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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01361126
First received: May 25, 2011
Last updated: August 1, 2012
Last verified: August 2012
  Purpose

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.


Condition Intervention Phase
Hemophilia B
Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I/II Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Frequency of related adverse events [ Time Frame: Approximately 20 weeks ] [ Designated as safety issue: Yes ]
  • Number of subjects developing inhibitors against factor IX (FIX) [ Time Frame: Approximately 20 weeks ] [ Designated as safety issue: Yes ]
  • Number of subjects developing antibodies against rIX-FP [ Time Frame: Approximately 20 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Area under the curve (AUC) [ Time Frame: 168 Hours ] [ Designated as safety issue: No ]
    AUC to the last sample with quantifiable drug concentration (AUC0-t) of a single dose of rIX-FP

  • Half-life (t1/2) of a single dose of rIX-FP [ Time Frame: 168 hours ] [ Designated as safety issue: No ]
  • Incremental recovery of rIX-FP [ Time Frame: 30 minutes ] [ Designated as safety issue: No ]
  • Clearance of a single dose of rIX-FP [ Time Frame: 168 hours ] [ Designated as safety issue: No ]
  • Breakthrough bleeding events [ Time Frame: Week 9 to approximately Week 20 ] [ Designated as safety issue: No ]
    Number of breakthrough bleeding events in subjects receiving prophylactic treatment regimen with rIX-FP


Enrollment: 17
Study Start Date: July 2011
Study Completion Date: July 2012
Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: On-demand
The routine prophylactic therapy interval is targeted at every 7 days.
Biological: 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.
Other Name: CSL654
Experimental: Prophylactic
On-demand subjects will receive rIX-FP only for the treatment of a bleeding episode.
Biological: 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.
Other Name: CSL654

  Eligibility

Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01361126

Locations
Bulgaria
Study Site
Sofia, Bulgaria
Israel
Study Site
Tel Aviv, Israel
Sponsors and Collaborators
CSL Behring
Investigators
Study Director: Iris Jacobs, MD CSL Behring
  More Information

No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01361126     History of Changes
Other Study ID Numbers: CSL654_2004, 2010-023793-39
Study First Received: May 25, 2011
Last Updated: August 1, 2012
Health Authority: Bulgaria: Bulgarian Drug Agency
Israel: Ministry of Health

Additional relevant MeSH terms:
Hemophilia B
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on August 18, 2014