To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)

Eidos Therapeutics

Status and phase: Not yet enrolling; Phase 3

Conditions: Polyneuropathies; Amyloidosis in Transthyretin (TTR); Amyloidosis, Hereditary, Transthyretin-Related; Amyloidogenic Transthyretin (ATTR) Amyloidosis; Amyloid Cardiomyopathy; Amyloid Cardiomyopathy, Transthyretin-Related; Amyloidosis, Familial; Amyloidosis; Cardiomyopathies; Heart Disease

Treatments: Drug: Acoramidis (AG10)

Study type: Interventional

Funder types: Industry

Identifiers

NCT07116473

2024-513676-18-00 (EUCT) (Other Identifier)

AG10-504

U1111-1323-6219 (WHO) (Other Identifier)

Details and patient eligibility

About

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).

Full 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.

Enrollment: 250 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

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).

Key Exclusion Criteria:

1. 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.
2. History of AL or another non-TTR amyloid subtype (eg, ApoA-1, gelsolin).
3. History of a monoclonal paraprotein or abnormal light chains in serum or urine (i.e., MGUS) in which AL has not been ruled out.
4. Stage IV or V chronic kidney disease (corresponding to an eGFR ≤ 29 mL/min/1.73 m2), or undergoing renal dialysis, or recipient of a kidney transplant.
5. Active malignancy, except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix that has been successfully treated. In the event a participant developed a low-grade and treatable malignancy in the AG10-501 study (eg, low-grade, localized prostate cancer) and the decision by the Investigator at that time was to continue the participant in the AG10-501 study, that participant may still qualify for Study AG10-504 after documentation with the Medical Monitor.
6. History of any organ transplant (with the exception of corneal transplant).
7. Known hypersensitivity to acoramidis or any of the excipients within the study drug.
8. Treatment for ATTR-CM with any ATTR-oriented on- or off-label or OTC product.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

250 participants in 1 patient group

Acoramidis

Experimental group

Description:

Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID)

Treatment:

Drug: Acoramidis (AG10)