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

CSL Behring

Status and phase: Completed; Phase 3

Conditions: Hemophilia B

Treatments: Biological: rIX-FP

Study type: Interventional

Funder types: Industry

Identifiers

NCT01662531

CSL654_3002

2011-006032-23 (EudraCT Number)

Details and patient eligibility

About

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

Enrollment: 27 patients

Sex: Male

Ages: Under 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male subjects, younger than 12 years old.
• Severe hemophilia B (Factor IX [FIX] activity of ≤ 2%).
• Body weight ≥ 10 kg.
• Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs) (6 to < 12 years), and > 50 EDs (< 6 years).
• 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.

Exclusion criteria

• Known hypersensitivity to any FIX product or hamster protein.
• Known congenital or acquired coagulation disorder other than congenital FIX deficiency.
• Kidney or liver disease.
• Recent life-threatening bleeding episode.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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27 participants in 1 patient group

rIX-FP

Experimental group

Description:

Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) will be administered by IV infusion as routine weekly prophylaxis and episodic treatment for bleeding episodes.

Treatment:

Biological: rIX-FP