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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01425723
Registration number
NCT01425723
Ethics application status
Date submitted
19/08/2011
Date registered
30/08/2011
Date last updated
19/12/2020
Titles & IDs
Public title
Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B
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Scientific 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
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Secondary ID [1]
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2011-003075-11
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Secondary ID [2]
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9HB01EXT
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Universal Trial Number (UTN)
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Trial acronym
B-YOND
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Severe Hemophilia B
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - rFIXFc
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.
Experimental: Prophylaxis - Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Other interventions: rFIXFc
Administered as specified in the treatment arm.
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants With Any Positive Inhibitor Development
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Assessment method [1]
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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.
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Timepoint [1]
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Approximately 5 years
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Secondary outcome [1]
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Annualized Bleeding Rate (ABR)
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Assessment method [1]
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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.
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Timepoint [1]
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Approximately 5 years
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Secondary outcome [2]
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Annualized Spontaneous Joint Bleeding Episodes
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Assessment method [2]
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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.
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Timepoint [2]
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Approximately 5 years
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Secondary outcome [3]
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Total Number of Exposure Days (EDs)
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Assessment method [3]
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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.
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Timepoint [3]
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Approximately 5 years
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Secondary outcome [4]
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Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])
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Assessment method [4]
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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.
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Timepoint [4]
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Approximately 5 years
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Secondary outcome [5]
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Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale
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Assessment method [5]
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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.
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Timepoint [5]
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Approximately 5 years
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Secondary outcome [6]
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Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
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Assessment method [6]
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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.
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Timepoint [6]
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Approximately 5 years
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Eligibility
Key inclusion criteria
Key
- 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.
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Minimum age
No limit
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Maximum age
No limit
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
- High-titer inhibitor (>/=5.00 BU/mL)
NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
8/12/2011
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/10/2017
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Sample size
Target
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Accrual to date
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Final
120
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Recruitment in Australia
Recruitment state(s)
SA,VIC,WA
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Recruitment hospital [1]
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Research Site - Adelaide
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Recruitment hospital [2]
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Research site - Parkville
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Recruitment hospital [3]
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Research site - Murdoch
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Recruitment hospital [4]
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Research Site - Perth
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Recruitment postcode(s) [1]
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5000 - Adelaide
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment postcode(s) [3]
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6150 - Murdoch
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Recruitment postcode(s) [4]
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6008 - Perth
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Recruitment outside Australia
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United States of America
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Arizona
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California
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Colorado
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Georgia
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Hawaii
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Indiana
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Ontario
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China
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China
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Hampshire
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Bioverativ Therapeutics Inc.
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Address
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Ethics approval
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01425723
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Trial related presentations / publications
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Contacts
Principal investigator
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Medical Director
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Address
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Bioverativ Therapeutics Inc.
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01425723
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