Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B - Full Text View

Purpose

The primary objective of the study is to evaluate the long-term safety of rFIXFc in subjects with hemophillia B.

The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.

Condition	Intervention	Phase
Severe Hemophilia B	Biological: rFIXFc	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:	An Open-Label, Multicenter, Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia

Drug Information available for: Factor IX

U.S. FDA Resources

Further study details as provided by Biogen Idec:

Primary Outcome Measures:

Frequency of inhibitor development [ Time Frame: up to four years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

The number of annualized bleeding episodes (spontaneous and traumatic) per subject [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]
The number of annualized spontaneous joint bleeding episodes per subject [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]
The total number of days of exposure per subject per year [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]
The mean dose of rFIXFc per kg per subject per year per treatment regimen [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]
Physicians global assessment of response to traetment using a 4-point scale [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]
Subject's assessment of response to treatment using a 4-point scale [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]
The incidence of AEs and serious adverse events (SAEs) [ Time Frame: up to 4 years ] [ Designated as safety issue: Yes ]
Investigator/Surgeon assessment of hemostatic response to surgery using the 4-point bleeding response scale [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]
Number of injections and dose per injection to maintain hemostatis during the surgical period [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]
Estimated blood loss (mL) during surgery and post-operative period [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]
Number of blood product units transfused during surgery [ Time Frame: up to 4 years ] [ Designated as safety issue: No ]

Estimated Enrollment:	120
Study Start Date:	December 2011
Estimated Study Completion Date:	December 2015
Estimated Primary Completion Date:	December 2015 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: A On-Demand	Biological: rFIXFc IV Administration
Experimental: B Prophylaxis	Biological: rFIXFc IV Administration

Detailed Description:

Subjects will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the patient in the preceding studies, B-LONG 998HB102 (NCT01027364) and pediatric study 9HB02PED (NCT01440946)

Eligibility

Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Subjects who have completed previous rFIXFc studies (NCT01027364 and NCT01440946) Ability to understand the purposes & risks of the study and provide signed and dated informed consent.

Exclusion Criteria:

Confirmed positive high-titer inhibitor test

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Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01425723

Show 30 Study Locations

Sponsors and Collaborators

Biogen Idec

More Information

No publications provided

Responsible Party:	Biogen Idec
ClinicalTrials.gov Identifier:	NCT01425723 History of Changes
Other Study ID Numbers:	9HB01EXT, 2011-003075-11
Study First Received:	August 19, 2011
Last Updated:	May 15, 2013
Health Authority:	United States: Food and Drug Administration Belgium: Federal Agency for Medicinal Products and Health Products France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) China: Food and Drug Administration Australia: Department of Health and Ageing Therapeutic Goods Administration India: Ministry of Health Hong Kong: Department of Health Italy: Ministry of Health South Africa: Department of Health Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Japan: Pharmaceuticals and Medical Devices Agency (PMDA) Brazil: Ministry of Health Germany: Federal Institute for Drugs and Medical Devices Canada: Health Canada United Kingdom: Medicines and Healthcare Products Regulatory Agency Sweden: Medical Products Agency

Keywords provided by Biogen Idec:

B-LONG
B-LONG Extension
rFIXFc
Severe Hemophilia B

Additional relevant MeSH terms:

Hemophilia B
Hemophilia A
Hemorrhage
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases

Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Pathologic Processes

ClinicalTrials.gov processed this record on May 23, 2013

Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B (B-YOND)