Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-07-23

Study Completion Date

2018-03-28

Brief Summary

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The purpose of this study is to investigate the pharmacokinetics (PK), efficacy, and safety of rVIIa-FP (CSL689). The study will enroll approximately 54 male subjects, 12 to 65 years of age, with hemophilia types A or B who have developed inhibitors to FVIII or FIX. The study consists of 3 sequential parts (Parts 1, 2, 3): The purpose of Part 1 (PK part) is to evaluate the PK of a single treatment of CSL689 (low dose or high dose) and compare with the PK of a single treatment of Eptacog alfa (low dose or high dose). In Part 1, CSL689 and Eptacog alfa will be given by the doctor at the study center. The purpose of Part 2 (Dose-evaluation part) is to identify which of the 2 tested dose levels of CSL689 shows the best efficacy and safety in stopping acute bleeding events (this dose will be called the "population best dose"). The purpose of the final Part 3 (Repeated-dose part) is to confirm the efficacy and safety of the "population best dose" identified in Part 2. In Parts 2 and 3, subjects will self-administer a specified number of CSL689 infusions at home on-demand (ie, when a bleeding event occurs), will keep an electronic diary, and will visit the center at monthly intervals. This study is expected to last for up to 16 months for the subjects participating in all 3 parts, and up to 9 months for the subjects participating in Part 3 only.

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Detailed Description

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Conditions

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Hemophilia A With Inhibitors Hemophilia B With Inhibitors

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CSL689 low-dose

* Part 1: single injection of low-dose CSL689 for PK evaluation
* Part 2: up to 2 injections of low-dose CSL689 per bleeding event (bleeding events 1 to 3\*)
* Part 3: up to 3 injections of low-dose CSL689 per bleeding event \* Note: All subjects in the low-dose arm will be treated with high-dose CSL689 for bleeding events 4-6 in Part 2

Group Type EXPERIMENTAL

CSL689

Intervention Type DRUG

Recombinant fusion protein, linking activated coagulation factor VII with albumin. Two dose levels (low dose, high dose) will be studied in Parts 1, 2, and 3.

CSL689 high-dose

* Part 1: single injection of high-dose CSL689 for PK evaluation
* Part 2: up to 2 injections of high-dose CSL689 per bleeding event (bleeding events 4 to 6\*)
* Part 3: up to 3 injections of high-dose CSL689 per bleeding event

* Note: All subjects in the high-dose arm will be treated with low-dose CSL689 for bleeding events 1-3 in Part 2

Group Type EXPERIMENTAL

CSL689

Intervention Type DRUG

Recombinant fusion protein, linking activated coagulation factor VII with albumin. Two dose levels (low dose, high dose) will be studied in Parts 1, 2, and 3.

Eptacog alfa low-dose

Single injection of low-dose Eptacog alfa in Part 1 for PK evaluation

Group Type ACTIVE_COMPARATOR

Eptacog alfa (activated)

Intervention Type DRUG

Recombinant activated coagulation factor VII. Two dose levels (low dose, high dose) will be studied in Part 1.

Eptacog alfa high-dose

Single injection of high-dose Eptacog alfa in Part 1 for PK evaluation

Group Type ACTIVE_COMPARATOR

Eptacog alfa (activated)

Intervention Type DRUG

Recombinant activated coagulation factor VII. Two dose levels (low dose, high dose) will be studied in Part 1.

Interventions

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CSL689

Recombinant fusion protein, linking activated coagulation factor VII with albumin. Two dose levels (low dose, high dose) will be studied in Parts 1, 2, and 3.

Intervention Type DRUG

Eptacog alfa (activated)

Recombinant activated coagulation factor VII. Two dose levels (low dose, high dose) will be studied in Part 1.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Male subjects with hemophilia A or B and inhibitors.
* Age ≥ 12 and ≤ 65 years.
* High responding inhibitor with documented historical inhibitor titer \> 5 Bethesda Units/mL.

Exclusion Criteria

* Congenital or acquired coagulation disorders other than hemophilia A or B.
* Ongoing immune tolerance induction therapy or planned during study.
* Known or suspected hypersensitivity to activated recombinant human FVII or to any excipient of CSL689.
* Body mass index \> 30 kg/m².
* Major surgery within 28 days before screening or scheduled major and / or orthopedic surgery during the study.
* Advanced atherosclerotic disease (ie, known history of ischemic heart disease, or ischemic stroke).
* Any clinical signs or known history of thromboembolic events, including known deep vein thrombosis.
* Human immunodeficiency virus (HIV)-positive subjects who have low cluster of differentiation 4 (CD4)+ lymphocyte count (200/μL or less) at screening.
* Use of the following within the screening period or planned during study: a) plasma or coagulation factor concentrates other than rescue therapy or therapy during Part 1, b) other platelet inhibitors, c) desmopressin, and d) fibrinolysis inhibitors, except if used as local treatment (eg, for oral bleeds).

Minimum Eligible Age

12 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No