: 2021-12
• Study Completion: 2021-12-01
• Last Update Submitted: 2021-12-02
• Last Update Posted: 2021-12-17

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 18

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
MarzAA	MarzAA	Coagulation Factor VIIa variant, 60 µg/kg by subcutaneous route, administered on-demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis
Standard of Care	MarzAA	Standard of care administered on-demand during bleeding episodes

Primary Outcome Measures

Name	Time	Method
Bleeding episode treatment success	24 hours after the first administration of study drug	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.

Trial Locations

Locations (43)

"Institute of Blood Pathology and Transfusion Medicine of National Academy of Medical Sciences of Ukraine", Haematology Department; 🇺🇦 Lviv, Ukraine

Queen Elizabeth Hospital Birmingham; 🇬🇧 Birmingham, United Kingdom

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

Lombardi Comprehensive Cancer Center; 🇺🇸 Washington, District of Columbia, United States

JSC "K.Eristavi National Center of Experimental and Clinical Surgery"; 🇬🇪 Tbilisi, Georgia

All India Institute of Medical Sciences, Rishikesh; 🇮🇳 Rishīkesh, India

Hospital Ampang; 🇲🇾 Ampang, Malaysia

Raja Perempuan Zainab II Hospital; 🇲🇾 Kota Bharu, Malaysia

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, Mexico

Haemophilia Comprehensive Care Centre; 🇿🇦 Johannesburg, South Africa

Hospital Universitario Vall d'Hebron; 🇪🇸 Barcelona, Spain

Hospital de Malaga; 🇪🇸 Málaga, Spain

Changhua Christian Hospital (CCH); 🇨🇳 Changhua City, Taiwan

Akdeniz University Medical Faculty Hospital; 🇹🇷 Antalya, Turkey

Taichung Veterans General Hospital; 🇨🇳 Taichung, Taiwan

Istanbul University Faculty of Medicine; 🇹🇷 Istanbul, Turkey

Ege University Medical Faculty; 🇹🇷 İzmir, Turkey

"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

University Hospital Southampton NHS Foundation Trust; 🇬🇧 Southampton, United Kingdom

Kirov Research Institute of Hematology and Blood Transfusion; 🇷🇺 Kirov, Russian Federation

Sanjay Gandhi Postgraduate Institute of Medical Sciences; 🇮🇳 Lucknow, India

Queen Elizabeth Hospital Malaysia; 🇲🇾 Kota Kinabalu, Malaysia

Hospital Tunku Azizah Kuala Lumpur; 🇲🇾 Kuala Lumpur, Malaysia

Hematology Center after Prof. R. Yeolyan; 🇦🇲 Yerevan, Armenia

MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre; 🇮🇳 Mumbai, India

Tuanku Ja'afar Hospital, Seremban; 🇲🇾 Sungai Petani, Malaysia

Magyar Honvédség Egészségügyi Központ, Országos Haemophilia Központ; 🇭🇺 Budapest, Hungary

K.L.E.S Dr. Prabhakar Kore Hospital and Medical Research Centre; 🇮🇳 Karnad, India

Sahyadri Specialty Hospital; 🇮🇳 Mumbai, India

Medis, Llc; 🇷🇺 Nizhny Novgorod, Russian Federation

Korczowski Bartosz, Gabinet Lekarski; 🇵🇱 Rzeszów, Poland

"Cherkasy Regional Oncological Dispensary of Cherkasy Regional Council", Regional Treatment and Diagnostic Center of Hematology; 🇺🇦 Cherkasy, Ukraine

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, Italy

Nirmal Hospital; 🇮🇳 Gujrat, India

K.J Somaiya Hospital and Research Centre; 🇮🇳 Mumbai, India

Grant Medical Foundation, Ruby Hall Clinic; 🇮🇳 Pune, India

Özel Acibadem Adana Hastanesi; 🇹🇷 Seyhan, Turkey

Royal Liverpool University Hospital; 🇬🇧 Liverpool, United Kingdom