Clinical trial NCT01425723: Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Severe Hemophilia B
Keywords: B-LONG
B-YOND
B-LONG Extension
rFIXFc
Severe Hemophilia B
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2011-08-30
Enrollment: 120
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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)

Eligibility 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.

Primary outcomes:
measure: Number of Participants With Any Positive Inhibitor Development
timeFrame: Approximately 5 years
Secondary outcomes:
measure: Annualized Bleeding Rate (ABR)
timeFrame: Approximately 5 years
measure: Annualized Spontaneous Joint Bleeding Episodes
timeFrame: Approximately 5 years
measure: Total Number of Exposure Days (EDs)
timeFrame: Approximately 5 years
measure: Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])
timeFrame: Approximately 5 years
measure: Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale
timeFrame: Approximately 5 years
measure: Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
timeFrame: Approximately 5 years
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Research Site
state: Arizona
city: Sacramento
country: United States
facility: Research Site
state: California
city: Aurora
country: United States
facility: Research Site
state: Colorado
city: Atlanta
country: United States
facility: Research Site
state: Georgia
city: Honolulu
country: United States
facility: Research Site
state: Hawaii
city: Indianapolis
country: United States
facility: Research Site
state: Indiana
city: New Orleans
country: United States
facility: Research Site
state: Louisiana
city: East Lansing
country: United States
facility: Research Site
state: Michigan
city: Pittsburgh
country: United States
facility: Research Site
state: Pennsylvania
city: Seattle
country: United States
facility: Research Site
state: Washington
city: Adelaide
country: Australia
facility: Research Site
state: South Australia
city: Parkville
country: Australia
facility: Research site
state: Victoria
city: Murdoch
country: Australia
facility: Research site
state: Western Australia
city: Perth
country: Australia
facility: Research Site
state: Western Australia
city: Bruxelles
country: Belgium
facility: Research Site
city: Leuven
country: Belgium
facility: Research Site
city: Campinas
country: Brazil
facility: Research Site
state: Sao Paulo
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Montreal
country: Canada
facility: Research Site
state: Quebec
city: Beijing
country: China
facility: Research Site
state: Beijingshì
city: Guangzhou
country: China
facility: Research Site
state: Guangdongsheng
city: Shanghai
country: China
facility: Research Site
state: Shànghaishì
city: Tianjing
country: China
facility: Research Site
state: Tianjinshì
city: Marseille
country: France
facility: Research Site
state: Bouches-Du-Rhône
city: Bonn
country: Germany
facility: Research Site
state: North Rhine-westphalia
city: Hong Kong
country: Hong Kong
facility: Research Site
state: New Territories
city: Hong Kong
country: Hong Kong
facility: Research site
city: Bangalore
country: India
facility: Research Site
state: Karnataka
city: Pune
country: India
facility: Research Site
state: Maharashtra
city: Vellore
country: India
facility: Research Site
state: Tamil Nadu
city: Dublin
country: Ireland
facility: Research Site
city: Florence
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Nagoya-Shi
country: Japan
facility: Research Site
state: Aichi-Ken
city: Kitakyushu
country: Japan
facility: Research Site
state: Fukuoka-Ken
city: Kawasaki
country: Japan
facility: Research Site
state: Kanagawa-Ken
city: Kashihara-shi
country: Japan
facility: Research Site
state: Nara-Ken
city: Shinjuku-ku
country: Japan
facility: Research Site
state: Tokyo-To
city: Tokyo
country: Japan
facility: Research Site
state: Tokyo-To
city: Utrecht
country: Netherlands
facility: Research Site
city: Lodz
country: Poland
facility: Research Site
city: Johannesburg
country: South Africa
facility: Research Site
state: Gauteng
city: Cape Town
country: South Africa
facility: Research Site
state: Western Cape
city: Malmö
country: Sweden
facility: Research Site
city: Stockholm
country: Sweden
facility: Research Site
city: Cambridge
country: United Kingdom
facility: Research Site
state: Cambridgeshire
city: London
country: United Kingdom
facility: Research Site
state: Greater London
city: London
country: United Kingdom
facility: Research site
state: Greater London
city: Basingstoke
country: United Kingdom
facility: Research Site
state: Hampshire
Study officials:
affiliation: Bioverativ Therapeutics Inc.
name: Medical Director
role: STUDY_DIRECTOR
References:
citation: 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.