Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor
About this trial
This is an interventional treatment trial for Factor VII Deficiency
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
Sites / Locations
- UC Davis Medical Center
- University of California -San Francisco
- University of Colorado Hemophilia and Thrombosis Center
- Rush University
- Children's Hospital of Michigan
- Michigan State University Center for Bleeding Disorders & Clotting Disorders
- East Carolina University
- Mazumdar Shaw Medical Centre
- St. John's Medical College Hospital
- Amrita Institute of Medical Sciences and Research Centre
- K. J. Somaiya Hospital and Research Centre
- Sahyadri Super Speciality Hospital
- Careggi University Hospital
- Center for Thrombosis and Haemorrhagic Diseases
- Maggiore Polyclinic Hospital, IRCCS Ca' Granda
- Children's Hospital BambiNo Gesù, IRCCS (PEDS)
- City of Health and Science of Turin
- Territorial Clinical Hospital
- National Medical Hematology Research Center under the Ministry of Healthcare of the Russian Federation
- Institute of Blood Pathology and Transfusion Medicine, Department of Surgery and Clinical Transfusiology
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
Cohort 1
Cohort 2
Cohort 3
For Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 10 μg/kg, 20 μg/kg, 30 μg/kg, 40 μg/kg, and 60 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 20 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis.
For Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 30 μg/kg, 45 μg/kg, 60 μg/kg, 120 μg/kg, and 180 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 60 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis.
For Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 30 μg/kg, 45 μg/kg, 60 μg/kg, 120 μg/kg, and 180 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 60 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis.