A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A

Description

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

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Study Overview

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Study Details

Study overview

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.

Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A

A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A

Condition
Hemophilia A
Intervention / Treatment

-

Contacts and Locations

Gilbert

Banner MD Anderson Cancer Center Site Number : 8400008, Gilbert, Arizona, United States, 85234-2165

Loma Linda

Loma Linda University Site Number : 8400015, Loma Linda, California, United States, 92354-3450

Los Angeles

Orthopedic Institute for Children Site Number : 8400004, Los Angeles, California, United States, 90007

Orange

Center for Inherited Blood Disorders (CIBD) Site Number : 8400001, Orange, California, United States, 92868

Aurora

University of Colorado Hemophilia and Thrombosis Center Site Number : 8400019, Aurora, Colorado, United States, 80045-7202

Gainesville

William A. Shands Hospital at the University of Florida Site Number : 8400032, Gainesville, Florida, United States, 32610-3003

Saint Petersburg

Johns Hopkins All Childrens Hospital Outpatient Care Center Site Number : 8400025, Saint Petersburg, Florida, United States, 33701-4804

Chicago

Rush University Medical Center -1725 W Harrison St Site Number : 8400014, Chicago, Illinois, United States, 60612-3863

Peoria

Bleeding and Clotting Disorders Institute Site Number : 8400005, Peoria, Illinois, United States, 61614-2868

Indianapolis

Indiana Hemophilia and Thrombosis Center Site Number : 8400013, Indianapolis, Indiana, United States, 46260-1920

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Sanofi,

Clinical Sciences & Operations, STUDY_DIRECTOR, Sanofi

Study Record Dates

2028-06-06