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

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

11 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-18

Study Completion Date

2019-04-13

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Hemophilia A With Inhibitor Hemophilia B With Inhibitor

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Part 1a

Coagulation Factor VIIa variant, 18 µg/kg by intravenous route

Group Type EXPERIMENTAL

Coagulation Factor VIIa variant

Intervention Type BIOLOGICAL

Single intravenous injection of MarzAA, followed by single subcutaneous injection of MarzAA, followed by daily subcutaneous injection of MarzAA for 50 days at final dose level required.

Part 1b

Coagulation Factor VIIa variant, 30 µg/kg by subcutaneous route

Group Type EXPERIMENTAL

Coagulation Factor VIIa variant

Intervention Type BIOLOGICAL

Single intravenous injection of MarzAA, followed by single subcutaneous injection of MarzAA, followed by daily subcutaneous injection of MarzAA for 50 days at final dose level required.

Part 2

Coagulation Factor VIIa variant, 30, 60, 90, 120 µg/kg by subcutaneous route

Group Type EXPERIMENTAL

Coagulation Factor VIIa variant

Intervention Type BIOLOGICAL

Single intravenous injection of MarzAA, followed by single subcutaneous injection of MarzAA, followed by daily subcutaneous injection of MarzAA for 50 days at final dose level required.

Interventions

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Coagulation Factor VIIa variant

Single intravenous injection of MarzAA, followed by single subcutaneous injection of MarzAA, followed by daily subcutaneous injection of MarzAA for 50 days at final dose level required.

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No