Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)Phase 4ASCEND-ATTRNCT07695701
Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis
A Phase 4 study testing Acoramidis (AG10) oral tablet in people with Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) and Amyloidosis, run by Eidos Therapeutics, a BridgeBio company at 1 site in United States.
Trial summary
The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy
- What is being tested?
- Acoramidis (AG10) oral tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 150 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2026to Nov 2030
- Phase
- Phase 4
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants aged 18 to 80
- Confirmed diagnosis of ATTR-CM requiring targeted therapy
- At screening: New York Heart Association (NYHA) Class I/II; estimated glomerular filtration rate (eGFR) ≥40 mL/min/1.73m2; left ventricular ejection fraction (LVEF) >35%; left ventricular (LV) wall thickness ≥12 mm.
- Heart failure condition has been clinically stable for at least 6 weeks prior to screening.
Who cannot join
- Current or planned implantation of a cardiac implantable electronic device (eg, pacemaker, implantable cardiac defibrillator).
- Acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening.
- Hemodynamic instability.
- Likely to undergo heart transplantation within 12 months from screening.
- Confirmed diagnosis of AL amyloidosis at screening.
- At screening: NYHA Class III/IV; National Amyloidosis Center (NAC) Stage 3.
- Prior or current treatment with acoramidis.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Washington University in St. Louis
St Louis, Missouri, 63110
About this study
Official title: An Open-Label Study to Investigate the Long-Term Effects of Acoramidis on Cardiac Function, Structure and Cardiac Amyloid Burden in Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM.
Read the detailed description
Study AG10-404 is an open-label study to investigate the long-term effects of acoramidis on cardiac functional and structural improvement, and the effects on cardiac amyloid burden in participants with ATTR-CM using two types of imaging, cardiac MRI and echocardiograms to track changes in heart structure and amyloid buildup over time.
Up to approximately 150 participants (ages 18-80) will be enrolled. Participants will receive study drug (712 mg equivalent to 800 mg of acoramidis hydrochloride) orally twice daily from Day 1 through Month 36. The study duration for an individual participant is approximately 38 months, including up to 45 days for screening, 36 months for open-label treatment and 1 month follow-up after the last dose of study drug. In-clinic visits will occur at Day 1, Day 28, Month 6, and every 6 months up to Month 36. Phone visits will occur at Month 3 and every 6 months thereafter up to Month 33. Participants will be followed throughout their participation in this study and for 30 days (+7 days) after the last dose of study drug.
- Conditions
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Being tested
- Acoramidis (AG10) oral tablet
- Keywords
- amyloidosis, cardiomyopathy, ATTR-CM, Amyloid, TTR
- Medical subject headings
- Amyloidosis, Amyloid Neuropathies, Familial, Amyloidosis, Hereditary, Transthyretin-Related, Cardiomyopathies, Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Acoramidis (AG10) oral tabletDrug
Study drug is defined as acoramidis 712 mg (equivalent to 800 mg acoramidis hydrochloride) taken orally twice daily for 36 months of open label treatment.
| Study group | Role | What they receive |
|---|---|---|
| Acoramidis (AG10) | Experimental | Open-label study where all participants will receive acoramidis (AG10) during the study Receives: Drug: Acoramidis (AG10) oral tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the effect of acoramidis on cardiac function improvement by CMR in participants with ATTR-CM.
Change from baseline in left ventricular ejection fraction (LVEF) or left ventricular stroke volume index (LVSVi) at Month 36.
Measured: Month 36
Secondary outcomes (5)
To assess the effect of acoramidis on cardiac function improvement or stabilization by CMR in participants with ATTR-CM.
Change from baseline in LVEF and LVSVi at Month 36
Measured: Month 36
To assess the effect of acoramidis on cardiac function stabilization by CMR in participants with ATTR-CM.
Change from baseline in LVEF and LVSVi at Month 36
Measured: Month 36
To evaluate the effect of treatment with acoramidis on individual parameters of left ventricular systolic function by CMR in participants with ATTR-CM
Mean change from baseline in LVEF, LVSVi, left ventricular global longitudinal strain (LVGLS) at Month 36
Measured: Month 36
To evaluate the effect of acoramidis on cardiac structure by CMR in participants with ATTR-CM
Mean change from baseline in left ventricular mass index (LVMi) at Month 36
Measured: Month 36
To evaluate the effect of treatment with acoramidis on cardiac amyloid burden by CMR in participants with ATTR-CM
Mean change from baseline in extracellular volume (ECV) at Month 36
Measured: Month 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2026
- Study startJul 2026 (estimated)
- Primary completionAug 2030 (estimated)
- Study completionNov 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07695701
- Sponsor's protocol ID
- AG10-404
- Other IDs
- 2026-526068-19-00, U1111-1339-5700
- Acronym
- ASCEND-ATTR
- Status last verified
- Jul 2026
- First submitted
- 6 Jul 2026
- Last updated
- 29 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided