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

NCT ID: NCT01361126

Last Updated: 2016-05-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-07-31
• Study Completion Date: 2012-07-31

Brief Summary

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.

Conditions

Hemophilia B

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

On-demand

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

Group Type: EXPERIMENTAL

Recombinant Coagulation Factor IX Albumin Fusion Protein

Intervention Type: BIOLOGICAL

Study subjects will receive a single dose of 25IU/kg of rIX\_FP for pharmacokinetic analysis. Subjects will then be treated for approximately 5 months. The treatment dose will be based on the subject's PK profile and the subject's bleeding phenotype.

Prophylactic

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

Group Type: EXPERIMENTAL

Recombinant Coagulation Factor IX Albumin Fusion Protein

Intervention Type: BIOLOGICAL

Study subjects will receive a single dose of 25IU/kg of rIX\_FP for pharmacokinetic analysis. Subjects will then be treated for approximately 5 months. The treatment dose will be based on the subject's PK profile and the subject's bleeding phenotype.

Interventions

Recombinant Coagulation Factor IX Albumin Fusion Protein

Study subjects will receive a single dose of 25IU/kg of rIX\_FP for pharmacokinetic analysis. Subjects will then be treated for approximately 5 months. The treatment dose will be based on the subject's PK profile and the subject's bleeding phenotype.

Intervention Type: BIOLOGICAL

Other Intervention Names

CSL654

Eligibility Criteria

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

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

CSL Behring

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Iris Jacobs, MD

Role: STUDY_DIRECTOR

CSL Behring

Locations

Study Site

Sofia, , Bulgaria

Study Site

Tel Aviv, , Israel

Countries

Bulgaria; Israel

Other Identifiers

2010-023793-39

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSL654_2004

Identifier Type: -

Identifier Source: org_study_id