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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01496274
First received: December 19, 2011
Last updated: October 5, 2014
Last verified: October 2014
  Purpose

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.


Condition Intervention Phase
Hemophilia B
Biological: rIX-FP
Phase 2
Phase 3

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 II/III 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:
  • Change in frequency of spontaneous bleeding events between on-demand and prophylaxis treatments (annualized) [ Time Frame: Approximately 14 months ] [ Designated as safety issue: No ]
  • Number of subjects developing inhibitors against factor IX (FIX) [ Time Frame: Approximately 14 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • The frequency of related Adverse Events [ Time Frame: Approximately 14 months ] [ Designated as safety issue: Yes ]
  • Number of subjects developing antibodies against rIX-FP [ Time Frame: Approximately 14 months ] [ Designated as safety issue: Yes ]
  • Proportion of bleeding episodes requiring one or ≤ two infusions of rIX-FP to achieve hemostasis [ Time Frame: Approximately 14 months ] [ Designated as safety issue: No ]
  • Investigator's overall clinical assessment of hemostatic efficacy for treatment of bleeding episodes, based on a four point ordinal scales (excellent, good, moderate, poor/ none) [ Time Frame: Approximately 14 months ] [ Designated as safety issue: No ]
  • rIX-FP consumed per month while maintaining assigned prophylactic treatment interval during routine prophylaxis. [ Time Frame: Approximately 14 months ] [ Designated as safety issue: No ]
  • Incremental recovery of rIX-FP [ Time Frame: 30 minutes ] [ Designated as safety issue: No ]
  • Half-life (t1/2) of a single dose of rIX-FP [ Time Frame: 240 hours ] [ Designated as safety issue: No ]
  • Area under the curve (AUC) [ Time Frame: 240 hours ] [ Designated as safety issue: No ]
    AUC to the last sample with quantifiable drug concentration (AUC0-t) of a single dose of rIX-FP

  • Clearance of a single dose of rIX-FP [ Time Frame: 240 hours ] [ Designated as safety issue: No ]
  • Investigator's (or surgeon's) overall clinical assessment of hemostatic efficacy for surgical prophylaxis, based on a four point ordinal scale (excellent, good, moderate, poor/ none) [ Time Frame: Approximately 14 days ] [ Designated as safety issue: No ]
  • Annualized spontaneous bleeding events during the 7-day prophylactic regimen compared to those during prophylactic regimens longer than 7-days. [ Time Frame: Approximately 14 months ] [ Designated as safety issue: No ]

Enrollment: 63
Study Start Date: February 2012
Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Prophylaxis

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.

Biological: rIX-FP
Recombinant IX-FP (rIX-FP) is a fusion protein linking coagulation factor IX with albumin, and will be administered by intravenous administration
Experimental: On-demand

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.

Biological: rIX-FP
Recombinant IX-FP (rIX-FP) is a fusion protein linking coagulation factor IX with albumin, and will be administered by intravenous administration

  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
  • 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
  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: NCT01496274

  Show 29 Study Locations
Sponsors and Collaborators
CSL Behring
Investigators
Study Director: Program Director CSL Behring
  More Information

No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01496274     History of Changes
Other Study ID Numbers: CSL654_3001, 2011-002415-28
Study First Received: December 19, 2011
Last Updated: October 5, 2014
Health Authority: Austria: Federal Office for Safety in Health Care
Bulgaria: Bulgarian Drug Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Paul-Ehrlich-Institut
Israel: Ministry of Health
Italy: Ministry of Health
Japan: Pharmaceuticals and Medical Devices Agency
Russia: Ministry of Health of the Russian Federation
Spain: Spanish Agency of Medicines
United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on November 20, 2014