Safety monitoring of Acoramidis in Subjects with ATTR-CM
Research type
Research Study
Full title
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)
IRAS ID
302733
Contact name
Julian Gillmore
Contact email
Sponsor organisation
Eidos Therapeutics, Inc.
Eudract number
2020-005643-22
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
133574, IND Number
Duration of Study in the UK
6 years, 9 months, 0 days
Research summary
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a life-threatening disease characterised by progressive heart
failure. Acoramidis is a medication under development for the treatment of ATTR-CM. This study will evaluate the long-term safety and tolerability of acoramidis in the context of background heart failure therapy. The study is planned to involve up to 632 participants who have symptomatic ATTR-CM and who have completed the ATTRibute-CM study with acoramidis or placebo through month 30. This is an Open Label Extension (OLE) study. There is only 1 treatment group. All participants will receive 712 mg of acoramidis (equivalent to 800 mg of acoramidis hydrochloride (HCl)) by taking 2 pills twice a day. There are two parts to the study. Part 1: ‘Treatment period’ approx. 60 months long. Part 2: ‘Follow-up period’ which has one visit approx. 30 days after participants’ last dose of study medicine. Treatment period and follow-up period will involve a total of 13 visits. After the first visit, participant visits will be at Month 1, 6, and every 6 months until Month 60. These visits will last approximately 2 hours. Participation is expected up to 61 months. Participants will undergo various assessments at each visit including; medical and surgery history, medical assessment of their heart condition, answering 2 questionnaires, blood and urine tests and a physical examination including weight, height, temperature, heart rate, blood pressure, breathing rate electrocardiogram (ECG). The study doctor will also review previous/current medications. The primary endpoints are treatment-emergent serious adverse events (SAEs) and AEs, AEs leading to treatment discontinuation, abnormal physical exam findings, abnormal vital signs, abnormal electrocardiogram (ECG) parameters, and changes in clinical safety laboratory parameters of clinical relevance. Up to 50 participants can enrol in a cardiac magnetic resonance (CMR) substudy which would involve having up to 6 CMR scans between baseline and month 60.REC name
Wales REC 1
REC reference
21/WA/0307
Date of REC Opinion
22 Oct 2021
REC opinion
Further Information Favourable Opinion