Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
NCT ID: NCT04644575
Last Updated: 2025-08-01
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
261 participants
INTERVENTIONAL
2021-02-23
2027-01-15
Brief Summary
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\- To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A
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 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 (QoL) outcomes.
* To evaluate the safety and tolerability of BIVV001 treatment.
* To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B).
* To evaluate the efficacy of BIVV001 for perioperative management
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Arm A: Previously treated in BIVV001 study
This arm includes participants who have completed study EFC16293 or study EFC16295, participants who have completed Arm B or Arm C of this study (LTS16294) and roll over into Arm A, and participants who have completed any other potential BIVV001 study. Participants in this arm will continue receiving BIVV001 prophylaxis treatment once weekly (QW) for a total of 100 exposure days (EDs) cumulative from the parent study and this study. Participants will have the opportunity to continue in this study for up to 4 years, unless BIVV001 is commercially available in their applicable participating country.
efanesoctocog alfa (BIVV001)
Pharmaceutical form:Solution for Injection Route of administration: Intravenous
Arm B: Newly initiated (China Only) in BIVV001
This arm includes Chinese participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) for 52 weeks. After 52 weeks of treatment in this arm B, participants will be able to roll over into arm A.
efanesoctocog alfa (BIVV001)
Pharmaceutical form:Solution for Injection Route of administration: Intravenous
Arm C: Newly initiated in BIVV001 with planned major surgery
This arm includes participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) and will undergo planned major surgery after at least 6 initial EDs with BIVV001, and within 26 weeks from Day 1. After 52 weeks of treatment in arm C, participants will be able to roll into arm A.
efanesoctocog alfa (BIVV001)
Pharmaceutical form:Solution for Injection Route of administration: Intravenous
Interventions
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efanesoctocog alfa (BIVV001)
Pharmaceutical form:Solution for Injection Route of administration: Intravenous
Eligibility Criteria
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Inclusion Criteria
* Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study.
* Male or Female
* For participants new to BIVV001 (Arm B and C)
* Participants who have severe hemophilia A, defined as \<1 IU/dL (\<1%) 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 EDs or 50 EDs for participants aged \<6 years.
* Platelet count ≥100 000 cells/μL at screening.
* A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to enrollment: CD4 lymphocyte count \>200 cells/mm³ and viral load of \<400 000 copies/mL
* Male
* Only for Arm B: Chinese participants
* Only for Arm C: planned major surgery within 6 months after Day 1.
Exclusion Criteria
* Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL.
* Participation in another study.
* For participants new to BIVV001 (Arm B and Arm C)
* Any concurrent clinically significant liver disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. This may include, but is not limited to cirrhosis, portal hypertension, and acute hepatitis.
* Serious active bacterial, fungal, 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.
* History of a positive inhibitor (to FVIII) test defined as ≥0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant.
* Positive inhibitor test (FVIII) result, defined as ≥0.6 BU/mL at screening.
* Treatment with acetylsalicylic acid (ASA) or antiplatelet agents that are not nonsteroidal anti-inflammatory drugs (NSAIDs) within 2 weeks prior to screening.
* Treatment with NSAIDs greater than the maximum dose specified in the regional prescribing information within 2 weeks prior to screening.
* Systemic treatment within 12 weeks prior to Screening with chemotherapy and/or other immunosuppressive drugs (except for the treatment of hepatitis C virus \[HCV\] or HIV).
* Emicizumab use within the 20 weeks prior to screening.
* Major surgery within 8 weeks prior to screening.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
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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Orthopaedic Institute for Children Site Number : 8400003
Los Angeles, California, United States
Children's Hospital Los Angeles Site Number : 8400009
Los Angeles, California, United States
University of California San Diego Site Number : 8400007
San Diego, California, United States
University of Florida Health Site Number : 8400008
Gainesville, Florida, United States
Children's Healthcare of Atlanta Site Number : 8400016
Atlanta, Georgia, United States
Rush University Medical Center Site Number : 8400010
Chicago, Illinois, United States
Children's Hospital Of Iowa Site Number : 8400011
Iowa City, Iowa, United States
University of Michigan Medical Center Site Number : 8400006
Ann Arbor, Michigan, United States
Michigan State University School Of Med Site Number : 8400002
East Lansing, Michigan, United States
Hemostasis and Thrombosis Center of Nevada Site Number : 8400001
Las Vegas, Nevada, United States
New York Presbyterian Hospital/Weill Cornell Medical Center Site Number : 8400017
New York, New York, United States
East Carolina University -2390 Hemby Ln Site Number : 8400015
Greenville, North Carolina, United States
