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

• ClinicalTrials.gov Identifier: NCT04548791
• Recruitment Status: Recruiting
• First Posted: September 16, 2020
• Last Update Posted: June 17, 2021
• Sponsor: Catalyst Biosciences
• Information provided by (Responsible Party): Catalyst Biosciences

Tracking Information

• First Submitted Date: September 8, 2020
• First Posted Date: September 16, 2020
• Last Update Posted Date: June 17, 2021
• Actual Study Start Date: May 17, 2021
• Estimated Primary Completion Date: December 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 8, 2020)

• 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

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
• Official Title: 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: Non-Randomized; Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Factor VII Deficiency
• Glanzmann Thrombasthenia
• Hemophilia A With Inhibitor

Intervention

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

Other Name: MarzAA

Study Arms

• Experimental: Cohort 1
  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.
  Intervention: Biological: Coagulation Factor VIIa variant
• Experimental: Cohort 2
  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.
  Intervention: Biological: Coagulation Factor VIIa variant
• Experimental: Cohort 3
  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.
  Intervention: Biological: Coagulation Factor VIIa variant

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: September 8, 2020): 24
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: March 2022
• Estimated Primary Completion Date: December 2021 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Howard Levy, MD, PhD, MMM; +1.650.266.8671; hlevy@catbio.com

Contact: Linda Neuman, MD; +1.650.266.8667; lneuman@catbio.com

• Listed Location Countries: India, Italy, Russian Federation, Ukraine, United States

Administrative Information

• NCT Number: NCT04548791
• Other Study ID Numbers: MAA-202
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Catalyst Biosciences
• Study Sponsor: Catalyst Biosciences
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Catalyst Biosciences
• Verification Date: June 2021