A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A
NCT ID: NCT04161495
Last Updated: 2025-09-17
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
159 participants
INTERVENTIONAL
2019-11-19
2022-02-03
Brief Summary
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\- To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment arm.
Secondary Objectives:
* To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
* To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes.
* To evaluate BIVV001 consumption for the prevention and treatment of bleeding episodes.
* To evaluate the effect of BIVV001 prophylaxis on joint health outcomes.
* To evaluate the effect of BIVV001 prophylaxis on Quality of Life outcomes.
* To evaluate the efficacy of BIVV001 for perioperative management.
* To evaluate the safety and tolerability of BIVV001 treatment.
* To assess the pharmacokinetics (PK) of BIVV001 based on the 1-stage activated partial thromboplastin time (aPTT) and 2-stage chromogenic coagulation factor VIII (FVIII) activity assays.
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Detailed Description
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The Sponsor planned to perform a long-term safety trial. Enrollment in this open-label extension study would be offered to participants completing the treatment period based on eligibility criteria.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Arm A: Prophylaxis
Participants who were on a prophylaxis treatment with a FVIII product prior to study EFC16293 including participants who rolled over from study OBS16221, received BIVV001 50 international units per kilogram (IU/kg) intravenous (IV) injection once-weekly (QW) for 52 weeks in the current study. Study OBS16221 participants with 6 months historical data on prophylaxis treatment with a marketed FVIII product prior to enrollment were analyzed as a subgroup (named as: Arm A: Historical Prophylaxis \[OBS16221\]) in the outcome measure analysis.
efanesoctocog alfa (BIVV001)
Pharmaceutical form: solution for injection Route of administration: IV injection
Arm B: On-Demand Then Prophylaxis
Participants who were on an on-demand treatment regimen with a FVIII product prior to study EFC16293, including participants who rolled over from study OBS16221, received BIVV001 50 IU/kg IV injection as an on-demand treatment (as needed for the treatment of bleeding episodes) from Week 1 to Week 26 in current study. At Week 26, participants in Arm B were switched to prophylaxis treatment, and received BIVV001 50 IU/kg, IV injection QW until Week 52.
efanesoctocog alfa (BIVV001)
Pharmaceutical form: solution for injection Route of administration: IV injection
Interventions
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efanesoctocog alfa (BIVV001)
Pharmaceutical form: solution for injection Route of administration: IV injection
Eligibility Criteria
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Inclusion Criteria
* Severe hemophilia A, defined as less than (\<) 1 international units per deciliter (IU/dL) (\<1 percent \[%\]) endogenous FVIII activity as documented either by central laboratory testing at Screening or in historical medical records from a clinical laboratory demonstrating \<1% FVIII coagulant activity (FVIII:C) or a documented genotype known to produce severe hemophilia A.
* Previous treatment for hemophilia A (prophylaxis or on demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 exposure days.
* Current regimen included one of the following:
* Prophylactic treatment regimen with a FVIII product or prophylactic emicizumab therapy for at least 6 months during the previous 12 months. Appropriate washout time needs to be taken into account.
* On-demand regimen with a FVIII product with a history of at least 12 bleeding episodes in the previous 12 months or at least 6 bleeding episodes in the previous 6 months prior to study enrollment.
* On-demand participant was accepted to move to a prophylaxis treatment regimen after 26-week on-demand period.
* Willingness and ability of the participant or surrogate (a caregiver or a family member greater than or equal to \[\>=\] 18 years of age) to complete training in the use of the study electronic Patient Diary (ePD) and to use the ePD throughout the study.
* Ability of the participant or his or her legally authorized representative (eg., parent or legal guardian) to understand the purpose and risks of the study, willing and able to comply with study requirements and provide signed and dated informed consent or assent (as applicable) and authorization to use protected health information in accordance with national and local participant privacy regulations.
Exclusion Criteria
* Serious active bacterial or viral infection (other than chronic hepatitis or HIV) present within 30 days of screening.
* Other known coagulation disorder(s) in addition to hemophilia A.
* History of hypersensitivity or anaphylaxis associated with any FVIII product.
* Positive inhibitor results, defined as \>=0.6 Bethesda unit per milliliter (BU/mL) at screening. History of a positive inhibitor test defined as \>=0.6 BU/mL. Family history of inhibitors would not exclude the participant.
* Use of Emicizumab within the 20 weeks prior to screening.
* Major surgery within 8 weeks prior to screening.
