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

Launched by SANOFI · Nov 8, 2024

Keywords

ClinConnect Summary

This clinical trial is looking at a treatment called efanesoctocog alfa for people with hemophilia A, a condition where blood doesn't clot properly. The study aims to understand how effective and safe this treatment is for long-term joint health in patients. Researchers will gather information about participants' health and treatment effects over a five-year period, recording data during regular medical visits. This trial is taking place in Taiwan and includes both past health records and ongoing observations.

To be eligible for the study, participants need to be diagnosed with moderate to severe hemophilia A and have started efanesoctocog alfa treatment within the last three months. They should also be at least six years old and able to have imaging tests like MRIs. Participants will not have to undergo any extra visits or treatments specifically for the study, as their usual medical care will continue. It's important to note that individuals with other bleeding disorders or who are pregnant cannot participate. This trial will help gather valuable information to improve treatment for people with hemophilia A.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Participants with all ages and diagnosis of moderate-severe hemophilia A without current and/or at least three years of un-detectable inhibitor (\<0.6 BU)
• • Participants with moderate to severe hemophilia A as defined by FVIII level ≤ 5%
• • Participants starting efanesoctocog alfa prophylaxis treatment as per standard of care no more than three months prior to the enrollment date
• • Participants aged 6 years and older are able to undergo MRI examinations (sedation given, if necessary, and per investigator discretion)
• • Participants are able to undergo joint examinations
• • Physician's decision to treat the participant with efanesoctocog alfa is made prior to and independently of participation in the study
• • Signed and dated informed consent provided by the participant, or by the participant's legally acceptable representative for participants under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric participants according to local regulations
• Exclusion Criteria:
• • Participants with coagulation disorders other than hemophilia A
• • Participants diagnosed with other known bleeding disorder
• • Participants currently receive factor therapy and have signs of decreased response to FVIII therapy
• • Participants with a baseline Radiological Pettersson score (PS) of greater than 6 for each individual ankle
• • Enrollment in another concurrent clinical interventional study, or intake of an Investigational Medicinal Product within 3 months prior to inclusion in this study
• • Pregnant female participants
• • The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.