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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1281-8840 | Registry Identifier | ICTRP |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
| |
| Cohort B (On-Demand treatment) | Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Efanesoctocog Alfa BIVV001 | Drug | Given per investigator's discretion |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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 | 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 | Up to 5 years |
| Measure | Description | Time Frame |
|---|---|---|
| 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) | At 1, 2, 3, 4, 5 years |
| Annual Bleeding Rate (ABR) by type for treated bleeds |
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Inclusion Criteria:
Exclusion Criteria:
Diagnosed with other known bleeding disorder
"The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial."
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Banner MD Anderson Cancer Center- Site Number : 8400008 | Gilbert | Arizona | 85234-2165 | United States | ||
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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Data will be reported for prophylactic cohorts (A1 and A2)
| 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) | At 1, 2, 3, 4, 5 years |
| ABR by location for treated bleeds | Data will be reported for prophylactic cohorts (A1 and A2) | 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) | 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) | At 1, 2, 3, 4, 5 years |
| Percentage of patients with zero joint bleeds | Data will be reported for prophylactic cohorts (A1 and A2) | 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) | 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) | 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) | 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) | 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) | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | Over 5 year period |
| Duration of hospitalization (major, minor) | Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period. | 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 | Over a 5 year period |
| Loma Linda University- Site Number : 8400015 |
| Loma Linda |
| California |
| 92354-3450 |
| United States |
| Orthopedic Institute for Children- Site Number : 8400004 | Los Angeles | California | 90007 | United States |
| The Center for Inherited Blood Disorders- Site Number : 8400001 | Orange | California | 92868 | United States |
| University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019 | Aurora | Colorado | 80045-7202 | United States |
| William A. Shands Hospital at the University of Florida- Site Number : 8400032 | Gainesville | Florida | 32610-3003 | United States |
| Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025 | St. Petersburg | Florida | 33701-4804 | United States |
| Rush University Medical Center -1725 W Harrison St- Site Number : 8400014 | Chicago | Illinois | 60612-3863 | United States |
| The Bleeding and Clotting Disorders Institute- Site Number : 8400005 | Peoria | Illinois | 61615 | United States |
| Indiana Hemophilia and Thrombosis Center- Site Number : 8400013 | Indianapolis | Indiana | 46260-1920 | United States |
| University Of Iowa Hospitals And Clinics- Site Number : 8400026 | Iowa City | Iowa | 52242-1009 | United States |
| ~Massachusetts General Hospital- Site Number : 8400017 | Boston | Massachusetts | 02114 | United States |
| Dana Farber and Boston Children's Hospital- Site Number : 8400031 | Boston | Massachusetts | 02215-5418 | United States |
| University of Michigan Hospital - 1500 E Medical Center Dr- Site Number : 8400023 | Ann Arbor | Michigan | 48109-5000 | United States |
| M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010 | Minneapolis | Minnesota | 55455-4800 | United States |
| Mayo Clinic - PPDS- Site Number : 8400035 | Rochester | Minnesota | 55905-0001 | United States |
| Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016 | Madison | Mississippi | 39110-6115 | United States |
| University of Nebraska Medical Center - 985400 Nebraska Medical Center- Site Number : 8400009 | Omaha | Nebraska | 68114-4113 | United States |
| Children's Specialty Center - Las Vegas- Site Number : 8400007 | Las Vegas | Nevada | 89109 | United States |
| Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021 | New York | New York | 10065-4870 | United States |
| Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400006 | Cincinnati | Ohio | 45229-3026 | United States |
| Hemophilia Center of Western Pennsylvania- Site Number : 8400012 | Pittsburgh | Pennsylvania | 15213-4306 | United States |
| Vanderbilt University Medical Center- Site Number : 8400003 | Nashville | Tennessee | 37232-0011 | United States |
| University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020 | Dallas | Texas | 75390-7202 | United States |
| University of Texas Southwestern Medical Center- Site Number : 8400011 | Dallas | Texas | 75390-8565 | United States |
| Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029 | Houston | Texas | 77030 | United States |
| Washington Institute for Coagulation- Site Number : 8400022 | Seattle | Washington | 98101-3932 | United States |
| Blood Center of Wisconnsin- Site Number : 8400027 | Milwaukee | Wisconsin | 53233-2121 | United States |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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