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
12 Years
ALL
No
Sponsors
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Bioverativ, a Sanofi company
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Sciences & Operations
Role: STUDY_DIRECTOR
Sanofi
Locations
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Investigational Site Number 920
Los Angeles, California, United States
Investigational Site Number 921
Los Angeles, California, United States
Investigational Site Number 911
San Diego, California, United States
Investigational Site Number 917
Gainesville, Florida, United States
Investigational Site Number 919
Ann Arbor, Michigan, United States
Investigational Site Number 908
Lansing, Michigan, United States
Investigational Site Number 906
Las Vegas, Nevada, United States
Investigational Site Number 902
Seattle, Washington, United States
Investigational Site Number 136
Buenos Aires, , Argentina
Investigational Site Number 137
Buenos Aires, , Argentina
Investigational Site Number 139
Mendoza, , Argentina
Investigational Site Number 121
Perth, , Australia
Investigational Site Number 122
Sydney, , Australia
Investigational Site Number 161
Brussels, , Belgium
Investigational Site Number 181
Campinas, , Brazil
Investigational Site Number 171
Plovdiv, , Bulgaria
Investigational Site Number 172
Sofia, , Bulgaria
Investigational Site Number 202
Hamilton, , Canada
Investigational Site Number 205
Hamilton, , Canada
Investigational Site Number 281
Brest, , France
Investigational Site Number 283
Lille, , France
Investigational Site Number 282
Lyon, , France
Investigational Site Number 284
Marseille, , France
Investigational Site Number 304
Berlin, , Germany
Investigational Site Number 302
Bonn, , Germany
Investigational Site Number 303
Frankfurt, , Germany
Investigational Site Number 321
Athens, , Greece
Investigational Site Number 312
Budapest, , Hungary
Investigational Site Number 314
Debrecen, , Hungary
Investigational Site Number 402
Milan, , Italy
Investigational Site Number 401
Vicenza, , Italy
Investigational Site Number 426
Kawasaki, , Japan
Investigational Site Number 423
Kitakyushu, , Japan
Investigational Site Number 425
Nagoya, , Japan
Investigational Site Number 422
Nara, , Japan
Investigational Site Number 421
Tokyo, , Japan
Investigational Site Number 424
Tokyo, , Japan
Investigational Site Number 435
Durango, , Mexico
Investigational Site Number 641
Utrecht, , Netherlands
Investigational Site Number 603
Daegu, , South Korea
Investigational Site Number 600
Seoul, , South Korea
Investigational Site Number 601
Seoul, , South Korea
Investigational Site Number 521
Madrid, , Spain
Investigational Site Number 531
Chang-hua, , Taiwan
Investigational Site Number 532
Taipei, , Taiwan
Investigational Site Number 581
Basingstoke, , United Kingdom
Investigational Site Number 581
London, , United Kingdom
Countries
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References
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Klamroth R, von Drygalski A, Hermans C, Park YS, Chan AKC, Kupesiz A, Alvarez-Roman MT, Malec L, Santagostino E, Neill G, Bystricka L, Dumont J, Abad-Franch L, Fetita LS, Khoo L. Perioperative Management With Efanesoctocog Alfa in Patients With Haemophilia A in the Phase 3 XTEND-1 and XTEND-Kids Studies. Haemophilia. 2025 May;31(3):391-400. doi: 10.1111/hae.70017. Epub 2025 Mar 18.
Weyand AC, Meunier S, Suzuki N, Bystricka L, Neill G, Abad-Franch L, Willemze A, Tosetto A. Treatment of Bleeding Episodes With Efanesoctocog Alfa in Previously Treated Patients With Severe Hemophilia A in the Phase 3 XTEND-1 Study. Am J Hematol. 2025 May;100(5):813-820. doi: 10.1002/ajh.27603. Epub 2025 Feb 10.
DiBenedetti D, Neme D, Pan-Petesch B, Willemze A, Wynn T, Kragh N, Wilson A. Patient Experience With Efanesoctocog Alfa for Severe Hemophilia A: Results From the XTEND-1 Phase 3 Clinical Study Exit Interviews. Clin Ther. 2024 Dec;46(12):1016-1023. doi: 10.1016/j.clinthera.2024.09.010. Epub 2024 Oct 15.
von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, Matino D, Klamroth R, Weyand AC, Jimenez-Yuste V, Nogami K, Poloskey S, Winding B, Willemze A, Knobe K; XTEND-1 Trial Group. Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A. N Engl J Med. 2023 Jan 26;388(4):310-318. doi: 10.1056/NEJMoa2209226.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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EFC16293 Plain Language Results Summary
Other Identifiers
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2019-002023-15
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
U1111-1223-4867
Identifier Type: REGISTRY
Identifier Source: secondary_id
EFC16293
Identifier Type: -
Identifier Source: org_study_id
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