Hemophilia APhase 3XTEND-edNCT04644575
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
A Phase 3 study testing efanesoctocog alfa (BIVV001) in people with Hemophilia A, run by Bioverativ, a Sanofi company at 85 sites in 23 countries.
Trial summary
Primary Objective: 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- What is being tested?
- efanesoctocog alfa (BIVV001)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 261 participants
- Where
- 85 sitesin 23 countries
- Study dates
- 23 Feb 2021to 15 Jan 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- For participants rolling over into Arm A
- 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.
Show all 11Show fewer
- 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.
Who cannot join
- For participants rolling over into Arm A
- 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.
Show all 16Show fewer
- 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.
Trial locations and contacts
85 sites in 23 countries.
85 sites in 23 countries
United States16 sites
University of Michigan Medical Center Site Number : 8400006
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta Site Number : 8400016
Atlanta, Georgia, 30322
Rush University Medical Center Site Number : 8400010
Chicago, Illinois, 60612-3833
Cincinnati Children's Hospital Medical Center Site Number : 8400012
Cincinnati, Ohio, 45229
Children's Research Institute Site Number : 8400013
Columbus, Ohio, 43205-2696
Michigan State University School Of Med Site Number : 8400002
East Lansing, Michigan, 48824
University of Florida Health Site Number : 8400008
Gainesville, Florida, 32608
East Carolina University -2390 Hemby Ln Site Number : 8400015
Greenville, North Carolina, 27834
Children's Hospital Of Iowa Site Number : 8400011
Iowa City, Iowa, 52242
Hemostasis and Thrombosis Center of Nevada Site Number : 8400001
Las Vegas, Nevada, 89113
Orthopaedic Institute for Children Site Number : 8400003
Los Angeles, California, 90007
Children's Hospital Los Angeles Site Number : 8400009
Los Angeles, California, 90027
Children's Hospital of Wisconsin Site Number : 8400014
Milwaukee, Wisconsin, 53226-0509
New York Presbyterian Hospital/Weill Cornell Medical Center Site Number : 8400017
New York, New York, 10021
University of California San Diego Site Number : 8400007
San Diego, California, 92121
Bloodworks Northwest Site Number : 8400005
Seattle, Washington, 98104
China10 sites
Investigational Site Number : 1560002
Beijing, 100045
Investigational Site Number : 1560006
Beijing, 100730
Investigational Site Number : 1560001
Guangzhou, 510515
Investigational Site Number : 1560003
Hangzhou, 310003
Investigational Site Number : 1560004
Hangzhou, 310003
Investigational Site Number : 1560005
Jinan, 250013
Investigational Site Number : 1560009
Kunming, 650032
Investigational Site Number : 1560010
Kunming, 650101
Investigational Site Number : 1560013
Lanzhou, 730000
Investigational Site Number : 1560007
Suzhou, 215006
Japan6 sites
Investigational Site Number : 3920422
Kashihara-Shi, Niigata, 634-8521
Investigational Site Number : 3920426
Kawasaki-shi, Kanagawa, 216-8511
Investigational Site Number : 3920423
Kitakyushu-shi, Fukuoka, 807-8556
Investigational Site Number : 3920425
Nagoya, Aichi-ken, 466-0065
Investigational Site Number : 3920421
Shinjuku-ku, Tokyo, 160-0023
Investigational Site Number : 3920424
Suginami-ku, Tokyo, 167-0035
France5 sites
Investigational Site Number : 2500005
Brest, 29200
Investigational Site Number : 2500004
Bron, 69500
Investigational Site Number : 2500001
Le Kremlin-Bicêtre, 94275
Investigational Site Number : 2500003
Lille, 59037
Investigational Site Number : 2500006
Marseille, 13385
Taiwan5 sites
Investigational Site Number : 1580005
Changhua County, 500
Investigational Site Number : 1580001
Taichung, 40201
Investigational Site Number : 1580003
Taichung, 407
Investigational Site Number : 1580002
Taipei, 10002
Investigational Site Number : 1580004
Taipei, 11031
Australia4 sites
Investigational Site Number : 0360004
Camperdown, New South Wales, 2050
Investigational Site Number : 0360003
Murdoch, Western Australia, 6961
Investigational Site Number : 0360002
South Brisbane, Queensland, 4101
Investigational Site Number : 0360001
Westmead, New South Wales, 2145
Canada4 sites
Investigational Site Number : 1240005
Hamilton, Ontario, L8L 8E7
Investigational Site Number : 1240004
Hamilton, Ontario, L8N 3Z5
Investigational Site Number : 1240002
Ottawa, Ontario, K1H 8L1
Investigational Site Number : 1240001
Toronto, Ontario, M5G 1X8
Germany4 sites
