Hemophilia ANCT05911763
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A
An observational study of Hemophilia A, run by Sanofi at 28 sites in United States.
Trial summary
This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan. 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
- How many people
- 200 participants
- Where
- 28 sitesUnited States
- Study dates
- 30 Jun 2023to 17 Sep 2030
- Phase
- Observational
- 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
Study population: Approximately 200 patients from sites in the US and Japan will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each of these two countries. To generate data for the broad real-world population of individuals with hemophilia A, eligible patients will include all ages, sexes and hemophilia A severities, whether on prophylactic or on-demand therapy or if efanesoctocog alfa is taken perioperatively.
Who may be able to join
- Have a diagnosis of hemophilia A
- Patients starting efanesoctocog alfa treatment as per standard of care no more than one month prior to the enrollment date, for either on demand or prophylaxis. Patients starting efanesoctocog alfa treatment for a surgery event may also be enrolled only if the treatment is prescribed at enrollment.
- Physician's decision to treat the patient with efanesoctocog alfa is made prior to and independently of participation in the study.
- Signed and dated informed consent provided by the patient, or by the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric patients according to local regulations.
Who cannot join
- Diagnosed with other known bleeding disorder
- Participation in an investigational medicinal product trial at enrollment visit, or intake of an Investigational Medicinal Product within 3 months prior to inclusion in this study
- Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥0.60 BU/mL
- "The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial."
Trial locations and contacts
28 sites in 1 country.
28 sites in 1 country
United States28 sites
University of Michigan Hospital - 1500 E Medical Center Dr- Site Number : 8400023
Ann Arbor, Michigan, 48109-5000
University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019
Aurora, Colorado, 80045-7202
~Massachusetts General Hospital- Site Number : 8400017
Boston, Massachusetts, 02114
Dana Farber and Boston Children's Hospital- Site Number : 8400031
Boston, Massachusetts, 02215-5418
Rush University Medical Center -1725 W Harrison St- Site Number : 8400014
Chicago, Illinois, 60612-3863
Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400006
Cincinnati, Ohio, 45229-3026
University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020
Dallas, Texas, 75390-7202
University of Texas Southwestern Medical Center- Site Number : 8400011
Dallas, Texas, 75390-8565
William A. Shands Hospital at the University of Florida- Site Number : 8400032
Gainesville, Florida, 32610-3003
Banner MD Anderson Cancer Center- Site Number : 8400008
Gilbert, Arizona, 85234-2165
Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029
Houston, Texas, 77030
Indiana Hemophilia and Thrombosis Center- Site Number : 8400013
Indianapolis, Indiana, 46260-1920
University Of Iowa Hospitals And Clinics- Site Number : 8400026
Iowa City, Iowa, 52242-1009
Children's Specialty Center - Las Vegas- Site Number : 8400007
Las Vegas, Nevada, 89109
Loma Linda University- Site Number : 8400015
Loma Linda, California, 92354-3450
Orthopedic Institute for Children- Site Number : 8400004
Los Angeles, California, 90007
Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016
Madison, Mississippi, 39110-6115
Blood Center of Wisconnsin- Site Number : 8400027
Milwaukee, Wisconsin, 53233-2121
M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010
Minneapolis, Minnesota, 55455-4800
Vanderbilt University Medical Center- Site Number : 8400003
Nashville, Tennessee, 37232-0011
Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021
New York, New York, 10065-4870
University of Nebraska Medical Center - 985400 Nebraska Medical Center- Site Number : 8400009
Omaha, Nebraska, 68114-4113
The Center for Inherited Blood Disorders- Site Number : 8400001
Orange, California, 92868
The Bleeding and Clotting Disorders Institute- Site Number : 8400005
Peoria, Illinois, 61615
Hemophilia Center of Western Pennsylvania- Site Number : 8400012
Pittsburgh, Pennsylvania, 15213-4306
Mayo Clinic - PPDS- Site Number : 8400035
Rochester, Minnesota, 55905-0001
Washington Institute for Coagulation- Site Number : 8400022
Seattle, Washington, 98101-3932
Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025
St. Petersburg, Florida, 33701-4804
About this study
Official title: Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A
This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan.
Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.
- 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 BIVV001Drug
Also called ALTUVIIIO
Given per investigator's discretion
| Study group | Role | What they receive |
|---|---|---|
| Cohort A (Prophylactic treatment) | All participants on efanesoctocog alfa prophylactic treatment fulfilling the overall study inclusion/exclusion criteria. The prophylactic cohort will include the following sub-cohorts:
Sub-cohort A1 (Joint imaging): Participants with severe hemophilia A and joint imaging by Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) or Joint Tissue Activity and Damage Exam (JADE) protocol performed within 6 months of initiating treatment with efanesoctocog alfa or within 3 months after initiating treatment with efanesoctocog alfa available.
