Amyloidosis in Transthyretin (TTR)Phase 3NCT07116473
To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)
A Phase 3 study testing Acoramidis (AG10) in people with Amyloidosis in Transthyretin (TTR) and Amyloidosis, Familial, run by Eidos Therapeutics, a BridgeBio company.
Trial summary
The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial, Amyloid Cardiomyopathy
- What is being tested?
- Acoramidis (AG10)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 250 participants
- Where
- No sites listed yet
- Study dates
- Nov 2026to Dec 2037
- Phase
- Phase 3
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must have completed the AG10-501 study (ACT-EARLY study) within the past 60 calendar days with a diagnosis of ATTR-CM (based on the AG10-501 protocol definition of ATTR-CM).
Who cannot join
- Participants who completed the AG10-501 study with a diagnosis of ATTR-PN only or who permanently discontinued study drug prior to diagnosis of ATTR-CM in AG10-501.
- History of light-chain amyloidosis (AL) or another non-TTR amyloid subtype (eg, ApoA-1, gelsolin).
- History of a monoclonal paraprotein or abnormal light chains in serum or urine (ie, monoclonal gammopathy of undetermined significance, MGUS) in which AL has not been ruled out.
- Stage IV or V chronic kidney disease.
- Active malignancy, except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix that has been successfully treated.
- History of any organ transplant (with the exception of corneal transplant).
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- Known hypersensitivity to acoramidis or any of its excipients.
- Major surgery planned during the next 6 months (some exceptions).
- Treatment of ATTR-CM with any ATTR-oriented on- or off-label or OTC product.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients With Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)
The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).
Read the detailed description
The AG10-504 study is the extension of the Phase 3 AG10-501 study (ACT-EARLY) which was designed to investigate if the use of acoramidis as a preventative intervention in individuals who are carriers of a known pathogenic transthyretin (TTR) variant but with no clinical evidence of ATTR could prevent or delay the onset of clinically detectable ATTR and the considerable morbidity and mortality that result from this devastating, progressive, and ultimately fatal disease. Only participants who have completed the AG10-501 study (ACT-EARLY) with a diagnosis of ATTR-CM may enroll in this AG10-504 Open Label Extension (OLE) study.
The primary objective of the AG10-504 study is to evaluate the long-term safety and tolerability of acoramidis in participants with newly diagnosed ATTR-CM. The duration of this study will be up to 5 years.
Currently, acoramidis is approved for the treatment of ATTR-CM in some regions including the United States, all member states of the European Union (EU), Albania, Bosnia Herzegovina, Brazil, Iceland, Israel, Japan, Liechtenstein, Montenegro, North Macedonia, Norway, Switzerland, and the United Kingdom (UK).
- Conditions
- Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial, Amyloid Cardiomyopathy, Amyloid Cardiomyopathy, Transthyretin-Related, Amyloidogenic Transthyretin (ATTR) Amyloidosis, Heart Disease
- Being tested
- Acoramidis (AG10)
- Keywords
- ATTR-CM, ATTR-PN, Amyloidosis, Transthyretin, Amyloid, TTR, Hereditary
- Medical subject headings
- Amyloidosis, Familial, Amyloid Neuropathies, Familial, Amyloidosis, Heart Diseases, Cardiomyopathies, Amyloidosis, Hereditary, Transthyretin-Related, Polyneuropathies, Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Acoramidis (AG10)Drug
Also called ALXN2060
TTR stabilizer administered orally twice daily (BID)
| Study group | Role | What they receive |
|---|---|---|
| Acoramidis | Experimental | Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID) Receives: Drug: Acoramidis (AG10) |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the long-term safety and tolerability of acoramidis in participants with newly diagnosed ATTR-CM
Proportion of participants with: treatment-emergent AEs and SAEs, AEs leading to treatment discontinuation, abnormal physical examination findings of clinical relevance, abnormal vital signs of clinical relevance, abnormal ECG parameters of clinical relevance, changes in clinical safety laboratory parameters of potential concern
Measured: The duration of study participation for the participants will be up to 5 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2025
- Study startNov 2026 (estimated)
- Primary completionNov 2037 (estimated)
- Study completionDec 2037 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyloidosis in Transthyretin (TTR)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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 250 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
- NCT07116473
- Sponsor's protocol ID
- AG10-504
- Other IDs
- U1111-1323-6219 (WHO), 2024-513676-18-00 (EUCT)
- Status last verified
- Aug 2026
- First submitted
- 7 Aug 2025
- Last updated
- 11 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No