Open-Label Study of AG10 in Patients With Cardiomyopathy
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ClinicalTrials.gov Identifier: NCT03536767 |
Recruitment Status :
Enrolling by invitation
First Posted : May 25, 2018
Last Update Posted : November 18, 2019
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Amyloid Cardiomyopathy | Drug: AG10 | Phase 2 |
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 up to 55 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 at Day 14, Day 45, 3 Months and every 3 months thereafter. There will be one 7 to 14-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.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 55 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | August 8, 2018 |
Estimated Primary Completion Date : | July 2023 |
Estimated Study Completion Date : | July 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: Open-Label |
Drug: AG10
AG10 800mg twice daily, oral administration
Other Name: TTR Stabilizer |
- Assessment of long-term safety and tolerability: Incidence of each treatment-emergent adverse events [ Time Frame: up to 60 Months or study completion by recommendation from safety monitoring committee ]Incidence of each treatment-emergent adverse events
- AG10 Pharmacokinetics AUC [ Time Frame: up to 60 Months or study completion by recommendation from safety monitoring committee ]Area under the plasma concentration-time curve (AUC)
- AG10 Pharmacodynamic Assessments of TTR stabilization by Fluorescent Polarization Exclusion Assay [ Time Frame: up to 60 Months or study completion by recommendation from safety monitoring committee ]AG10 binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: Fluorescent Polarization Exclusion Assay (FPE)
- AG10 Pharmacodynamic Assessments of TTR stabilization by Western Blot [ Time Frame: up to 60 Months or study completion by recommendation from safety monitoring committee ]AG10 binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: Immunoblotting (Western Blot)
- AG10 Pharmacodynamic Assessments: prealbumin [ Time Frame: up to 60 Months or study completion by recommendation from safety monitoring committee ]AG10 binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: quantitation of prealbumin (TTR).

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Ages Eligible for Study: | 18 Years to 90 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Completed participation in study AG10-201.
- Willing and able to comply with the study medication regimen and all study requirements.
- The ability to understand and provide informed consent, which must be obtained prior to initiation of study participation.
- Male patients and female patients of childbearing potential who engage in heterosexual intercourse must agree to use effective method(s) of contraception.
Exclusion Criteria:
- Hemodynamic instability that would pose too great a risk to the subject.
- Abnormalities in clinical laboratory tests that would pose too great a risk to the subject.
- Any clinically significant ongoing medical condition or any laboratory abnormality or condition that could adversely affect the safety of the patient.
- Known hypersensitivity to study drug (AG10), its metabolites, or formulation excipients.
- Likely to undergo heart transplantation or placement of a mechanical circulatory device within the next year.
- 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.
- Females who are pregnant or breastfeeding. Lactating females must agree to discontinue nursing before the study drug is administered.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03536767
United States, California | |
Stanford University | |
Palo Alto, California, United States, 94304 | |
University of California San Francisco | |
San Francisco, California, United States, 94143 | |
United States, Connecticut | |
Yale University | |
New Haven, Connecticut, United States, 06520 | |
United States, Illinois | |
Northwestern University | |
Chicago, Illinois, United States, 60611 | |
United States, Massachusetts | |
Brigham and Women's Hospital | |
Boston, Massachusetts, United States, 02115 | |
United States, Minnesota | |
Mayo Clinic | |
Rochester, Minnesota, United States, 55905 | |
United States, New York | |
Columbia University | |
New York, New York, United States, 10032 | |
United States, Oregon | |
Oregon Health & Science University | |
Portland, Oregon, United States, 97239 | |
United States, South Carolina | |
Medical University of South Carolina | |
Charleston, South Carolina, United States, 29424 |
Responsible Party: | Eidos Therapeutics |
ClinicalTrials.gov Identifier: | NCT03536767 |
Other Study ID Numbers: |
AG10-202 |
First Posted: | May 25, 2018 Key Record Dates |
Last Update Posted: | November 18, 2019 |
Last Verified: | November 2019 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Cardiomyopathies Heart Diseases Cardiovascular Diseases |