Trials / Not Yet Recruiting
Not Yet RecruitingNCT07116473
To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)
A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients With Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)
- Status
- Not Yet Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 250 (estimated)
- Sponsor
- Eidos Therapeutics, a BridgeBio company · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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).
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 prophylactic 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, United Kingdom, Japan, and the European Union.
Conditions
- Amyloidosis in Transthyretin (TTR)
- Amyloidosis, Familial
- Amyloid Cardiomyopathy
- Amyloid Cardiomyopathy, Transthyretin-Related
- Amyloidogenic Transthyretin (ATTR) Amyloidosis
- Heart Disease
- Cardiomyopathies
- Amyloidosis, Hereditary, Transthyretin-Related
- Polyneuropathies
- Amyloidosis
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Acoramidis (AG10) | TTR stabilizer administered orally twice daily (BID) |
Timeline
- Start date
- 2025-11-01
- Primary completion
- 2037-11-01
- Completion
- 2037-12-01
- First posted
- 2025-08-11
- Last updated
- 2025-08-11
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT07116473. Inclusion in this directory is not an endorsement.