Investigational Site Number : 2760304
Berlin, 10249
Investigational Site Number : 2760302
Bonn, 53127
Investigational Site Number : 2760001
Frankfurt am Main, 60590
Investigational Site Number : 2760002
München, 80337
United Kingdom4 sites
Investigational Site Number : 8260003
Birmingham, B4 6NH
Investigational Site Number : 8260004
Hampshire, RG24 9NA
Investigational Site Number : 8260005
London, London, City of, NW3 2QG
Investigational Site Number : 8260001
London, London, City of, WC1N 3JH
Argentina3 sites
Investigational Site Number : 0320003
Buenos Aires, C1015ABO
Investigational Site Number : 0320001
CABA, Buenos Aires F.D., C1425BWE
Investigational Site Number : 0320002
Godoy Cruz, Mendoza Province, M5504FKD
Hungary3 sites
Investigational Site Number : 3480002
Budapest, 1134
Investigational Site Number : 3480004
Debrecen, 4093
Investigational Site Number : 3480005
Pécs, 7623
Italy3 sites
Investigational Site Number : 3800001
Milan, 20121
Investigational Site Number : 3800002
Naples, Campania, 80123
Investigational Site Number : 3800003
Vicenza, 36100
South Korea3 sites
Investigational Site Number : 4100603
Daegu, Daegu, 41404
Investigational Site Number : 4100601
Seoul, Seoul-teukbyeolsi, 03722
Investigational Site Number : 4100600
Seoul, Seoul-teukbyeolsi, 05278
Turkey (Türkiye)3 sites
Investigational Site Number : 7920004
Antalya, 07059
Investigational Site Number : 7920001
Istanbul, 34390
Investigational Site Number : 7920003
Izmir, TR-35100
Bulgaria2 sites
Investigational Site Number : 1000171
Plovdiv, 4002
Investigational Site Number : 1000172
Sofia, 1756
Netherlands2 sites
Investigational Site Number : 5280002
Amsterdam, 1105 AZ
Investigational Site Number : 5280001
Utrecht, 3584 CX
Spain2 sites
Investigational Site Number : 7240002
Esplugues de Llobregat, Catalunya [Cataluña], 08950
Investigational Site Number : 7240001
Madrid, Madrid, Comunidad de, 28046
Belgium1 site
Investigational Site Number : 0560003
Woluwe-Saint-Lambert, 1200
Brazil1 site
Hemocentro Campinas - UNICAMP Site Number : 0760001
Campinas, São Paulo, 13083-970
About this study
Official title: A Phase 3 Open-label, Multicenter Study of the Long-term Safety and Efficacy of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients With Severe Hemophilia A
Primary Objective:
- To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A
Read more
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
Read the detailed description
Participants will receive BIVV001 once weekly for a total of at least 100 exposure days to BIVV001 (including exposure during a BIVV001 parent study, if applicable). 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.
- Conditions
- Hemophilia A
- Being tested
- efanesoctocog alfa (BIVV001)
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
efanesoctocog alfa (BIVV001)Drug
Pharmaceutical form:Solution for Injection Route of administration: Intravenous
| Study group | Role | What they receive |
|---|---|---|
| Arm A: Previously treated in BIVV001 study | Experimental | 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. Receives: Drug: efanesoctocog alfa (BIVV001) |
| Arm B: Newly initiated (China Only) in BIVV001 | Experimental | 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. Receives: Drug: efanesoctocog alfa (BIVV001) |
| Arm C: Newly initiated in BIVV001 with planned major surgery | Experimental | 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. Receives: Drug: efanesoctocog alfa (BIVV001) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII])
The number of participants with the occurrence of inhibitor development (neuatralizing antibodies detected against factor VIII [FVIII]) as determined via the Nijmegen modified Bethesda assay.
Measured: Baseline to month 48
Secondary outcomes (33)
Annual bleeding rate (ABR)
Annualized bleeding rate (ABR) for treated bleeding episodes and all bleeding episodes (including untreated bleeds).
Measured: Baseline to month 48
Annualized bleeding rate (ABR) by type of bleed
Annualized bleeding rate (ABR) by type during prophylaxis treatment per study arm and parent study.
Measured: Baseline to month 48
Annualized bleeding rate (ABR) by location
Annualized bleeding rate (ABR) by location during prophylaxis treatment per study arm and parent study.
Measured: Baseline to month 48
Percentage of patients who maintain factor VIII (FVIII) above prespecified activity levels
Percentage of participants who maintain factor VIII (FVIII) activity levels over 7 days post dose during prophylaxis treatment per study arm and per parent study or arm.
