Clinical trial NCT05911763: A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A
Official title: Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hemophilia A
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2023-06-22
Enrollment: 200
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

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.


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."

Primary outcomes:
measure: Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
timeFrame: Up to 5 years
measure: Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds
timeFrame: Up to 5 years
measure: Number of Target joint development, resolution and/or recurrence
timeFrame: Up to 5 years
Secondary outcomes:
measure: Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
timeFrame: At 1, 2, 3, 4, 5 years
measure: Annual Bleeding Rate (ABR) by type for treated bleeds
timeFrame: At 1, 2, 3, 4, 5 years
measure: ABR by type all (treated and untreated) bleeds
timeFrame: At 1, 2, 3, 4, 5 years
measure: ABR by location for treated bleeds
timeFrame: At 1, 2, 3, 4, 5 years
measure: ABR by location for all (treated and untreated) bleeds
timeFrame: At 1, 2, 3, 4, 5 years
measure: ABR for all bleeding episodes (including untreated bleeding episodes)
timeFrame: At 1, 2, 3, 4, 5 years
measure: Percentage of patients with zero joint bleeds
timeFrame: At 1, 2, 3, 4, 5 years
measure: Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.
timeFrame: At 1, 2, 3, 4, 5 years
measure: Annualized injection frequency per participant (assessed by prescription) during the follow- up period
timeFrame: At 1, 2, 3, 4, 5 years
measure: Treatment adherence (%) as judged by the physician during the follow-up period
timeFrame: At 1, 2, 3, 4, 5 years
measure: Number of injections of efanesoctocog alfa to treat a bleeding episode.
timeFrame: At 1, 2, 3, 4, 5 years
measure: Total dose of efanesoctocog alfa to treat a bleeding episode.
timeFrame: At 1, 2, 3, 4, 5 years
measure: Number of injections of efanesoctocog alfa to treat a bleeding episode
timeFrame: At 1, 2, 3, 4, 5 years
measure: Total dose of efanesoctocog alfa to treat a bleeding episode
timeFrame: At 1, 2, 3, 4, 5 years
measure: Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.
timeFrame: At 1, 2, 3, 4, 5 years
measure: Occurrence of a change in treatment regimen (on-demand to prophylactic or prophylactic to on-demand) at baseline and follow-up.
timeFrame: At 1, 2, 3, 4, 5 years
measure: Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS)
timeFrame: At 1, 2, 3, 4 and 5 years
measure: 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
timeFrame: At 6 months, 1, 2, 3, 4 and 5 years
measure: Occurrence of adverse events (AEs) and serious adverse events (SAEs)
timeFrame: Over 5 year period
measure: Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay.
timeFrame: Over 5 year period
measure: Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) pain intensity 3a questionnaire (adults)
timeFrame: At 3 months, 6 months, 1, 2, 3, 4 and 5 years
measure: Change from baseline in Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) 3.0 (boys ≤18 years old) questionnaire (including Parent Proxy)
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Change from baseline in the occurrence of inpatient and outpatient visits, and length of hospital stay related to hemophilia A
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa.
timeFrame: Over a 5 year period
measure: Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor)
timeFrame: Over 5 year period
measure: Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
timeFrame: Over 5 year period
measure: Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
timeFrame: Over 5 year period
measure: Number of blood component transfusions used during perioperative period for surgery
timeFrame: Over 5 year Period
measure: Type of blood component transfusions used during perioperative period for surgery
timeFrame: Over 5 year period
measure: Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery
timeFrame: Over 5 year period
measure: Number of transfusions required for surgery (intraoperative and post-operative period)
timeFrame: Over 5 year period
measure: Duration of hospitalization (major, minor)
timeFrame: Over 5 years period
measure: Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
timeFrame: Over a 5 year period
Other outcomes:
Locations:
city: Gilbert
country: United States
facility: Banner MD Anderson Cancer Center- Site Number : 8400008
state: Arizona
city: Loma Linda
country: United States
facility: Loma Linda University Health- Site Number : 8400015
state: California
city: Los Angeles
country: United States
facility: Orthopedic Institute for Children- Site Number : 8400004
state: California
city: Orange
country: United States
facility: The Center for Inherited Blood Disorders- Site Number : 8400001
state: California
city: Aurora
country: United States
facility: University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019
state: Colorado
city: Gainesville
country: United States
facility: William A. Shands Hospital at the University of Florida- Site Number : 8400032
state: Florida
city: Saint Petersburg
country: United States
facility: Johns Hopkins All Childrens Hospital Outpatient Care Center- Site Number : 8400025
state: Florida
city: Chicago
country: United States
facility: Rush University Medical Center- Site Number : 8400014
state: Illinois
city: Peoria
country: United States
facility: The Bleeding and Clotting Disorders Institute- Site Number : 8400005
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Hemophilia and Thrombosis Center- Site Number : 8400013
state: Indiana
city: Iowa City
country: United States
facility: University Of Iowa Hospitals And Clinics- Site Number : 8400026
state: Iowa
city: Boston
country: United States
facility: Massachusetts General Hospital- Site Number : 8400017
state: Massachusetts
city: Boston
country: United States
facility: Dana Farber and Boston Children's Hospital- Site Number : 8400031
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Health System - Ann Arbor- Site Number : 8400023
state: Michigan
city: Minneapolis
country: United States
facility: M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010
state: Minnesota
city: Madison
country: United States
facility: Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016
state: Mississippi
city: Omaha
country: United States
facility: University of Nebraska Medical Center- Site Number : 8400009
state: Nebraska
city: Las Vegas
country: United States
facility: Children's Specialty Center - Las Vegas- Site Number : 8400007
state: Nevada
city: New York
country: United States
facility: Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center- Site Number : 8400006
state: Ohio
city: Pittsburgh
country: United States
facility: The Hemophilia Center of Western Pennsylvania - Vitalant- Site Number : 8400012
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center- Site Number : 8400003
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020
state: Texas
city: Dallas
country: United States
facility: University of Texas - Southwestern Medical Center- Site Number : 8400011
state: Texas
city: Houston
country: United States
facility: Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029
state: Texas
city: Seattle
country: United States
facility: Washington Institute for Coagulation- Site Number : 8400022
state: Washington
city: Milwaukee
country: United States
facility: Blood Center of Wisconnsin- Site Number : 8400027
state: Wisconsin
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: