An Open-Label Extension and Safety Monitoring Study of Patients With Symptomatic Transthyretin Cardiomyopathy Who Have Completed the Phase II Study AG10-201

AG10-202

This prospective, multicenter open-label study will evaluate the long-term safety, tolerability, PK and PD of AG10 administered on a background of stable heart failure therapy.

2018-08-08

2027-07-01

2027-07-01

Active (not recruiting)

Early phase I

55

Inclusion Criteria: 1. Completed participation in study AG10-201. 2. Willing and able to comply with the study medication regimen and all study requirements. 3. The ability to understand and provide informed consent, which must be obtained prior to initiation of study participation. 4. Male patients and female patients of childbearing potential who engage in heterosexual intercourse must agree to use effective method(s) of contraception. Exclusion Criteria: 1. Hemodynamic instability that would pose too great a risk to the subject. 2. Abnormalities in clinical laboratory tests that would pose too great a risk to the subject. 3. Any clinically significant ongoing medical condition or any laboratory abnormality or condition that could adversely affect the safety of the patient. 4. Known hypersensitivity to study drug (AG10), its metabolites, or formulation excipients. 5. Likely to undergo heart transplantation or placement of a mechanical circulatory device within the next year. 6. Receiving current treatment with diflunisal, tafamidis, green tea, doxycycline, TUDCA/Ursodiol, patisiran, inotersen or any other investigational ATTR agent within 14 days or 5 half-lives of the prior investigational agent (whichever is longer) prior to dosing with study drug. 7. Females who are pregnant or breastfeeding. Lactating females must agree to discontinue nursing before the study drug is administered.

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2023-02-03