Measured: Baseline to month 48
Number of injections and dose of BIVV0001 to treat a bleeding episode
Measured: Month 48
Percentage of bleeding episode treated with a single injection of BIVV001
Measured: Month 48
Assessment of response to BIVV001 treatment of individual bleeding episodes
Assessment of response to BIVV001 treatment of individual bleeding episodes based on the International Society on Thrombosis and Haemostasis (ISTH) 4-point response scale
Measured: Baseline to month 48
Physician's global assessment (PGA) of participants response to BIVV001
Physician's global assessment (PGA) of participant's response to BIVV001 treatment based on a 4-point response scale .
Measured: Baseline to month 48
Total annualized BIVV001 consumption
Total annualized BIVV001 consumption per participant during prophylaxis treatment
Measured: Baseline to month 48
Annualized joint bleeding rate (AJBR)
Measured: Baseline to month 48
Target joint resolution
Target joint development, resolution and maintenance of target joint resolution based on ISTH criteria.
Measured: Month 48
Change from baseline in Hemophilia Joint Health Score (HJHS)
Change from Baseline to the end of study visit in total score and domain scores (eg, swelling and strength) assessed by the Hemophilia Joint Health Score (HJHS)
Measured: Baseline to month 48
Change from baseline in PROMIS-SF Physical Function
Change in Quality of Life (QoL) measures from baseline to end of study visit per study arm and per parent study arm: PROMIS-SF Physical Function (participants aged ≥18 years old).)
Measured: Baseline to month 48
Change from baseline in Haem-A-QoL total score and physical health score
Change from baseline in Haemophilia QoL Questionnaire for Adults (Haem-A-QoL) total and physical health domain score on participants aged ≥17 years old.
Measured: Baseline to month 48
Change from baselin in Haemo-QoL total score and physical health score
Change from baseline in Haemophilia QoL Questionnaire for Children (Haemo-QoL) total and physical health domain score on participants aged ≥4 to 16 years old and parent proxy for participants aged ≥4 to to <12 years old.
Measured: Baseline to month 48
Number of participants with adverse events (AEs) and serious adverse events (SAEs)
Participants with occurrences of treatment emergent adverse events (AEs) and serious adverse events (SAEs).
Measured: Baseline to month 48
Number of participants with the occurrence of embolic and thrombotic events
Participants with the occurrence of embolic and thrombotic events.
Measured: Baseline to month 48
PK parameter: Maximum activity (Cmax)
Measured: Baseline to week 52
PK parameter: Elimination half-life (t1/2)
Measured: Baseline to week 26
PK parameter: Total clearance (CL)
Measured: Baseline to week 26
PK parameter: Total clearance at steady state (CLss)
Measured: Baseline to week 26
PK parameter: Accumulation index (AI)
Measured: Baseline to week 26
PK parameter: Area under the activity time curve (AUC)
Measured: Baseline to week 26
PK parameter: Volume of distribution at steady state (Vss)
Measured: Baseline to week 26
PK parameter: Mean residence time (MRT)
Measured: Baseline to week 26
PK parameter: Incremental recovery (IR)
Measured: Baseline to week 52
PK parameter: Trough activity (Ctrough)
Measured: Baseline to week 52
PK parameter: Time above FVIII activity levels
Measured: Baseline to week 26
Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment
Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment on the ISTH 4 point response for surgical procedures scale.
Measured: Baseline to month 48
Number of injections and dose to maintain hemostasis during perioperative period for major surgery
Measured: Baseline to month 48
Total BIVV001 consumption during perioperative period for major surgery
Measured: Baseline to month 48
Number and type of blood component transfusions used during perioperative period for major surgery
Measured: Baseline to month 48
Estimated blood loss during perioperative period for major surgery
Measured: Baseline to month 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2020
- Study start23 Feb 2021
- Primary completion15 Jan 2027 (estimated)
- Study completion15 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- It is a 3-arm study with single intervention
- Masking
- None (open label)
- Enrolment
- 261 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioverativ, a Sanofi company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04644575
- Sponsor's protocol ID
- LTS16294
- Other IDs
- U1111-1244-0517, 2023-508929-27-00, 2020-002215-22, 2023
- Acronym
- XTEND-ed
- Status last verified
- Jul 2026
- First submitted
- 23 Nov 2020
- Last updated
- 28 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Publications
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.
