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

Investigating the Impact of an Investigational Medication on Joint Health in Hemophilia A

Recruiting
All
Phase N/A
200 participants needed
9 Locations

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."

Updated on 08 Mar 2024. Study ID: NCT05911763

This study investigates the impact of an investigational medication on long-term joint health in participants with Hemophilia A. Hemophilia A is a genetic disorder where blood does not clot properly due to a deficiency in a clotting factor called factor VIII. This can lead to excessive bleeding and joint damage.

Participants in this observational study will not receive any study medication. Instead, data will be collected during routine visits over a period of up to five years to assess the effectiveness, safety, and usage of the investigational medication. An observational study is a type of research study where data is collected by observing participants receiving routine care, without changing their treatment, which may focus on people using specific medications or having certain conditions to better understand how treatments work.

  • Who can participate: Individuals diagnosed with Hemophilia A who are starting treatment with the investigational medication as part of their standard care may be eligible. Participants should not be part of any other investigational medicinal product trial and must not have a current diagnosis of a factor VIII inhibitor.
  • Study details: Participants will attend routine visits where data on the investigational medication's effectiveness and safety will be collected. No study medication is provided, and participation involves no changes to routine care.
  • Study timelines: The study will last up to 5 years.

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