A Phase 3 interventional study of efanesoctocog alfa (BIVV001) in Hemophilia A, sponsored by Bioverativ, a Sanofi company. Active, not recruiting at 85 sites in 23 countries. Per ClinicalTrials.gov, last updated 2026-07-28.
Sponsored by Bioverativ, a Sanofi company (part of Sanofi) · Phase 3, Interventional, and Treatment
Among 11 studies testing efanesoctocog alfa in Hemophilia A — 1 open to participants now, 5 under way, 2 of them phase 3. efanesoctocog alfa · Hemophilia A.
Among the 11 studies testing efanesoctocog alfa in Hemophilia A, this one has the largest enrollment, is one of 3 that started in 2021, is one of 5 in phase 3, and was the 3rd registered.
844 studies on the registry are indexed under Hemophilia A; 136 are open to participants now.
Browse Hemophilia A studies →1 of the 11 studies testing efanesoctocog alfa in Hemophilia A is open now, in 5 countries. Recruiting first, the newest first:
Sanofi · Sites in United States, Australia, Canada and 2 more countries
Ages 12+
By country, each count listing its studies:
efanesoctocog alfa: first FDA approval 2023 (ALTUVIIIO), first EMA approval 2024. What it is approved for, and how studies give it, is on its page →
Of the 86 Hemophilia A studies recruiting now, 51 (59%) set no upper age limit and 30 (35%) stop at 75 or younger; 54 take children under 18; 10 take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
11 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 3 · Phase 2 1 · Phase 3 5 · Phase 4 3
First in 2017; this study’s year is marked. 3 started in 2021.
This study's enrollment of 261 is above the median of 30 across 499 interventional studies indexed under Hemophilia A.
Median enrollment 74 across the 11 interventional studies. Of the 6 that completed or were terminated, 3 (50%) have results posted.
This tab as a report to print or save as PDF → With an account.
11 of 11 have an industry lead sponsor. The 3 most frequent:
This study is registered under Bioverativ, a Sanofi company, one of the names counted as Sanofi. Under that name alone:
Bioverativ, a Sanofi company is the lead sponsor of 16 studies on the registry; none are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 7 (58%) have results posted.
29 studies in Hemophilia A by Sanofi: 4 open now, 8 under way, 3 of them phase 3. Of 21 finished, 14 (67%) have results posted.
Among the 29 studies in Hemophilia A by Sanofi, this one is the 2nd largest by enrollment of the 24 interventional ones that give a count, is one of 3 that started in 2021, is one of 14 in phase 3, and was the 19th registered.
Sanofi’s Hemophilia A studies by the year they started, since 2009.
4 of the studies testing efanesoctocog alfa in Hemophilia A that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04644575Phase 3Bioverativ, a Sanofi companyLong-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A | 2027-01-15 |
| NCT06716814Phase 3Swedish Orphan BiovitrumStudy to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa | 2027-03 |
| NCT06752850Swedish Orphan BiovitrumA Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis | 2026-11-17 |
| NCT06941870SanofiEfanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy | 2027-10-25 |
Add all 4 to your calendar (.ics) → With an account.
Among the 11 studies testing efanesoctocog alfa in Hemophilia A, from their registry updates:
Nothing recorded among them since 2026-09-25.
Among Sanofi’s 29 in Hemophilia A:
This tab as a report to print or save as PDF → With an account.
Primary Objective:
- To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A
Secondary Objectives:
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
For participants rolling over into Arm A
For participants new to BIVV001 (Arm B and C)
Exclusion criteria:
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.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CD4 count
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
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.
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.
Drug: efanesoctocog alfa (BIVV001)
Pharmaceutical form:Solution for Injection Route of administration: Intravenous
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.
Time frame: Baseline to month 48
Annual bleeding rate (ABR)
Annualized bleeding rate (ABR) for treated bleeding episodes and all bleeding episodes (including untreated bleeds).
Time frame: 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.
Time frame: 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.
Time frame: 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.
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
Assessment of response to BIVV001 treatment of individual bleeding episodes based on the International Society on Thrombosis and Haemostasis (ISTH) 4-point response scale
Time frame: 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 .
Time frame: Baseline to month 48
Total annualized BIVV001 consumption
Total annualized BIVV001 consumption per participant during prophylaxis treatment
Time frame: Baseline to month 48
Annualized joint bleeding rate (AJBR)
Time frame: Baseline to month 48
Target joint resolution
Target joint development, resolution and maintenance of target joint resolution based on ISTH criteria.
Time frame: 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)
Time frame: 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).)
Time frame: 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.
Time frame: 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.
Time frame: 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).
Time frame: Baseline to month 48
Number of participants with the occurrence of embolic and thrombotic events
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
Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment on the ISTH 4 point response for surgical procedures scale.
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
Plan to share: 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
Started Feb 2021 (actual). Primary completion is estimated for Jan 2027, 70 months after the start.
Of 89 industry-sponsored phase 3 interventional studies under Hemophilia A, half reached primary completion within 32 months of starting (the median), counting those still running; a quarter within 18 and three quarters within 56. This study's planned 70 months is above that.
Counting every sponsor, half of 91 studies reached it within 33 months; a quarter within 20 and three quarters within 56.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Efanesoctocog alfa
Bioverativ, a Sanofi company →