PubMed 39585605
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Open-label, Multicenter Study of the Long-term Safety and Efficacy of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients With Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT04644575
- Acronym / sponsor ID
- XTEND-ed / LTS16294
- Conditions
- Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 261 participants (actual)
- Lead sponsor
- Bioverativ, a Sanofi companyIndustry (pharma, biotech, devices)
- Study start
- 23 Feb 2021 (actual)
- Primary completion
- 15 Jan 2027 (estimated)
- Study completion
- 15 Jan 2027 (estimated)
- First posted
- 25 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary Objective:
- 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
Detailed description
Participants will receive BIVV001 once weekly for a total of at least 100 exposure days to BIVV001 (including exposure during a BIVV001 parent study, if applicable). 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- It is a 3-arm study with single intervention
| Arm | Type | Receives |
|---|---|---|
| Arm A: Previously treated in BIVV001 study | Experimental | Drug: efanesoctocog alfa (BIVV001)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. |
| Arm B: Newly initiated (China Only) in BIVV001 | Experimental | Drug: efanesoctocog alfa (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. |
| Arm C: Newly initiated in BIVV001 with planned major surgery | Experimental | Drug: efanesoctocog alfa (BIVV001)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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- For participants rolling over into Arm A
- For participants new to BIVV001 (Arm B and C)
Key exclusion criteria (16 listed)
- For participants rolling over into Arm A
- For participants new to BIVV001 (Arm B and Arm C)
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| efanesoctocog alfa (BIVV001)Pharmaceutical form:Solution for Injection Route of administration: Intravenous | Drug | Arm A: Previously treated in BIVV001 study, Arm B: Newly initiated (China Only) in BIVV001, Arm C: Newly initiated in BIVV001 with planned major surgery |
Primary outcome
Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII])
Time frame: Baseline to month 48
The number of participants with the occurrence of inhibitor development (neuatralizing antibodies detected against factor VIII [FVIII]) as determined via the Nijmegen modified Bethesda assay.
33 secondary outcomes
Annual bleeding rate (ABR)
Time frame: Baseline to month 48
Annualized bleeding rate (ABR) by type of bleed
Time frame: Baseline to month 48
Annualized bleeding rate (ABR) by location
Time frame: Baseline to month 48
Percentage of patients who maintain factor VIII (FVIII) above prespecified activity levels
Time frame: Baseline to month 48
Number of injections and dose of BIVV0001 to treat a bleeding episode
Time frame: Month 48
Percentage of bleeding episode treated with a single injection of BIVV001
Time frame: Month 48
Assessment of response to BIVV001 treatment of individual bleeding episodes
Time frame: Baseline to month 48
Physician's global assessment (PGA) of participants response to BIVV001
Time frame: Baseline to month 48
Total annualized BIVV001 consumption
Time frame: Baseline to month 48
Annualized joint bleeding rate (AJBR)
Time frame: Baseline to month 48
Target joint resolution
Time frame: Month 48
Change from baseline in Hemophilia Joint Health Score (HJHS)
Time frame: Baseline to month 48
Change from baseline in PROMIS-SF Physical Function
Time frame: Baseline to month 48
Change from baseline in Haem-A-QoL total score and physical health score
Time frame: Baseline to month 48
Change from baselin in Haemo-QoL total score and physical health score
Time frame: Baseline to month 48
Number of participants with adverse events (AEs) and serious adverse events (SAEs)
Time frame: Baseline to month 48
Number of participants with the occurrence of embolic and thrombotic events
Time frame: Baseline to month 48
PK parameter: Maximum activity (Cmax)
Time frame: Baseline to week 52
PK parameter: Elimination half-life (t1/2)
Time frame: Baseline to week 26
PK parameter: Total clearance (CL)
Time frame: Baseline to week 26
PK parameter: Total clearance at steady state (CLss)
Time frame: Baseline to week 26
PK parameter: Accumulation index (AI)
Time frame: Baseline to week 26
PK parameter: Area under the activity time curve (AUC)
Time frame: Baseline to week 26
PK parameter: Volume of distribution at steady state (Vss)
Time frame: Baseline to week 26
PK parameter: Mean residence time (MRT)
Time frame: Baseline to week 26
PK parameter: Incremental recovery (IR)
Time frame: Baseline to week 52
PK parameter: Trough activity (Ctrough)
Time frame: Baseline to week 52
PK parameter: Time above FVIII activity levels
Time frame: Baseline to week 26
Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment
Time frame: Baseline to month 48
Number of injections and dose to maintain hemostasis during perioperative period for major surgery
Time frame: Baseline to month 48
Total BIVV001 consumption during perioperative period for major surgery
Time frame: Baseline to month 48
Number and type of blood component transfusions used during perioperative period for major surgery
Time frame: Baseline to month 48
Estimated blood loss during perioperative period for major surgery
Time frame: Baseline to month 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2026.
- Derived from this study: PubMed 39585605Quintilla 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.
- LTS16294 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
85 sites in 23 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing efanesoctocog alfa (BIVV001) in people with Hemophilia A, run by Bioverativ, a Sanofi company at 85 sites in 23 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 28 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 85 sites in United States, China, Japan, France, Taiwan and 18 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII]) (time frame: Baseline to month 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04644575, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jul 2026.