A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A
- Conditions
- Hemophilia A
- Interventions
- Drug: Efanesoctocog Alfa BIVV001
- Registration Number
- NCT05911763
- Lead Sponsor
- Sanofi
- Brief 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.
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 200
- 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.
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."
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Cohort B (On-Demand treatment) Efanesoctocog Alfa BIVV001 Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria Cohort A (Prophylactic treatment) Efanesoctocog Alfa BIVV001 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
- Primary Outcome Measures
Name Time Method Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds 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 treated bleeds 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 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
- Secondary Outcome Measures
Name Time Method Annual Bleeding Rate (ABR) by type for treated bleeds At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (A1 and A2)
ABR by type all (treated and untreated) bleeds At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (A1 and A2)
ABR by location for treated bleeds At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (A1 and A2)
ABR for all bleeding episodes (including untreated bleeding episodes) At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (A1 and A2)
Percentage of patients with zero joint bleeds At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (A1 and A2)
Number of injections of efanesoctocog alfa to treat a bleeding episode At 1, 2, 3, 4, 5 years Data will be reported for On-demand cohort
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa. At 1, 2, 3, 4, 5 years Data will be reported for On-demand cohort
Occurrence of a change in treatment regimen (on-demand to prophylactic or prophylactic to on-demand) at baseline and follow-up. At 1, 2, 3, 4, 5 years Data will be reported for On-demand cohort
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores At 1, 2, 3, 4, 5 years HJHS total/domain scores will be reported for prophylactic cohorts (A1 ad A2)
ABR by location for all (treated and untreated) bleeds At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (A1 and A2)
Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period. At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (A1 and A2)
Annualized injection frequency per participant (assessed by prescription) during the follow- up period At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (A1 and A2)
Treatment adherence (%) as judged by the physician during the follow-up period At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (A1 and A2)
Number of injections of efanesoctocog alfa to treat a bleeding episode. At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
Total dose of efanesoctocog alfa to treat a bleeding episode. At 1, 2, 3, 4, 5 years Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
Total dose of efanesoctocog alfa to treat a bleeding episode At 1, 2, 3, 4, 5 years Data will be reported for On-demand cohort
Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores At 1, 2, 3, 4 and 5 years HEAD-US total/domain scores will be reported for joint imaging sub cohort
Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS) At 1, 2, 3, 4 and 5 years Data will be reported for consenting participants from the joint imaging sub-cohort
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 At 6 months, 1, 2, 3, 4 and 5 years Data will be reported from the consenting participants from the joint imaging sub-cohort
Change from baseline in the occurrence of inpatient and outpatient visits, and length of hospital stay related to hemophilia A At 1, 2, 3, 4 and 5 years Healthcare resource use (HCRU) in efanesoctocog alfa related to hemophilia A over a 5-year period
Occurrence of adverse events (AEs) and serious adverse events (SAEs) Over 5 year period The safety and tolerability of efanesoctocog alfa over 5 years evaluated in all participants
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) pain intensity 3a questionnaire (adults) At 3 months, 6 months, 1, 2, 3, 4 and 5 years Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
Change from baseline in Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) 3.0 (boys ≤18 years old) questionnaire (including Parent Proxy) At 1, 2, 3, 4 and 5 years Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay. Over 5 year period The safety and tolerability of efanesoctocog alfa over 5 years assessed in all participants
Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa. Over a 5 year period Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor) Over 5 year period Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor) Over a 5 year period Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period for On-demand Cohort
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor) Over 5 year period Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Number of blood component transfusions used during perioperative period for surgery Over 5 year Period Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Duration of hospitalization (major, minor) Over 5 years period Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period.
Type of blood component transfusions used during perioperative period for surgery Over 5 year period Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery Over 5 year period Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Number of transfusions required for surgery (intraoperative and post-operative period) Over 5 year period Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Trial Locations
- Locations (27)
Banner MD Anderson Cancer Center- Site Number : 8400008
🇺🇸Gilbert, Arizona, United States
Loma Linda University Health- Site Number : 8400015
🇺🇸Loma Linda, California, United States
Orthopedic Institute for Children- Site Number : 8400004
🇺🇸Los Angeles, California, United States
The Center for Inherited Blood Disorders- Site Number : 8400001
🇺🇸Orange, California, United States
University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019
🇺🇸Aurora, Colorado, United States
William A. Shands Hospital at the University of Florida- Site Number : 8400032
🇺🇸Gainesville, Florida, United States
Johns Hopkins All Childrens Hospital Outpatient Care Center- Site Number : 8400025
🇺🇸Saint Petersburg, Florida, United States
Rush University Medical Center- Site Number : 8400014
🇺🇸Chicago, Illinois, United States
The Bleeding and Clotting Disorders Institute- Site Number : 8400005
🇺🇸Peoria, Illinois, United States
Indiana Hemophilia and Thrombosis Center- Site Number : 8400013
🇺🇸Indianapolis, Indiana, United States
University Of Iowa Hospitals And Clinics- Site Number : 8400026
🇺🇸Iowa City, Iowa, United States
Massachusetts General Hospital- Site Number : 8400017
🇺🇸Boston, Massachusetts, United States
Dana Farber and Boston Children's Hospital- Site Number : 8400031
🇺🇸Boston, Massachusetts, United States
University of Michigan Health System - Ann Arbor- Site Number : 8400023
🇺🇸Ann Arbor, Michigan, United States
M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010
🇺🇸Minneapolis, Minnesota, United States
Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016
🇺🇸Madison, Mississippi, United States
University of Nebraska Medical Center- Site Number : 8400009
🇺🇸Omaha, Nebraska, United States
Children's Specialty Center - Las Vegas- Site Number : 8400007
🇺🇸Las Vegas, Nevada, United States
Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021
🇺🇸New York, New York, United States
Cincinnati Children's Hospital Medical Center- Site Number : 8400006
🇺🇸Cincinnati, Ohio, United States
The Hemophilia Center of Western Pennsylvania - Vitalant- Site Number : 8400012
🇺🇸Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical Center- Site Number : 8400003
🇺🇸Nashville, Tennessee, United States
University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020
🇺🇸Dallas, Texas, United States
University of Texas - Southwestern Medical Center- Site Number : 8400011
🇺🇸Dallas, Texas, United States
Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029
🇺🇸Houston, Texas, United States
Washington Institute for Coagulation- Site Number : 8400022
🇺🇸Seattle, Washington, United States
Blood Center of Wisconnsin- Site Number : 8400027
🇺🇸Milwaukee, Wisconsin, United States