for people ages 18-90 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:



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.

Official Title

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


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

The primary objective of this study is to evaluate the long-term safety and tolerability of AG10 administered to adult patients with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM) in patients who have completed the study AG10-201.

This study will be an Open-Label Extension and Safety Monitoring Study of 45 male and/or female patients with symptomatic ATTR-CM aged 18 through 90 years, who have completed the Phase II Study AG10-201. Enrollment into the study will be followed by visits every 3-months and a 30-day follow-up visit after last dose.

If all doses are well tolerated, the duration of each patient's participation in the study will continue based on periodic recommendations of the AG10 Data Monitoring Committee (DMC) and/or registration of the product for the treatment of symptomatic ATTR-CM.

The secondary objectives of this study are to characterize the pharmacokinetics (PK) of AG10 administered orally twice daily in patients with symptomatic ATTR-CM, and to describe the long-term pharmacodynamic (PD) properties of AG10 as assessed by established assays of transthyretin (TTR) stabilization, including Fluorescent Probe Exclusion (FPE) assay and Western blot, and to describe the PK-PD relationship of AG10 in adult patients with symptomatic ATTR-CM.


Amyloid Cardiomyopathy Cardiomyopathies AG10 Open-Label


You can join if…

Open to people ages 18-90

  1. Has completed the study AG10-201.
  2. Is, in the opinion of the investigator, willing and able to comply with the study medication regimen and all other study requirements.
  3. Agrees not to participate in another investigational drug or device study while participating in this open-label extension study.
  4. Has the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures.
  5. Male patients and female patients of childbearing potential who engage in heterosexual intercourse must agree to use appropriate method(s) of contraception.

You CAN'T join if...

  1. Has hemodynamic instability at Screening that, in the judgment of the PI, would pose too great a risk for participation in the study.
  2. Has estimated glomerular filtration rate (GFR) <30 mL/min/1.73 m2 at Screening.
  3. Has abnormalities in clinical laboratory tests at Screening that, in the judgment of the PI, would pose too great a risk for participation in the study.
  4. Has any clinically significant ongoing medical condition that, in the judgment of the PI, might jeopardize the patient's safety or interfere with the study, including participation in another investigational drug or investigational device study within the 30 days prior to Screening with potential residual effects that might confound the results of this study.
  5. Has any laboratory abnormality or condition that, in the investigator's opinion, could adversely affect the safety of the patient or impair the assessment of study results.
  6. Has known hypersensitivity to study drug (AG10 or placebo), its metabolites, or formulation excipients.
  7. Is likely to undergo heart transplantation or placement of a mechanical circulatory device within the next year.
  8. Is 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 Screening.
  9. Females who are pregnant or breastfeeding. Lactating females must agree to discontinue nursing before the study drug is administered. A negative serum pregnancy test at Screening and a negative urine pregnancy test at Day 1 visit are required for female patients of childbearing potential.


  • University of California San Francisco
    San Francisco California 94143 United States
  • Stanford University
    Palo Alto California 94304 United States


accepting new patients by invitation only
Start Date
Completion Date
Eidos Therapeutics
Phase 2
Study Type
Last Updated
May 30, 2018