Clinical trial NCT01454739: Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A
Official title: An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia A
Keywords: rFVIIIFc
A-LONG Extension
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2011-10-19
Enrollment: 240
Sex: MALE
Minimum age (years): 0
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 recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.

Detailed description: Participant will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the participant in the preceding studies A-LONG - 997HA301 (NCT01181128), pediatric study 8HA02PED (NCT01458106), 997HA307 (NCT02083965) and 997HA309 (NCT02502149).

Eligibility criteria: Key Inclusion Criteria:

* Subjects who have completed previous rFVIIIFc studies (NCT01181128, NCT02083965, NCT01458106 and NCT02502149)
* Ability to understand purposes and risks of the study and to provide signed and dated informed consent (or assent, as applicable).

Key Exclusion Criteria:

* Confirmed positive 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 rFVIIIFc Consumption (International Units Per Kilogram [IU/kg])
timeFrame: Approximately 5 years
measure: Physicians' Global Assessment of Participant's Response to rFVIIIFc Regimen Using a 4-Point Scale
timeFrame: Approximately 5 years
measure: Participant's Assessment of Response (Excellent or Good Response) to rFVIIIFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
timeFrame: Approximately 5 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Research Site
state: California
city: Los Angeles
country: United States
facility: Research Site
state: California
city: Orange
country: United States
facility: Research Site
state: California
city: Sacramento
country: United States
facility: Research Site
state: California
city: San Diego
country: United States
facility: Research Site
state: California
city: Washington
country: United States
facility: Research Site
state: District of Columbia
city: Indianapolis
country: United States
facility: Research Site
state: Indiana
city: Iowa City
country: United States
facility: Research Site
state: Iowa
city: New Orleans
country: United States
facility: Research Site
state: Louisiana
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: East Lansing
country: United States
facility: Research Site
state: Michigan
city: Saint Louis
country: United States
facility: Research Site
state: Missouri
city: Las Vegas
country: United States
facility: Research Site
state: Nevada
city: Chapel Hill
country: United States
facility: Research Site
state: North Carolina
city: Cincinnati
country: United States
facility: Research Site
state: Ohio
city: Portland
country: United States
facility: Research Site
state: Oregon
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Research Site
state: Pennsylvania
city: Houston
country: United States
facility: Research Site
state: Texas
city: Salt Lake City
country: United States
facility: Research Site
state: Utah
city: Seattle
country: United States
facility: Research Site
state: Washington
city: Camperdown
country: Australia
facility: Research Site
state: New South Wales
city: South Brisbane
country: Australia
facility: Research Site
state: Queensland
city: Adelaide
country: Australia
facility: Research Site
state: South Australia
city: Melbourne
country: Australia
facility: Research Site
state: Victoria
city: Melbourne
country: Australia
facility: Research site
state: Victoria
city: Murdoch
country: Australia
facility: Research Site
state: Western Australia
city: Subiaco
country: Australia
facility: Research Site
state: Western Australia
city: Vienna
country: Austria
facility: Research Site
city: Bruxelles
country: Belgium
facility: Research Site
state: Brussels
city: Campinas
country: Brazil
facility: Research Site
state: Sao Paulo
city: Vancouver
country: Canada
facility: Research Site
state: British Columbia
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Bron cedex
country: France
facility: Research Site
state: Rhone
city: Bonn
country: Germany
facility: Research Site
state: Nordrhein Westfalen
city: Berlin
country: Germany
facility: Research Site
city: Hong Kong
country: Hong Kong
facility: Children Cancer Centre
state: New Territories
city: Hong Kong
country: Hong Kong
facility: Sir Yue Kong Pao Center for Cancer
state: New Territories
city: Hong Kong
country: Hong Kong
facility: Research Site
city: New Delhi
country: India
facility: Research Site
state: Delhi
city: Bangalore
country: India
facility: Research Site
state: Karnataka
city: Pune
country: India
facility: Research Site
state: Maharashtra
city: Ludhiana
country: India
facility: Research Site
state: Punjab
city: Vellore
country: India
facility: Research Site
state: Tamilnadu
city: Dublin
country: Ireland
facility: Research Site
city: Ramat Gan
country: Israel
facility: Research Site
city: Firenze
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Vicenza
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: Groningen
country: Netherlands
facility: Research Site
city: Auckland
country: New Zealand
facility: Research Site
city: Christchurch
country: New Zealand
facility: Research Site
city: Hamilton
country: New Zealand
facility: Research site
city: Palmerston North
country: New Zealand
facility: Research Site
city: Wellington
country: New Zealand
facility: Research site
city: Lublin
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: Barcelona
country: Spain
facility: Research Site
city: Madrid
country: Spain
facility: Research Site
city: Göteborg
country: Sweden
facility: Research Site
city: Zuerich
country: Switzerland
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: London
country: United Kingdom
facility: Research Site
state: Greater London
city: Basingstoke
country: United Kingdom
facility: Research Site
state: Hampshire
city: Hamstead
country: United Kingdom
facility: Research Site
state: London
city: Glasgow
country: United Kingdom
facility: Research Site
state: Strathclyde
city: Glasgow
country: United Kingdom
facility: Research Site
state: Strathclyde
city: London
country: United Kingdom
facility: Research Site
Study officials:
affiliation: Bioverativ Therapeutics Inc.
name: Medical Director
role: STUDY_DIRECTOR
References:
citation: Nolan B, Mahlangu J, Pabinger I, Young G, Konkle BA, Barnes C, Nogami K, Santagostino E, Pasi KJ, Khoo L, Winding B, Yuan H, Fruebis J, Rudin D, Oldenburg J. Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study. Haemophilia. 2020 May;26(3):494-502. doi: 10.1111/hae.13953. Epub 2020 Mar 30.
citation: Nolan B, Mahlangu J, Perry D, Young G, Liesner R, Konkle B, Rangarajan S, Brown S, Hanabusa H, Pasi KJ, Pabinger I, Jackson S, Cristiano LM, Li X, Pierce GF, Allen G. Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A. Haemophilia. 2016 Jan;22(1):72-80. doi: 10.1111/hae.12766. Epub 2015 Jul 27.