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Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B

C

Catalyst Biosciences

Status and phase

Completed
Phase 2

Conditions

Hemophilia B With Inhibitor
Hemophilia A With Inhibitor

Treatments

Biological: Coagulation Factor VIIa variant

Study type

Interventional

Funder types

Industry

Identifiers

NCT03407651
MAA-201

Details and patient eligibility

About

Phase 2, multi-center, open-label study designed to evaluate the PK, bioavailability, PD, efficacy and safety of a daily subcutaneous [SC] treatment regimen with MarzAA for bleeding prophylaxis in 12 adult subjects with hemophilia A or B with an inhibitor and history of frequent spontaneous bleeding episodes.

Full description

Multi-center, open-label Phase 2 study to evaluate the PK, bioavailability, PD, efficacy and safety of a daily SC treatment regimen with MarzAA for bleeding prophylaxis in adult subjects with hemophilia A or B with an inhibitor. The study will enroll and dose, both intravenously and subcutaneously, a total of 12 adult male subjects with severe congenital hemophilia A or B with an inhibitor, and history of frequent bleeding episodes during the 6 months prior to enrollment, as per the individual's bleeding and treatment records.

Once a subject is enrolled into the trial, the study will be conducted in three parts (occurring consecutively):

Part 1a (24 hours): Single IV administration of MarzAA; Part 1b (48 hours): Single SC administration of MarzAA; Part 2: Daily SC administration. Dose escalation in Part 2 will occur if breakthrough bleeding occurs. Subjects are treated for 50 days at the final dose level required.

Enrollment

11 patients

Sex

Male

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Severe congenital hemophilia A or B with an inhibitor.
  • History of frequent spontaneous bleeding episodes.
  • Male, age 18 or older.
  • Affirmation of informed consent with signature confirmation before any trial-related activities.

Exclusion criteria

  • Receiving prophylaxis treatment.
  • Previous participation in a clinical trial evaluating a modified rFVIIa agent.
  • Known positive antibody to FVII or FVIIa detected by central laboratory at screening.
  • Have a coagulation disorder other than hemophilia A or B.
  • Significant contraindication to participation.

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

11 participants in 3 patient groups

Part 1a
Experimental group
Description:
Coagulation Factor VIIa variant, 18 µg/kg by intravenous route
Treatment:
Biological: Coagulation Factor VIIa variant
Part 1b
Experimental group
Description:
Coagulation Factor VIIa variant, 30 µg/kg by subcutaneous route
Treatment:
Biological: Coagulation Factor VIIa variant
Part 2
Experimental group
Description:
Coagulation Factor VIIa variant, 30, 60, 90, 120 µg/kg by subcutaneous route
Treatment:
Biological: Coagulation Factor VIIa variant

Trial documents
2

Trial contacts and locations

9

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Data sourced from clinicaltrials.gov

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