Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding… (NCT04548791) | Clinical Trial Compass
TerminatedPhase 1/2
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
Stopped: Company decision (not a safety issue)
United States, India, Italy19 participantsStarted 2021-05-17
Plain-language 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.
Who can participate
Age range
12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
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
2
Bleeding episode treatment success
Timeframe: 24 hours after the first administration of study drug