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
Trial Snapshot
- Phase
- Phase 1
- Status
- Terminated
- Sponsor
- Enrollment
- 19
- Locations
- 27
- Primary Endpoint
- Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 12 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Outcomes
Primary Outcomes
Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
Time Frame: Dosing period for each stage in a cohort will be approximately 5 to 11 days
Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.
Bleeding episode treatment success
Time Frame: 24 hours after the first administration of study drug
Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment
Secondary Outcomes
No secondary outcomes reported