Sub-cohort A2 (Children with no prior joint damage):Participants with severe hemophilia A who have no prior joint damage Receives: Drug: Efanesoctocog Alfa BIVV001 | |
| Cohort B (On-Demand treatment) | Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria Receives: Drug: Efanesoctocog Alfa BIVV001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Measured: Up to 5 years
Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Measured: Up to 5 years
Number of Target joint development, resolution and/or recurrence
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic coho
Measured: Up to 5 years
Secondary outcomes (34)
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
HJHS total/domain scores will be reported for prophylactic cohorts (A1 ad A2)
Measured: At 1, 2, 3, 4, 5 years
Annual Bleeding Rate (ABR) by type for treated bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
ABR by type all (treated and untreated) bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
ABR by location for treated bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
ABR by location for all (treated and untreated) bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
ABR for all bleeding episodes (including untreated bleeding episodes)
Data will be reported for prophylactic cohorts (A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
Percentage of patients with zero joint bleeds
Data will be reported for prophylactic cohorts (A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.
Data will be reported for prophylactic cohorts (A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
Annualized injection frequency per participant (assessed by prescription) during the follow- up period
Data will be reported for prophylactic cohorts (A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
Treatment adherence (%) as judged by the physician during the follow-up period
Data will be reported for prophylactic cohorts (A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode.
Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode.
Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
Measured: At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode
Data will be reported for On-demand cohort
Measured: At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode
Data will be reported for On-demand cohort
Measured: At 1, 2, 3, 4, 5 years
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.
Data will be reported for On-demand cohort
Measured: At 1, 2, 3, 4, 5 years
Occurrence of a change in treatment regimen (on-demand to prophylactic or prophylactic to on-demand) at baseline and follow-up.
Data will be reported for On-demand cohort
Measured: At 1, 2, 3, 4, 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores
HEAD-US total/domain scores will be reported for joint imaging sub cohort
Measured: At 1, 2, 3, 4 and 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS)
Data will be reported for consenting participants from the joint imaging sub-cohort
Measured: At 1, 2, 3, 4 and 5 years
Change from baseline in synovial hypertrophy by change in mm thickness AND/OR HEAD-US synovitis domain, AND/OR by JADE MSKUS synovial hypertrophy +/- power doppler signal
Data will be reported from the consenting participants from the joint imaging sub-cohort
Measured: At 6 months, 1, 2, 3, 4 and 5 years
Occurrence of adverse events (AEs) and serious adverse events (SAEs)
The safety and tolerability of efanesoctocog alfa over 5 years evaluated in all participants
Measured: Over 5 year period
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay.
The safety and tolerability of efanesoctocog alfa over 5 years assessed in all participants
Measured: Over 5 year period
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) pain intensity 3a questionnaire (adults)
Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
Measured: At 3 months, 6 months, 1, 2, 3, 4 and 5 years
Change from baseline in Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) 3.0 (boys ≤18 years old) questionnaire (including Parent Proxy)
Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
Measured: At 1, 2, 3, 4 and 5 years
Change from baseline in the occurrence of inpatient and outpatient visits, and length of hospital stay related to hemophilia A
Healthcare resource use (HCRU) in efanesoctocog alfa related to hemophilia A over a 5-year period
Measured: At 1, 2, 3, 4 and 5 years
Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa.
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Measured: Over a 5 year period
Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Measured: Over 5 year period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Measured: Over 5 year period
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Measured: Over 5 year period
Number of blood component transfusions used during perioperative period for surgery
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Measured: Over 5 year Period
Type of blood component transfusions used during perioperative period for surgery
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Measured: Over 5 year period
Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Measured: Over 5 year period
Number of transfusions required for surgery (intraoperative and post-operative period)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Measured: Over 5 year period
Duration of hospitalization (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period.
Measured: Over 5 years period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period for On-demand Cohort
Measured: Over a 5 year period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2023
- Study start30 Jun 2023
- Primary completion6 Jun 2028 (estimated)
- Study completion17 Sep 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05911763
- Sponsor's protocol ID
- OBS17523
- Other IDs
- U1111-1281-8840
- Status last verified
- Sep 2026
- First submitted
- 12 May 2023
- Last updated
- 29 Sep 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
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A
- ClinicalTrials.gov ID
- NCT05911763
- Acronym / sponsor ID
- OBS17523
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 30 Jun 2023 (actual)
- Primary completion
- 6 Jun 2028 (estimated)
- Study completion
- 17 Sep 2030 (estimated)
- First posted
- 22 Jun 2023 (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.
This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan.
Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort A (Prophylactic treatment) | – | Drug: Efanesoctocog Alfa BIVV001All participants on efanesoctocog alfa prophylactic treatment fulfilling the overall study inclusion/exclusion criteria. The prophylactic cohort will include the following sub-cohorts: Sub-cohort A1 (Joint imaging): Participants with severe hemophilia A and joint imaging by Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) or Joint Tissue Activity and Damage Exam (JADE) protocol performed within 6 months of initiating treatment with efanesoctocog alfa or within 3 months after initiating treatment with efanesoctocog alfa available. Sub-cohort A2 (Children with no prior joint damage):Participants with severe hemophilia A who have no prior joint damage |
| Cohort B (On-Demand treatment) | – | Drug: Efanesoctocog Alfa BIVV001Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Approximately 200 patients from sites in the US and Japan will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each of these two countries. To generate data for the broad real-world population of individuals with hemophilia A, eligible patients will include all ages, sexes and hemophilia A severities, whether on prophylactic or on-demand therapy or if efanesoctocog alfa is taken perioperatively.
Key inclusion criteria (4 listed)
- Have a diagnosis of hemophilia A
- Patients starting efanesoctocog alfa treatment as per standard of care no more than one month prior to the enrollment date, for either on demand or prophylaxis. Patients starting efanesoctocog alfa treatment for a surgery event may also be enrolled only if the treatment is prescribed at enrollment.
- Physician's decision to treat the patient with efanesoctocog alfa is made prior to and independently of participation in the study.
- Signed and dated informed consent provided by the patient, or by the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric patients according to local regulations.
Key exclusion criteria (4 listed)
- Diagnosed with other known bleeding disorder
- Participation in an investigational medicinal product trial at enrollment visit, or intake of an Investigational Medicinal Product within 3 months prior to inclusion in this study
- Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥0.60 BU/mL
- "The above information is not intended to contain all considerations relevant to a 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 BIVV001Also: ALTUVIIIOGiven per investigator's discretion | Drug | Cohort A (Prophylactic treatment), Cohort B (On-Demand treatment) |
Primary outcome
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Time frame: Up to 5 years
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds
Time frame: Up to 5 years
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Number of Target joint development, resolution and/or recurrence
Time frame: Up to 5 years
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic coho
34 secondary outcomes
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
Time frame: At 1, 2, 3, 4, 5 years
Annual Bleeding Rate (ABR) by type for treated bleeds
Time frame: At 1, 2, 3, 4, 5 years
ABR by type all (treated and untreated) bleeds
Time frame: At 1, 2, 3, 4, 5 years
ABR by location for treated bleeds
Time frame: At 1, 2, 3, 4, 5 years
ABR by location for all (treated and untreated) bleeds
Time frame: At 1, 2, 3, 4, 5 years
ABR for all bleeding episodes (including untreated bleeding episodes)
Time frame: At 1, 2, 3, 4, 5 years
Percentage of patients with zero joint bleeds
Time frame: At 1, 2, 3, 4, 5 years
Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.
Time frame: At 1, 2, 3, 4, 5 years
Annualized injection frequency per participant (assessed by prescription) during the follow- up period
Time frame: At 1, 2, 3, 4, 5 years
Treatment adherence (%) as judged by the physician during the follow-up period
Time frame: At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode.
Time frame: At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode.
Time frame: At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode
Time frame: At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode
Time frame: At 1, 2, 3, 4, 5 years
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.
Time frame: At 1, 2, 3, 4, 5 years
Occurrence of a change in treatment regimen (on-demand to prophylactic or prophylactic to on-demand) at baseline and follow-up.
Time frame: At 1, 2, 3, 4, 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS)
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in synovial hypertrophy by change in mm thickness AND/OR HEAD-US synovitis domain, AND/OR by JADE MSKUS synovial hypertrophy +/- power doppler signal
Time frame: At 6 months, 1, 2, 3, 4 and 5 years
Occurrence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: Over 5 year period
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay.
Time frame: Over 5 year period
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) pain intensity 3a questionnaire (adults)
Time frame: At 3 months, 6 months, 1, 2, 3, 4 and 5 years
Change from baseline in Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) 3.0 (boys ≤18 years old) questionnaire (including Parent Proxy)
Time frame: At 1, 2, 3, 4 and 5 years
Change from baseline in the occurrence of inpatient and outpatient visits, and length of hospital stay related to hemophilia A
Time frame: At 1, 2, 3, 4 and 5 years
Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa.
Time frame: Over a 5 year period
Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor)
Time frame: Over 5 year period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Time frame: Over 5 year period
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
Time frame: Over 5 year period
Number of blood component transfusions used during perioperative period for surgery
Time frame: Over 5 year Period
Type of blood component transfusions used during perioperative period for surgery
Time frame: Over 5 year period
Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery
Time frame: Over 5 year period
Number of transfusions required for surgery (intraoperative and post-operative period)
Time frame: Over 5 year period
Duration of hospitalization (major, minor)
Time frame: Over 5 years period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Time frame: Over a 5 year period
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 29 Sep 2026.
- 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
28 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Sanofi at 28 sites in United States.
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 4 inclusion and 4 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 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States. 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.
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?
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds (time frame: Up to 5 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Sep 2026.