A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B

CSL Behring

Status and phase: Completed; Phase 2; Phase 1

Conditions: Hemophilia B

Treatments: Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein

Study type: Interventional

Funder types: Industry

Identifiers

NCT01361126

CSL654_2004

2010-023793-39 (EudraCT Number)

Details and patient eligibility

About

This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B. The study consists of a screening period, a pharmacokinetic (PK) period, followed by approximately a 5 month treatment period. Subjects will receive weekly routine prophylactic therapy and on-demand treatment for bleeding episodes. In addition, subjects who are not on routine factor replacement therapy prior to the study will receive only on-demand treatment for bleeding episodes.

Enrollment: 17 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 obtained before undergoing any study specific procedures

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, abnormal kidney function, or liver disease
• On-demand subjects experiencing less than 12 or 6 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months, respectively
• Planned major surgical intervention during the study period

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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17 participants in 2 patient groups

On-demand

Experimental group

Description:

The routine prophylactic therapy interval is targeted at every 7 days.

Treatment:

Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein

Prophylactic

Experimental group

Description:

On-demand subjects will receive rIX-FP only for the treatment of a bleeding episode.

Treatment:

Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein