Clinical trial NCT04548791: Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Factor VII Deficiency
Glanzmann Thrombasthenia
Hemophilia A With Inhibitor
Lead sponsor: Catalyst Biosciences
Sponsor class: INDUSTRY
First posted: 2020-09-16
Enrollment: 19
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
timeFrame: Dosing period for each stage in a cohort will be approximately 5 to 11 days
measure: Bleeding episode treatment success
timeFrame: 24 hours after the first administration of study drug
Secondary outcomes:
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: San Francisco
country: United States
facility: University of California -San Francisco
state: California
city: Aurora
country: United States
facility: University of Colorado Hemophilia and Thrombosis Center
state: Colorado
city: Chicago
country: United States
facility: Rush University
state: Illinois
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: East Lansing
country: United States
facility: Michigan State University Center for Bleeding Disorders & Clotting Disorders
state: Michigan
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Bengaluru
country: India
facility: Mazumdar Shaw Medical Centre
city: Bengaluru
country: India
facility: St. John's Medical College Hospital
city: Kochi
country: India
facility: Amrita Institute of Medical Sciences and Research Centre
city: Mumbai
country: India
facility: K. J. Somaiya Hospital and Research Centre
city: Pune
country: India
facility: Sahyadri Super Speciality Hospital
city: Florence
country: Italy
facility: Careggi University Hospital
city: Milan
country: Italy
facility: Center for Thrombosis and Haemorrhagic Diseases
city: Milan
country: Italy
facility: Maggiore Polyclinic Hospital, IRCCS Ca' Granda
city: Roma
country: Italy
facility: Children's Hospital BambiNo Gesù, IRCCS (PEDS)
city: Turin
country: Italy
facility: City of Health and Science of Turin
city: Barnaul
country: Russian Federation
facility: Territorial Clinical Hospital
city: Moscow
country: Russian Federation
facility: National Medical Hematology Research Center under the Ministry of Healthcare of the Russian Federation
city: Lviv
country: Ukraine
facility: Institute of Blood Pathology and Transfusion Medicine, Department of Surgery and Clinical Transfusiology
Study officials:
References: