Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders

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

Sponsors

Lead Sponsor: Catalyst Biosciences

Source Catalyst Biosciences
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.

Overall Status Not yet recruiting
Start Date December 2020
Completion Date March 2022
Primary Completion Date December 2021
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose Dosing period for each stage in a cohort will be approximately 5 to 11 days
Bleeding episode treatment success 24 hours after the first administration of study drug
Enrollment 24
Condition
Intervention

Intervention Type: Biological

Intervention Name: Coagulation Factor VIIa variant

Description: Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes

Other Name: MarzAA

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

Gender: All

Minimum Age: 12 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Contact

Last Name: Howard Levy, MD, PhD, MMM

Phone: +1.650.266.8671

Email: [email protected]

Verification Date

September 2020

Responsible Party

Type: Sponsor

Condition Browse
Number Of Arms 3
Arm Group

Label: Cohort 1

Type: Experimental

Description: 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.

Label: Cohort 2

Type: Experimental

Description: 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.

Label: Cohort 3

Type: Experimental

Description: 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.

Patient Data No
Study Design Info

Allocation: Non-Randomized

Intervention Model: Sequential Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov