NCT04988386

Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants With Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 389 (actual)
• Sponsor: Eidos Therapeutics, a BridgeBio company · Industry
• Sex: All
• Age: 18 Years – 90 Years
• Healthy volunteers: Not accepted

Summary

Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Detailed description

The primary objective of this prospective, multi-center, open-label study is to evaluate the long-term safety and tolerability of acoramidis in patients with an established diagnosis of ATTR-CM and heart failure who are concomitantly treated with currently recommended heart failure therapies. Secondary efficacy and PD objectives will be assessed. Exploratory objectives may also be assessed. All participants who complete 30 months of blinded study treatment and the Month 30 assessments of the double-blind treatment period of the Phase 3 ATTRibute-CM trial (AG10-301) may be eligible to participate in this Open Label Extension (OLE) study of acoramidis. The Day 1 visit in Study AG10-304 may be the same as the Month 30 visit in Study AG10-301. Under these circumstances, the last dose of Investigational Medicinal Product (IMP) in Study AG10-301 (either blinded acoramidis or matching placebo) will be the night before the day of the Month 30 visit. The first dose of open-label acoramidis in Study AG10-304 will be during the Study AG10-304 Day 1 visit after baseline assessments have been completed. Currently, tafamidis is approved for the treatment of ATTR-CM in some regions. Participants are not allowed to be treated with tafamidis or any other ATTR-CM-specific approved or investigational treatment, or therapies used off-label or as non-prescription supplements for ATTR-CM at any time during the study. If participants choose treatment with an alternative treatment as described above, they will be asked to discontinue acoramidis and complete an End of Treatment (EoT) form, and they may be asked to discontinue/withdraw from the study. If a participant discontinues/withdraws from the study, the participant will be asked to complete an early termination visit and a safety follow-up visit. Participants are not permitted to participate in another interventional clinical trial (except Study AG10-301) within 30 days prior to dosing and throughout Study AG10-304. Participants who choose to participate in another interventional clinical trial will be asked to withdraw from acoramidis and complete an EoT form, and they may be asked to discontinue/withdraw from the study. If a participant discontinues/withdraws from the study, the participant will be asked to complete an early termination visit and a safety follow-up visit. Participation in observational and/or registry studies should be discussed with the Medical Monitor.

Conditions

• Amyloid Cardiomyopathy, Transthyretin-Related

Interventions

Type	Name	Description
DRUG	Acoramidis (AG10)	Acoramidis (AG10) twice daily

Timeline

• Start date: 2021-10-27
• Primary completion: 2028-04-15
• Completion: 2028-05-15
• First posted: 2021-08-03
• Last updated: 2025-10-14

Locations

72 sites across 17 countries: United States, Australia, Belgium, Brazil, Canada, Czechia, Denmark, Greece, Ireland, Israel, Italy, Netherlands, New Zealand, Portugal, South Korea, Spain, United Kingdom

Regulatory

• FDA-regulated drug study