Factor VIII DeficiencyPhase 4SYNOVIIIUSNCT06941870
Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy
A Phase 4 study testing Efanesoctocog alfa in people with Factor VIII Deficiency, run by Sanofi at 14 sites in 4 countries.
Trial summary
The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A. The study duration for each participant is approximately 12 months. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Factor VIII Deficiency
- What is being tested?
- Efanesoctocog alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 14 sitesin 4 countries
- Study dates
- 23 Sep 2025to 25 Oct 2027
- Phase
- Phase 4
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to (≤)5 percent (%) of endogenous FVIII clotting activity) at the time of consent/assent.
- Participant is more than or equal to (≥)12 years old at the time of consent/assent.
- Participant has existing synovial hypertrophy, defined as at least 1 eligible* joint by the HEAD-US score (Synovitis score: 1 or 2) at the time of consent/assent.
- Participant has at least 1 eligible* joint without planned future major orthopedic procedures (example, arthroscopic synovectomy, radioisotopic or chemical synoviorthesis), or major orthopedic procedures in the past 3 months prior to the screening visit (Visit 1).
- An eligible joint is a joint with existing synovial hypertrophy, as defined by a HEAD-US synovitis score of 1 or 2, considering hypertrophic synovium as an indication of the presence of synovitis
- Participant has received prophylactic treatment with hemophilia prophylaxis prescriptions in the last 12 months prior to the baseline visit (Visit 2).
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- Participant is capable of understanding the written Informed Consent Form (ICF)/assent form, provides a signed and witnessed written ICF/assent form, and agrees to comply with the protocol requirements.
- If male, no contraceptive measures are required for this study.
- If female, is not pregnant or breastfeeding, and one of the following conditions applies:
- Is a women of nonchildbearing potential (WONCBP)
- Is a WOCBP and agrees to use a highly effective contraceptive method, with a failure rate of less than (<)1 percent during the study treatment period (at least until the Week 52/ End of Treatment [EoT] visit).
- A WOCBP must have a negative serum pregnancy test at the screening visit (Visit 1)
Who cannot join
- Participants are excluded from the study if any of the following criteria apply:
- Has other associated clotting disorders at the time of consent/assent.
- Is already under efanesoctocog alfa treatment.
- Has a current diagnosis of an factor VIII (FVIII) inhibitor, defined as inhibitor titer ≥0.60 BU/mL.
- Has ITI within the last 2 years prior to the baseline visit (Visit 2).
- Has been enrolled in a concurrent clinical interventional study or exposed to other investigational drug(s) within 3 months prior to screening for this study.
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- Is currently in an institution because of regulatory or legal order (that is, is a prisoner or a patient who is legally institutionalized).
- Is not suitable for participation, whatever the reason, as judged by the investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
- Is an employee or family member of the investigator or site personnel.
- Is involved in a specific situation during study implementation or the course of the study that may raise ethics considerations.
- Has hypersensitivity to efanesoctocog alfa or its components or any of its excipients that, in the opinion of the investigator, contraindicates participation in the study.
- 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
14 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
Trial Transparency email recommended (Toll free for US & Canada) contact
14 sites in 4 countries
United States5 sites, 3 recruiting
- Completed
The Ohio State University Wexner Medical Center - CarePoint East- Site Number : 8400001
Columbus, Ohio, 43203
- Recruiting
Indiana Hemophilia and Thrombosis Center- Site Number : 8400006
Indianapolis, Indiana, 46260
- Recruiting
University of Iowa- Site Number : 8400004
Iowa City, Iowa, 52242
- Completed
Orthopedic Institute for Children- Site Number : 8400009
Los Angeles, California, 90007
- Recruiting
Washington Center for Bleeding Disorders- Site Number : 8400008
Seattle, Washington, 98101-3932
Japan4 sites, 2 recruiting
- Completed
Investigational Site Number : 3920002
Hiroshima, 734-8551
- Recruiting
Investigational Site Number : 3920003
Kawasaki, Kanagawa, 216-8511
- Recruiting
Investigational Site Number : 3920001
Saitama, 330-8777
- Completed
Investigational Site Number : 3920004
Tokyo, 160-0023
Taiwan3 sites, 3 recruiting
- Recruiting
Investigational Site Number: 1580003
Kaohsiung City, 80756
- Recruiting
Investigational Site Number: 1580001
Taichung, 407
- Recruiting
Investigational Site Number: 1580002
Taipei, 114
Canada2 sites, 1 recruiting
- Recruiting
Investigational Site Number : 1240003
Hamilton, Ontario, L8N 3Z5
- Completed
Investigational Site Number : 1240004
Hamilton, Ontario, L8N 3Z5
About this study
Official title: Prospective Interventional Study of Effectiveness of Efanesoctocog Alfa Prophylaxis on Synovial Hypertrophy in Patients With Hemophilia A
The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A.
