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

CSL Behring

Status and phase: Completed; Phase 3; Phase 2

Conditions: Hemophilia B

Treatments: Biological: rIX-FP

Study type: Interventional

Funder types: Industry

Identifiers

NCT01496274

CSL654_3001

2011-002415-28 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 63 patients

Sex: Male

Ages: 12 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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63 participants in 2 patient groups

Prophylaxis

Experimental group

Description:

Routine weekly prophylaxis and episodic treatment for bleeding episodes. An individualized dosing interval may be tested in sub-group subjects during the 2nd part of the trial. Subjects may participate in a surgical 'sub-study' in which rIX-FP may be administered prior to, during and after surgical intervention.

Treatment:

Biological: rIX-FP

On-demand

Experimental group

Description:

Episodic treatment for bleeding episodes during the first 6 months then switch to routine weekly prophylaxis for a further 6 months Subjects may participate in a surgical 'sub-study' in which rIX-FP may be administered prior to, during and after surgical intervention.

Treatment:

Biological: rIX-FP