A Non-interventional Cohort Safety Study of Patients With hATTR-PN

Launched by AKCEA THERAPEUTICS · Apr 14, 2021

Keywords

ClinConnect Summary

This clinical trial is studying the long-term safety of a medication called TEGSEDI (inotersen) in patients with a condition known as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). This condition affects the nerves and can lead to various symptoms, including pain and weakness. The trial is open to patients who have either recently taken TEGSEDI or who are eligible to start treatment but haven't taken it yet. Participants will be monitored over time to see how well they tolerate the medication in a real-world setting.

To be eligible for this study, patients need to be diagnosed with hATTR-PN and must be receiving care in Canada, Europe, or the United States. There are no specific exclusions, which means most patients with the right diagnosis can participate. By joining this study, participants can help researchers gain valuable insights into the safety of TEGSEDI, contributing to better understanding and management of their condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• 1. Either:
• • 1. TEGSEDI Exposed Cohort: Patients diagnosed with hATTR-PN who have taken any dose of TEGSEDI within 25 weeks prior to enrollment
• • 2. TEGSEDI Unexposed Cohort: Patients diagnosed with hATTR-PN who have not taken any dose of TEGSEDI within 25 weeks prior to enrollment and are eligible for TEGSEDI treatment per applicable product label
• • 2. Clinically managed in Canada, Europe, or the US
• • 3. Have provided appropriate written informed consent
• Exclusion Criteria:
• • None