A Phase 1/2 interventional study of Coagulation Factor VIIa variant in Factor VII Deficiency, Glanzmann Thrombasthenia and Hemophilia A With Inhibitor, sponsored by Catalyst Biosciences. Terminated at 20 sites in 5 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2021-12-21.
Sponsored by Catalyst Biosciences · Phase 1/2, Interventional, and Treatment
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.
503 studies on the registry are indexed under Hemostatic Disorders; 71 are open to participants now.
This study's enrollment of 19 is below the median of 51 across 253 interventional studies indexed under Hemostatic Disorders.
Browse Hemostatic Disorders studies →Catalyst Biosciences is the lead sponsor of 8 studies on the registry; none are open to participants now.
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Exclusion Criteria:
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.
Biological: Coagulation Factor VIIa variant
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.
Biological: Coagulation Factor VIIa variant
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.
Biological: Coagulation Factor VIIa variant
Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes
Also known as: MarzAA
Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.
Time frame: Dosing period for each stage in a cohort will be approximately 5 to 11 days
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
Time frame: 24 hours after the first administration of study drug
Plan to share: No
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This study is terminated, as verified in Dec 2021. You cannot join it, but the record below documents what was studied.
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Catalyst Biosciences