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

NCT07116473 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: AG10-504U1111-1323-6219 (WHO)2024-513676-18-00 (EUCT)
• Study Type: interventional
• Target: 250
• Locations: 0 countries
• Sites: N/A

Brief 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).

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

Keywords

ATTR-CM; ATTR-PN; Amyloidosis; Transthyretin; Amyloid; TTR; Hereditary

Outcome Measures

Primary Outcomes (1)

• 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

  The duration of study participation for the participants will be up to 5 years.

Study Arms (1)

Acoramidis

EXPERIMENTAL

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

Drug: Acoramidis (AG10)

Interventions

Acoramidis (AG10) DRUG

TTR stabilizer administered orally twice daily (BID)

Also known as: ALXN2060

Acoramidis

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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 AL or another non-TTR amyloid subtype (eg, ApoA-1, gelsolin).
• History of a monoclonal paraprotein or abnormal light chains in serum or urine (i.e., MGUS) in which AL has not been ruled out.
• 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.
• 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.
• History of any organ transplant (with the exception of corneal transplant).
• Known hypersensitivity to acoramidis or any of the excipients within the study drug.
• Treatment for ATTR-CM with any ATTR-oriented on- or off-label or OTC product.

Sponsors & Collaborators

• Eidos Therapeutics, a BridgeBio company: lead

MeSH Terms

Conditions

Amyloidosis, Familial; Amyloid Neuropathies, Familial; Amyloidosis; Heart Diseases; Cardiomyopathies; Amyloidosis, Hereditary, Transthyretin-Related; Polyneuropathies; Alzheimer Disease

Interventions

attruby

Condition Hierarchy (Ancestors)

Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Proteostasis Deficiencies; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Cardiovascular Diseases; Dementia; Brain Diseases; Central Nervous System Diseases; Tauopathies; Neurocognitive Disorders; Mental Disorders

Central Study Contacts

Medical Information

CONTACT

1-415-887-1471; medinfo@eidostx.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2025
• First Posted: August 11, 2025
• Study Start: November 1, 2025
• Primary Completion (Estimated): November 1, 2037
• Study Completion (Estimated): December 1, 2037
• Last Updated: August 11, 2025

Record last verified: 2025-08