The study duration for each participant is approximately 12 months.
- Conditions
- Factor VIII Deficiency
- Being tested
- Efanesoctocog alfa
- 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 alfaDrug
Also called BIVV001, ALTUVIIIO®
Pharmaceutical form:Lyophilized powder in a sterile vial that requires reconstitution with sterile water for injection (diluent)-Route of administration:Intravenous
| Study group | Role | What they receive |
|---|---|---|
| Efanesoctocog Alfa | Experimental | Participants will receive Efanesoctocog Alfa at 50 international unit per kilogram (IU/kg) intravenous (IV) QW Receives: Drug: Efanesoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 52
Secondary outcomes (18)
Change from baseline in the distribution of joint HEAD-US synovitis score
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 52
Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 52
Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 52
Number of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 52
Percentage of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 52
Number of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 52
Percentage of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 52
Change from baseline in average HEAD-US synovitis domain score across all index joints
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 52
Change from baseline in total/ domain scores of the HEAD-US
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Measured: From Baseline up to Week 26
Change from baseline in total/domain scores of the HJHS
HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.
Measured: From Baseline up to Week 26
Change from baseline in total/domain scores of the HJHS
HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.
Measured: From Baseline up to Week 52
Change from baseline in total/ domain scores of magnetic resonance imaging (MRI)
MRI will be used to detect changes in synovial hypertrophy in index joints of participants.
Measured: From Baseline up to Week 52
Change from baseline in patient-reported outcome (PRO)
Change from baseline in PRO will be assessed by the EQ-5D 5-level (EQ-5D-5L), Patient-Reported Outcomes Measurement Information System - Short Form (PROMIS-SF) version 2.0 Pain Intensity 3a, PROMIS-SF version 1.1 Pain Interference 6a, and PROMIS-SF version 2.0 Physical Function 6b.
Measured: From Baseline up to Week 52
Patient reported treatment preference and satisfaction
Patient reported treatment preference and satisfaction will be assessed through surveys and exit interviews.
Measured: At Week 52
Change from baseline in annualized bleeding rate (ABR) (spontaneous, traumatic)
The ABR will be defined as (number of bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25.
Measured: At Week 52
Change from baseline in annualized joint bleeding rate (AjBR) (spontaneous, traumatic)
The AjBR will be defined as (number of joint bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25.
Measured: At Week 52
Change from baseline in target joint resolution, recurrence, or development
Change from baseline in target joint resolution, recurrence, or development will be assessed.
Measured: At Week 52
The occurrence of Adverse Events (AEs), all AEs leading to treatment discontinuation, Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs)
The safety and tolerability of efanesoctocog alfa evaluated in all participants
Measured: Up to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2025
- Study start23 Sep 2025
- Primary completion25 Oct 2027 (estimated)
- Study completion25 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Factor VIII DeficiencyStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Phase 3 in Taiwan, Canada and Australia, Phase 4 in US and Japan.
- Masking
- None (open label)
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06941870
- Sponsor's protocol ID
- LPS18168
- Other IDs
- U1111-1311-1701
- Acronym
- SYNOVIIIUS
- Status last verified
- Oct 2026
- First submitted
- 16 Apr 2025
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- 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