Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
NCT ID: NCT04759131
Last Updated: 2025-09-11
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
74 participants
INTERVENTIONAL
2021-02-19
2023-01-18
Brief Summary
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\- To evaluate the safety of BIVV001 in previously treated pediatric participants 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 efficacy of BIVV001 for perioperative management.
* To evaluate the safety and tolerability of BIVV001 treatment.
* To assess the pharmacokinetics (PK) of BIVV001.
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Detailed Description
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All participants completing or remaining at the end of study were offered participation in the planned extension trial.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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BIVV001: Participants aged <6 Years
Participants aged less than (\<) 6 years received BIVV001 at a dose of 50 international units per kilogram (IU/kg) intravenous (IV) injection once-weekly (QW) prophylaxis for 52 weeks.
efanesoctocog alfa (BIVV001)
Pharmaceutical form: solution for injection Route of administration: IV
BIVV001: Participants aged 6 to <12 Years
Participants aged 6 to \<12 years received BIVV001 at a dose of 50 IU/kg IV injection QW prophylaxis for 52 weeks.
efanesoctocog alfa (BIVV001)
Pharmaceutical form: solution for injection Route of administration: IV
Interventions
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efanesoctocog alfa (BIVV001)
Pharmaceutical form: solution for injection Route of administration: IV
Eligibility Criteria
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Inclusion Criteria
* Severe hemophilia A defined as \<1 international units per deciliter (IU/dL) (\<1 percent \[%\]) endogenous Factor VIII (FVIII) 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 (EDs) for participants aged 6 to \<12 years and above 50 EDs for participants aged \<6 years.
* Weight above or equal to 10 kg.
Exclusion Criteria
* History of a positive inhibitor (to FVIII) test defined as greater than or equal to (\>=) 0.6 Bethesda units (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 would not exclude the participant.
* Positive inhibitor test result, defined as \>=0.6 BU/mL at Screening.
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
11 Years
MALE
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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Children's Hospital Los Angeles-Site Number:8400006
Los Angeles, California, United States
University of Florida-Site Number:8400009
Gainesville, Florida, United States
Children's Healthcare of Atlanta-Site Number:8400019
Atlanta, Georgia, United States
Rush University Medical Center-Site Number:8400001
Chicago, Illinois, United States
University of Iowa Hospitals and Clinics-Site Number:8400002
Iowa City, Iowa, United States
NY Presbyterian - Weill Cornell Medical Center-Site Number:8400020
New York, New York, United States
East Carolina University/Brody Medical Sciences Building-Site Number:8400015
Greenville, North Carolina, United States
Cincinnati Children's Hospital Medical Center-Site Number:8400008
Cincinnati, Ohio, United States
Childrens Hospital Of Columbus-Site Number:8400012
Columbus, Ohio, United States
Children's Hospital Of Wisconsin-Site Number:8400005
Milwaukee, Wisconsin, United States
Investigational Site Number :0360001
Westmead, New South Wales, Australia
Investigational Site Number :0360002
South Brisbane, Queensland, Australia
Investigational Site Number :1240004
Hamilton, Ontario, Canada
Investigational Site Number :1240003
London, Ontario, Canada
Investigational Site Number :1240002
Ottawa, Ontario, Canada
Investigational Site Number :1240001
Toronto, Ontario, Canada
Investigational Site Number :2500004
Bron, , France
Investigational Site Number :2500001
Le Kremlin-Bicêtre, , France
Investigational Site Number :2500003
Lille, , France
Investigational Site Number :2760001
Frankfurt am Main, , Germany
Investigational Site Number :2760002
München, , Germany
Investigational Site Number :3480005
Pécs, , Hungary
Investigational Site Number :3720001
Dublin, , Ireland
Investigational Site Number :3800001
Milan, , Italy
Investigational Site Number :3800002
Napoli, , Italy
Investigational Site Number :5280002
Amsterdam, , Netherlands
Investigational Site Number :5280001
Utrecht, , Netherlands
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 :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 :8260003
Birmingham, , United Kingdom
Investigational Site Number :8260001
London, , United Kingdom
Countries
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References
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Malec L, Mathias M, Dunn AL, Nolan B, Wong N, Bystricka L, Khan U, Neill G, Santagostino E, Gunawardena S, Tarango C, Peyvandi F. Post hoc analysis of bleeding episodes and clinically relevant pharmacokinetic parameters among children <12 years old with severe hemophilia A receiving once-weekly efanesoctocog alfa prophylaxis in the XTEND-Kids phase 3 multinational trial. J Thromb Haemost. 2025 Jul 21:S1538-7836(25)00434-9. doi: 10.1016/j.jtha.2025.07.011. Online ahead of print.
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.
Malec L, Peyvandi F, Chan AKC, Konigs C, Zulfikar B, Yuan H, Simpson M, Alvarez Roman MT, Carcao M, Staber JM, Dunn AL, Chou SC, d'Oiron R, Albisetti M, Demissie M, Santagostino E, Yarramaneni A, Wong N, Abad-Franch L, Gunawardena S, Fijnvandraat K; XTEND-Kids Trial Group. Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A. N Engl J Med. 2024 Jul 18;391(3):235-246. doi: 10.1056/NEJMoa2312611.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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EFC16295 Plain Language Results Summary
Other Identifiers
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2020-000769-18
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
U1111-1244-0558
Identifier Type: OTHER
Identifier Source: secondary_id
017464
Identifier Type: OTHER
Identifier Source: secondary_id
EFC16295
Identifier Type: -
Identifier Source: org_study_id
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