Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A
Overview
- Phase
- N/A
- Intervention
- Efanesoctocog Alfa BIVV001
- Conditions
- Hemophilia A
- Sponsor
- Sanofi
- Enrollment
- 200
- Locations
- 28
- Primary Endpoint
- Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds
- Status
- Active, Not Recruiting
- Last Updated
- 7 months ago
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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.
Exclusion Criteria
- •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."
Arms & Interventions
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
Intervention: Efanesoctocog Alfa BIVV001
Cohort B (On-Demand treatment)
Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria
Intervention: Efanesoctocog Alfa BIVV001
Outcomes
Primary Outcomes
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
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
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
Secondary Outcomes
- Annual Bleeding Rate (ABR) by type for treated bleeds(At 1, 2, 3, 4, 5 years)
- ABR by type all (treated and untreated) bleeds(At 1, 2, 3, 4, 5 years)
- ABR by location for treated bleeds(At 1, 2, 3, 4, 5 years)
- ABR for all bleeding episodes (including untreated bleeding episodes)(At 1, 2, 3, 4, 5 years)
- Percentage of patients with zero joint bleeds(At 1, 2, 3, 4, 5 years)
- Number of injections of efanesoctocog alfa to treat a bleeding episode(At 1, 2, 3, 4, 5 years)
- Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.(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.(At 1, 2, 3, 4, 5 years)
- Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores(At 1, 2, 3, 4, 5 years)
- ABR by location for all (treated and untreated) bleeds(At 1, 2, 3, 4, 5 years)
- Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.(At 1, 2, 3, 4, 5 years)
- Annualized injection frequency per participant (assessed by prescription) during the follow- up period(At 1, 2, 3, 4, 5 years)
- Treatment adherence (%) as judged by the physician during the follow-up period(At 1, 2, 3, 4, 5 years)
- Number of injections of efanesoctocog alfa to treat a bleeding episode.(At 1, 2, 3, 4, 5 years)
- Total dose of efanesoctocog alfa to treat a bleeding episode.(At 1, 2, 3, 4, 5 years)
- Total dose of efanesoctocog alfa to treat a bleeding episode(At 1, 2, 3, 4, 5 years)
- Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores(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)(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(At 6 months, 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(At 1, 2, 3, 4 and 5 years)
- Occurrence of adverse events (AEs) and serious adverse events (SAEs)(Over 5 year period)
- 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)
- 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)
- Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay.(Over 5 year period)
- Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa.(Over a 5 year period)
- Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor)(Over 5 year period)
- Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)(Over a 5 year period)
- Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)(Over 5 year period)
- Number of blood component transfusions used during perioperative period for surgery(Over 5 year Period)
- Duration of hospitalization (major, minor)(Over 5 years period)
- Type of blood component transfusions used during perioperative period for surgery(Over 5 year period)
- Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery(Over 5 year period)
- Number of transfusions required for surgery (intraoperative and post-operative period)(Over 5 year period)