Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa

Launched by SWEDISH ORPHAN BIOVITRUM · Nov 29, 2024

Keywords

ClinConnect Summary

This clinical trial is focused on providing patients with severe Haemophilia A continued access to a treatment called efanesoctocog alfa. It is designed for patients aged 6 years and older who have already participated in earlier studies involving this treatment. The goal is to ensure that these patients can keep benefiting from efanesoctocog alfa while researchers continue to monitor its safety and effectiveness until it becomes available for general use in their countries or until March 2027.

To be eligible for this trial, participants must have completed one of the previous studies and be experiencing positive results from the efanesoctocog alfa treatment. They also need to be able to give consent, or have a parent or guardian do so if they are under 18. Throughout the study, patients will have visits approximately every 13 weeks, either in person or via phone, and will need to keep a diary to track their treatment experience. It’s important to note that individuals with certain health issues, such as a specific blood test result indicating inhibitors, or those participating in other clinical studies, may not be eligible to join.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Capable of giving signed informed consent. Parents or legally designated representatives' consent is required for patients who are below 18 years of age or unable to give consent. Patients who are below 18 years of age may provide assent in addition to the parents'/legally designated representatives' consent, if appropriate.
• • Must have completed one of the required parent studies: Sobi.BIVV001-001, Sobi.BIVV001-003, or LTS16294, and be receiving a clinical benefit from the efanesoctocog alfa treatment, as judged by the Investigator.
• • Willingness and ability of patient or their parent or legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study.
• Exclusion Criteria:
• • Positive inhibitor result (assessed by central laboratory), defined as ≥0.6 Bethesda units (BU)/mL, at Baseline Visit.
• • Ongoing or planned participation in any interventional clinical study at Baseline Visit.
• • Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.