Cincinnati Children's Hospital Medical Center Site Number : 8400012
Cincinnati, Ohio, United States
Children's Research Institute Site Number : 8400013
Columbus, Ohio, United States
Bloodworks Northwest Site Number : 8400005
Seattle, Washington, United States
Children's Hospital of Wisconsin Site Number : 8400014
Milwaukee, Wisconsin, United States
Investigational Site Number : 0320001
CABA, Buenos Aires F.D., Argentina
Investigational Site Number : 0320002
Godoy Cruz, Mendoza Province, Argentina
Investigational Site Number : 0320003
Buenos Aires, , Argentina
Investigational Site Number : 0360004
Camperdown, New South Wales, Australia
Investigational Site Number : 0360001
Westmead, New South Wales, Australia
Investigational Site Number : 0360002
South Brisbane, Queensland, Australia
Investigational Site Number : 0360003
Murdoch, Western Australia, Australia
Investigational Site Number : 0560003
Woluwe-Saint-Lambert, , Belgium
Hemocentro Campinas - UNICAMP Site Number : 0760001
Campinas, São Paulo, Brazil
Investigational Site Number : 1000171
Plovdiv, , Bulgaria
Investigational Site Number : 1000172
Sofia, , Bulgaria
Investigational Site Number : 1240005
Hamilton, Ontario, Canada
Investigational Site Number : 1240004
Hamilton, Ontario, Canada
Investigational Site Number : 1240002
Ottawa, Ontario, Canada
Investigational Site Number : 1240001
Toronto, Ontario, Canada
Investigational Site Number : 1560002
Beijing, , China
Investigational Site Number : 1560006
Beijing, , China
Investigational Site Number : 1560001
Guangzhou, , China
Investigational Site Number : 1560003
Hangzhou, , China
Investigational Site Number : 1560004
Hangzhou, , China
Investigational Site Number : 1560005
Jinan, , China
Investigational Site Number : 1560009
Kunming, , China
Investigational Site Number : 1560010
Kunming, , China
Investigational Site Number : 1560013
Lanzhou, , China
Investigational Site Number : 1560007
Suzhou, , China
Investigational Site Number : 2500005
Brest, , France
Investigational Site Number : 2500004
Bron, , France
Investigational Site Number : 2500001
Le Kremlin-Bicêtre, , France
Investigational Site Number : 2500003
Lille, , France
Investigational Site Number : 2500006
Marseille, , France
Investigational Site Number : 2760304
Berlin, , Germany
Investigational Site Number : 2760302
Bonn, , Germany
Investigational Site Number : 2760001
Frankfurt am Main, , Germany
Investigational Site Number : 2760002
München, , Germany
Investigational Site Number : 3000001
Athens, , Greece
Investigational Site Number : 3480002
Budapest, , Hungary
Investigational Site Number : 3480004
Debrecen, , Hungary
Investigational Site Number : 3480005
Pécs, , Hungary
Investigational Site Number : 3720001
Dublin, , Ireland
Investigational Site Number : 3800002
Napoli, Campania, Italy
Investigational Site Number : 3800001
Milan, , Italy
Investigational Site Number : 3800003
Vicenza, , Italy
Investigational Site Number : 3920425
Nagoya, Aichi-ken, Japan
Investigational Site Number : 3920423
Kitakyushu-shi, Fukuoka, Japan
Investigational Site Number : 3920426
Kawasaki-shi, Kanagawa, Japan
Investigational Site Number : 3920422
Kashihara-Shi, Niigata, Japan
Investigational Site Number : 3920421
Shinjuku-ku, Tokyo, Japan
Investigational Site Number : 3920424
Suginami-ku, Tokyo, Japan
Investigational Site Number : 5280002
Amsterdam, , Netherlands
Investigational Site Number : 5280001
Utrecht, , Netherlands
Investigational Site Number : 4100603
Daegu, Daegu, South Korea
Investigational Site Number : 4100601
Seoul, Seoul-teukbyeolsi, South Korea
Investigational Site Number : 4100600
Seoul, Seoul-teukbyeolsi, South Korea
Investigational Site Number : 7240002
Esplugues de Llobregat, Catalunya [Cataluña], Spain
Investigational Site Number : 7240001
Madrid, Madrid, Comunidad de, Spain
Investigational Site Number : 7520001
Malmo, , Sweden
Investigational Site Number : 7560001
Zurich, , Switzerland
Investigational Site Number : 1580005
Changhua County, , Taiwan
Investigational Site Number : 1580001
Taichung, , Taiwan
Investigational Site Number : 1580003
Taichung, , Taiwan
Investigational Site Number : 1580002
Taipei, , Taiwan
Investigational Site Number : 1580004
Taipei, , Taiwan
Investigational Site Number : 7920004
Antalya, , Turkey (Türkiye)
Investigational Site Number : 7920001
Istanbul, , Turkey (Türkiye)
Investigational Site Number : 7920003
Izmir, , Turkey (Türkiye)
Investigational Site Number : 8260005
London, London, City of, United Kingdom
Investigational Site Number : 8260001
London, London, City of, United Kingdom
Investigational Site Number : 8260003
Birmingham, , United Kingdom
Investigational Site Number : 8260004
Hampshire, , United Kingdom
Countries
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References
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Quintilla JM, de la Gala C, Berrueco R, Claverol J, Figueres B, Bergos A, Rodriguez L, Mora A, DiBiaso V, Llanos C, Nafria B. High-Fidelity Clinical Simulation to Improve a Pediatric Clinical Trial Design: Lessons Learned and Conceptualization of the Return on Investment (ROI) and Return on Engagement (ROE) Analysis. Paediatr Drugs. 2025 Jan;27(1):73-84. doi: 10.1007/s40272-024-00660-8. Epub 2024 Nov 25.
Other Identifiers
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U1111-1244-0517
Identifier Type: REGISTRY
Identifier Source: secondary_id
2023-508929-27-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
2020-002215-22
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
LTS16294
Identifier Type: -
Identifier Source: org_study_id
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