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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
Secondary ID [1] 0 0
2011-003075-11
Secondary ID [2] 0 0
9HB01EXT
Universal Trial Number (UTN)
Trial acronym
B-YOND
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Severe Hemophilia B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - 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.


Treatment: Other: rFIXFc
Administered as specified in the treatment arm.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Any Positive Inhibitor Development
Timepoint [1] 0 0
Approximately 5 years
Secondary outcome [1] 0 0
Annualized Bleeding Rate (ABR)
Timepoint [1] 0 0
Approximately 5 years
Secondary outcome [2] 0 0
Annualized Spontaneous Joint Bleeding Episodes
Timepoint [2] 0 0
Approximately 5 years
Secondary outcome [3] 0 0
Total Number of Exposure Days (EDs)
Timepoint [3] 0 0
Approximately 5 years
Secondary outcome [4] 0 0
Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])
Timepoint [4] 0 0
Approximately 5 years
Secondary outcome [5] 0 0
Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale
Timepoint [5] 0 0
Approximately 5 years
Secondary outcome [6] 0 0
Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
Timepoint [6] 0 0
Approximately 5 years

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.

Key
Minimum age
No limit
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* High-titer inhibitor (>/=5.00 BU/mL)

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/12/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/10/2017
Sample size
Target
Accrual to date
Final
120
Recruitment in Australia
Recruitment state(s)
SA,VIC,WA
Recruitment hospital [1] 0 0
Research Site - Adelaide
Recruitment hospital [2] 0 0
Research site - Parkville
Recruitment hospital [3] 0 0
Research site - Murdoch
Recruitment hospital [4] 0 0
Research Site - Perth
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment postcode(s) [3] 0 0
6150 - Murdoch
Recruitment postcode(s) [4] 0 0
6008 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Hawaii
Country [6] 0 0
United States of America
State/province [6] 0 0
Indiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Louisiana
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Washington
Country [11] 0 0
Belgium
State/province [11] 0 0
Bruxelles
Country [12] 0 0
Belgium
State/province [12] 0 0
Leuven
Country [13] 0 0
Brazil
State/province [13] 0 0
Sao Paulo
Country [14] 0 0
Canada
State/province [14] 0 0
Ontario
Country [15] 0 0
Canada
State/province [15] 0 0
Quebec
Country [16] 0 0
China
State/province [16] 0 0
Beijingshì
Country [17] 0 0
China
State/province [17] 0 0
Guangdongsheng
Country [18] 0 0
China
State/province [18] 0 0
Shànghaishì
Country [19] 0 0
China
State/province [19] 0 0
Tianjinshì
Country [20] 0 0
France
State/province [20] 0 0
Bouches-Du-Rhône
Country [21] 0 0
Germany
State/province [21] 0 0
North Rhine-westphalia
Country [22] 0 0
Hong Kong
State/province [22] 0 0
New Territories
Country [23] 0 0
Hong Kong
State/province [23] 0 0
Hong Kong
Country [24] 0 0
India
State/province [24] 0 0
Karnataka
Country [25] 0 0
India
State/province [25] 0 0
Maharashtra
Country [26] 0 0
India
State/province [26] 0 0
Tamil Nadu
Country [27] 0 0
Ireland
State/province [27] 0 0
Dublin
Country [28] 0 0
Italy
State/province [28] 0 0
Florence
Country [29] 0 0
Italy
State/province [29] 0 0
Milano
Country [30] 0 0
Japan
State/province [30] 0 0
Aichi-Ken
Country [31] 0 0
Japan
State/province [31] 0 0
Fukuoka-Ken
Country [32] 0 0
Japan
State/province [32] 0 0
Kanagawa-Ken
Country [33] 0 0
Japan
State/province [33] 0 0
Nara-Ken
Country [34] 0 0
Japan
State/province [34] 0 0
Tokyo-To
Country [35] 0 0
Netherlands
State/province [35] 0 0
Utrecht
Country [36] 0 0
Poland
State/province [36] 0 0
Lodz
Country [37] 0 0
South Africa
State/province [37] 0 0
Gauteng
Country [38] 0 0
South Africa
State/province [38] 0 0
Western Cape
Country [39] 0 0
Sweden
State/province [39] 0 0
Malmö
Country [40] 0 0
Sweden
State/province [40] 0 0
Stockholm
Country [41] 0 0
United Kingdom
State/province [41] 0 0
Cambridgeshire
Country [42] 0 0
United Kingdom
State/province [42] 0 0
Greater London
Country [43] 0 0
United Kingdom
State/province [43] 0 0
Hampshire

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Bioverativ Therapeutics Inc.
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT01425723
Trial related presentations / publications
Shapiro AD, Pasi KJ, Ozelo MC, Kulkarni R, Barnowski C, Winding B, Szamosi J, Lethagen S. Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B. Res Pract Thromb Haemost. 2018 Nov 29;3(1):109-113. doi: 10.1002/rth2.12163. eCollection 2019 Jan.
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Bioverativ Therapeutics Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01425723