A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

CSL Behring63 enrolled

Overview

This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemophilia B

Interventions

rIX-FPBIOLOGICAL

Recombinant IX-FP (rIX-FP) is a fusion protein linking coagulation factor IX with albumin, and will be administered by intravenous administration

Eligibility

Sex: MALEMin age: 12 YearsMax age: 65 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male subjects, 12 to 65 years old * Severe hemophilia B (FIX activity of ≤ 2%) * Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for \> 150 exposure days (EDs) * No history of FIX inhibitor formation, no detectable inhibitors at Screening and no family history of inhibitors against FIX * Written informed consent for study participation * On-demand subjects only, who have experienced a minimum average of 2 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months Exclusion Criteria: * Known hypersensitivity to any FIX product or hamster protein * Known congenital or acquired coagulation disorder other than congenital FIX deficiency * HIV positive subjects with a CD4 count \< 200/mm3 * Low platelet count, kidney or liver dysfunction * Recent life-threatening bleeding episode

Locations (30)

Outcomes

Primary Outcomes

Change in Frequency of Spontaneous Bleeding Events Between On-demand and Prophylaxis Treatments (Annualized)

Subjects in the on-demand arm received on-demand dosing with rIX-FP for up to 26 weeks (on-demand regimen), and then received weekly prophylaxis with rIX-FP for the remainder of the study (prophylaxis regimen). The effectiveness of prophylaxis in comparison to on-demand therapy was investigated by comparing the same subject's annualized spontaneous bleeding rate (AsBR) during the on-demand regimen and during the prophylaxis regimen.

Time frame: Up to 26 weeks for on-demand regimen, and between 1 and 17 months for prophylaxis regimen.

Number of Subjects Developing Inhibitors Against Factor IX (FIX)

The number of participants developing inhibitors against factor IX (FIX) along with the 95% Clopper-Pearson confidence interval, are summarized for subjects with 50 or more exposure days (EDs) to rIX-FP, and for all participants in the study.

Time frame: Up to 27.7 months (maximum)

Secondary Outcomes

The Frequency of Related Adverse Events

The percentage of participants experiencing treatment-related adverse-events (TEAEs).

Time frame: For the duration of the study; median 20.27 months.

Number of Subjects Developing Antibodies Against rIX-FP

Time frame: For the duration of the study; median 20.27 months.

Proportion of Bleeding Episodes Requiring One or ≤ Two Injections of rIX-FP to Achieve Hemostasis

Number of injections required to achieve hemostasis expressed as a percentage of the bleeding episodes requiring treatment.

Time frame: For the duration of the study; median 20.27 months.

Investigator's Overall Clinical Assessment of Hemostatic Efficacy for Treatment of Bleeding Episodes, Based on a Four Point Ordinal Scales (Excellent, Good, Moderate, Poor/No Response)

Data from ClinicalTrials.gov

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Rush University Medical Center

Chicago, Illinois, United States

Indiana Hemophilia and Thrombosis Center, Inc.

Indianapolis, Indiana, United States

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

BloodCenter of Wisconsin

Milwaukee, Wisconsin, United States

AKH Wien [Hämatologie, Hämostaseol

Vienna, Austria

SHAT "Joan Pavel" OOD [Hemorrhagic Diathesis and Anemia]

Sofia, Bulgaria

Centre Hospitalier Universitaire de Brest/CHU Morvan

Brest, France

C.R.T.H. Hôp. Bicêtre-Hémophilie

Le Kremlin-Bicêtre, France

CHU de Lyon - Hôpital Edouard Herriot [Hemophilie]

Lyon, France

Hôpital Necker-CRTH

Paris, France

...and 20 more locations

Number of bleeding episodes requiring treatment that resulted in hemostatic efficacy of excellent, good, moderate, poor/no response, according to the Investigator's clinical assessment of hemostatic efficacy, expressed as a percentage of the bleeding episodes requiring treatment.

Time frame: For the duration of the study; median 20.27 months

rIX-FP Consumed Per Month While Maintaining Assigned Prophylactic Treatment Interval During Routine Prophylaxis.

Time frame: For Prophylaxis Arm 7-, 10- and 14-day regimens, median 269, 240 and 386 days respectively. For On-demand Arm, prophylaxis regimen, median 316 days.

Time frame: Median 269, 240, 386 and 316 days, respectively (see Description)

Incremental Recovery of rIX-FP

Pharmacokinetic (PK) data are presented for a single 50 IU/kg dose of rIX-FP.

Time frame: 336 hours

Half-life (t1/2) of a Single Dose of rIX-FP

PK data are presented for a single 50 IU/kg dose of rIX-FP.

Time frame: 336 hours

Area Under the Curve (AUC)

AUC to the last sample with quantifiable drug concentration (AUClast) of a single dose of rIX-FP. PK data are presented for a single 50 IU/kg dose of rIX-FP.

Time frame: 336 hours

Clearance of a Single Dose of rIX-FP

PK data are presented for a single 50 IU/kg dose of rIX-FP.

Time frame: 336 hours

Investigator's (or Surgeon's) Overall Clinical Assessment of Hemostatic Efficacy for Surgical Prophylaxis, Based on a Four Point Ordinal Scale (Excellent, Good, Moderate, Poor/No Response)

Number of surgical events treated prophylactically with rIX-FP that resulted in hemostatic efficacy of excellent, good, moderate, poor/no response, according to the Investigator's (surgeon's) overall assessment of hemostatic efficacy for surgical prophylaxis.

Time frame: Up to 14 days after surgery

Annualized Spontaneous Bleeding Events Compared Between 7 Day Prophylactic and Extended Regimens

Median number of spontaneous bleeds per year per subject comparing 7-, 10- and 14- day prophylactic regimens.

Time frame: During treatment, between median 240 and 386 days per subject.