This study is currently not recruiting participants.

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

Evaluating Long-term Joint Health in Hemophilia A with Study Treatment

Not Recruiting
All
Phase N/A
200 participants needed

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.

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Hemophilia A
  • Gender: All

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."

The purpose of this study is to investigate how an investigational medication affects joint health over a long period in individuals with hemophilia A.

This study evaluates the safety and effectiveness of the investigational medication when used as part of standard care. It will collect information on how the medication is used and its impact on patients' health.

  • Who can participate: Participants must have hemophilia A and be starting treatment with the investigational medication no more than one month before enrolling. They must not have other known bleeding disorders or a current diagnosis of a FVIII inhibitor.
  • Study details: Participants' medication use is part of their standard care. No study medication is provided by the study.
  • Study timelines and visits: The study will last up to 5 years. The study requires annual/semi-annual visits.
Updated on 02 Oct 2025. Study ID: NCT05911763