A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A
Observational Study on the Use of Medication for Hemophilia A
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
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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."
This study investigates the real-world effectiveness, safety, and treatment usage of an investigational medication in patients with Hemophilia A. Hemophilia A is a genetic disorder where blood does not clot properly due to a lack of clotting factor VIII, leading to excessive bleeding. The purpose of this study is to observe how patients respond to this investigational medication when it is used as part of their standard care in the US and Japan.
Participants will be enrolled and their data collected during routine medical visits. These visits will occur annually or semi-annually for up to five years. The study does not provide the investigational medication; instead, it will collect information on its effectiveness and safety as part of the patients' usual treatment.
- Who can participate: Individuals diagnosed with Hemophilia A who have started treatment with the medication within one month prior to enrollment may participate. Informed consent is required, and participants must not have other known bleeding disorders or be involved in other drug trials.
- Study details: Participants will continue their usual care with the medication, and data will be collected during routine medical visits. The study will track the medication's effectiveness and safety over time.
- Study Timelines: The study will last 5 years.
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