To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Acoramidis (AG10).
- Who it may be relevant to
- Registry conditions: Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial, Amyloid Cardiomyopathy, Amyloid Cardiomyopathy, Transthyretin-Related. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients With Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)
Overview
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.
Interventions
- Drug Acoramidis (AG10)
TTR stabilizer administered orally twice daily (BID)
Primary outcome measures
- To evaluate the long-term safety and tolerability of acoramidis in participants with newly diagnosed ATTR-CM [Time frame: The duration of study participation for the participants will be up to 5 years.]
Eligibility criteria
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).
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07116473 · AG10-504 · U1111-1323-6219 (WHO) · 2024-513676-18-00 (EUCT)