Clinical trial NCT04489537: Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Hemophilia A or B
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Hemophilia A With Inhibitor
Hemophilia B With Inhibitor
Lead sponsor: Catalyst Biosciences
Sponsor class: INDUSTRY
First posted: 2020-07-28
Enrollment: 18
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Bleeding episode treatment success
timeFrame: 24 hours after the first administration of study drug
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Washington
country: United States
facility: Lombardi Comprehensive Cancer Center
state: District of Columbia
city: Yerevan
country: Armenia
facility: Hematology Center after Prof. R. Yeolyan
city: Tbilisi
country: Georgia
facility: JSC "K.Eristavi National Center of Experimental and Clinical Surgery"
city: Budapest
country: Hungary
facility: Magyar Honvédség Egészségügyi Központ, Országos Haemophilia Központ
city: Gujrat
country: India
facility: Nirmal Hospital
city: Karnad
country: India
facility: K.L.E.S Dr. Prabhakar Kore Hospital and Medical Research Centre
city: Lucknow
country: India
facility: Sanjay Gandhi Postgraduate Institute of Medical Sciences
city: Mumbai
country: India
facility: K.J Somaiya Hospital and Research Centre
city: Mumbai
country: India
facility: MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre
city: Mumbai
country: India
facility: Sahyadri Specialty Hospital
city: Pune
country: India
facility: Grant Medical Foundation, Ruby Hall Clinic
city: Rishīkesh
country: India
facility: All India Institute of Medical Sciences, Rishikesh
city: Firenze
country: Italy
facility: Azienda Ospedaliero-Universitaria Careggi
city: Milan
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
city: Torino
country: Italy
facility: Citta della Salute e della Scienza di Torino - Ospedale le Molinette
city: Ampang
country: Malaysia
facility: Hospital Ampang
city: Kota Bharu
country: Malaysia
facility: Raja Perempuan Zainab II Hospital
city: Kota Kinabalu
country: Malaysia
facility: Queen Elizabeth Hospital Malaysia
city: Kuala Lumpur
country: Malaysia
facility: Hospital Tunku Azizah Kuala Lumpur
city: Sungai Petani
country: Malaysia
facility: Tuanku Ja'afar Hospital, Seremban
city: Ciudad de mexico
country: Mexico
facility: Instituto Nacional de Pediatria
city: Durango
country: Mexico
facility: Instituto de Investigaciones Aplicada a la Neurociencia A.C.
city: Mexico City
country: Mexico
facility: Investigación e Innovación en Medicina Traslacional S.A.P.I. de C.V.
city: Rzeszów
country: Poland
facility: Korczowski Bartosz, Gabinet Lekarski
city: Kirov
country: Russian Federation
facility: Kirov Research Institute of Hematology and Blood Transfusion
city: Nizhny Novgorod
country: Russian Federation
facility: Medis, Llc
city: Johannesburg
country: South Africa
facility: Haemophilia Comprehensive Care Centre
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron
city: Málaga
country: Spain
facility: Hospital de Malaga
city: Changhua City
country: Taiwan
facility: Changhua Christian Hospital (CCH)
city: Taichung
country: Taiwan
facility: Taichung Veterans General Hospital
city: Antalya
country: Turkey
facility: Akdeniz University Medical Faculty Hospital
city: Istanbul
country: Turkey
facility: Istanbul University Faculty of Medicine
city: İzmir
country: Turkey
facility: Ege University Medical Faculty
city: Seyhan
country: Turkey
facility: Özel Acibadem Adana Hastanesi
city: Cherkasy
country: Ukraine
facility: "Cherkasy Regional Oncological Dispensary of Cherkasy Regional Council", Regional Treatment and Diagnostic Center of Hematology
city: Kyiv
country: Ukraine
facility: "Kyiv City Clinical Hospital #9" of Executive Body of Kyiv City Council (Kyiv City State Administration), City Scientific-Practical Centre of Diagnostics and Treatment
city: Lviv
country: Ukraine
facility: "Institute of Blood Pathology and Transfusion Medicine of National Academy of Medical Sciences of Ukraine", Haematology Department
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital Birmingham
city: Liverpool
country: United Kingdom
facility: Royal Liverpool University Hospital
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
Study officials:
References: