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 is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01496274
First received: December 19, 2011
Last updated: April 7, 2014
Last verified: April 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 ]

Estimated Enrollment: 60
Study Start Date: December 2011
Estimated Primary Completion Date: June 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01496274

  Show 33 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: April 7, 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
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 April 15, 2014