A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A
Study on Investigational 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 use of an investigational medication in patients with hemophilia A, a condition where the blood does not clot properly due to the lack of a certain protein. The purpose of this study is to observe how this medication is used in the United States and Japan as part of standard care.
Participants will have their treatment information collected during regular doctor visits over a period of up to five years. These visits may happen once or twice a year. The study will not provide any medication, and the treatment will be decided by the patient's doctor.
- Who can participate: Individuals diagnosed with hemophilia A who have started treatment with the investigational medication within one month prior to joining the study can participate. They must not have other bleeding disorders or be involved in another clinical trial.
- Study details: Participants will continue their usual treatment, with health and treatment information collected during regular doctor visits. The study does not provide any medication, and the treatment decision is made independently by the doctor.
- Study Timelines: The study will last up to 5 years.
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