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Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B (B-YOND)

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ClinicalTrials.gov Identifier: NCT01425723
Recruitment Status : Completed
First Posted : August 30, 2011
Results First Posted : November 23, 2018
Last Update Posted : December 19, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Bioverativ Therapeutics Inc. )

Study Description
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Brief Summary:

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

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


Condition or disease Intervention/treatment Phase
Severe Hemophilia B Biological: rFIXFc Phase 3

Detailed Description:
Participants 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 Kids B-LONG study 9HB02PED (NCT01440946)
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 120 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (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
Actual Study Start Date : December 8, 2011
Actual Primary Completion Date : October 2017
Actual Study Completion Date : October 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding

Arms and Interventions
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Arm Intervention/treatment
Experimental: On-Demand
The individual dose of rFIXFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor IX peak (recovery) levels.
 Biological: rFIXFc
Administered as specified in the treatment arm.
Other Names:
  • coagulation factor IX (recombinant) Fc fusion protein
  • Alprolix
  • BIIB029
Experimental: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
 Biological: rFIXFc
Administered as specified in the treatment arm.
Other Names:
  • coagulation factor IX (recombinant) Fc fusion protein
  • Alprolix
  • BIIB029


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Any Positive Inhibitor Development [ Time Frame: Approximately 5 years ]
    An inhibitor test result greater than or equal to (>=)0.6 Bethesda units per milliliter (BU/mL), confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Data was summarized by treatment regimen for participants from Study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from Study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.


Secondary Outcome Measures :
  1. Annualized Bleeding Rate (ABR) [ Time Frame: Approximately 5 years ]
    ABR is annualized number of bleeding episodes per participant per year. Bleeding episodes were classified as spontaneous if participant records bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity and classified as traumatic if participant records bleeding event when there is known reason for bleed. ABR=(Number of bleeding episodes during efficacy period/number of days during efficacy period)*365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. ABR was summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.

  2. Annualized Spontaneous Joint Bleeding Episodes [ Time Frame: Approximately 5 years ]
    Bleeding episodes were classified as spontaneous if participant records a bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity. In addition, location of bleed (joint, internal, skin/mucosa or muscle) were also collected. Annualized spontaneous joint bleeding episodes=(Number of spontaneous joint bleeding episodes during efficacy period/number of days during efficacy period)*365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Bleeding episodes were summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.

  3. Total Number of Exposure Days (EDs) [ Time Frame: Approximately 5 years ]
    An exposure day is a 24-hour period in which one or more rFIXFc injections are given. The total number of days of exposure to rFIXFc were summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.

  4. Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg]) [ Time Frame: Approximately 5 years ]
    Annualized consumption = (total international unit per kilogram [IU/kg] of study treatment received during the efficacy period / total number of days during the efficacy period) multiplied by 365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Annualized consumption was summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.

  5. Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale [ Time Frame: Approximately 5 years ]
    Participants were assessed for response to their rFIXFc regimen using following 4-point scale: 1=Excellent: bleeding episodes responded to less than or equal to (<=)usual number of injections or dose of rFIXFc or rate of breakthrough bleeding during prophylaxis was <= that usually observed; 2=Effective: most bleeding episodes responded to same number of injections and dose, but some required more injections or higher doses, or there was minor increase in rate of breakthrough; 3=Partially Effective: bleeding episodes most often required more injections and/or higher doses than expected or adequate breakthrough bleeding prevention during prophylaxis required more frequent injections and/or higher doses and 4=Ineffective: routine failure to control hemostasis/hemostatic control require additional agents. Total number of scale responses =total count of scale responses for all participants; multiple responses per participant including those at scheduled and unscheduled visits are counted.

  6. Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale [ Time Frame: Approximately 5 years ]
    Using eDiary, participant received rating for treatment response to any bleeding episode (BE) using 4-point scale- 1=Excellent: Abrupt pain relief and/or improvement in signs of bleeding within approximately (approx.) 8 hours (h) after initial injection (inj.); 2=Good: Definite pain relief and/or improvement in signs of bleeding within approx. 8h after an injection, but possibly requiring more than 1 injection after 24-48h for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8h after initial injection and requires more than 1 injection and 4=None: No improvement, or condition worsens within approx. 8h after initial injection. This assessment was to be made approx. 8 to 12h from time the injection was given to treat BE and prior to any additional doses of rFIXFc given for same bleeding episode. Percentages are based on the number of bleeding episodes for which a response (excellent or good) was provided for the first injection during the efficacy period.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Subjects who have completed studies 998HB102 (NCT01027364) or 9HB02PED (NCT01440946) or other studies with rFIXFc
  • Ability to understand the purposes & risks of the study and provide signed and dated informed consent.

Key Exclusion Criteria:

  • High-titer inhibitor (>/=5.00 BU/mL)

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01425723


Locations
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Sponsors and Collaborators
Bioverativ Therapeutics Inc.
Investigators
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Study Director: Medical Director Bioverativ Therapeutics Inc.
  Study Documents (Full-Text)

Documents provided by Sanofi ( Bioverativ Therapeutics Inc. ):
Study Protocol  [PDF] November 13, 2013
Statistical Analysis Plan  [PDF] September 5, 2017

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT01425723    
Other Study ID Numbers: 9HB01EXT
2011-003075-11
First Posted: August 30, 2011    Key Record Dates
Results First Posted: November 23, 2018
Last Update Posted: December 19, 2020
Last Verified: November 2018
Keywords provided by Sanofi ( Bioverativ Therapeutics Inc. ):
B-LONG
B-YOND
B-LONG Extension
rFIXFc
Severe Hemophilia B
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked