Open-Label Study of AG10 in Patients With Cardiomyopathy



Status:Enrolling by invitation
Conditions:Cardiology
Therapuetic Areas:Cardiology / Vascular Diseases
Healthy:No
Age Range:18 - 90
Updated:8/11/2018
Start Date:August 8, 2018
End Date:July 2023

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

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.

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.

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.
We found this trial at
9
sites
75 Francis street
Boston, Massachusetts 02115
(617) 732-5500
Brigham and Women's Hosp Boston’s Brigham and Women’s Hospital (BWH) is an international leader in...
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116th St and Broadway
New York, New York 10027
(212) 854-1754
Columbia University In 1897, the university moved from Forty-ninth Street and Madison Avenue, where it...
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New York, NY
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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Portland, OR
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171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
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303 East Superior Street
Chicago, Illinois 60611
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Chicago, IL
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New Haven, Connecticut 6520
(203) 432-4771
Yale University Yale's roots can be traced back to the 1640s, when colonial clergymen led...
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New Haven, CT
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Palo Alto, California 94304
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Rochester, Minnesota 55905
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Rochester, MN
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San Francisco, California 94143
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San Francisco, CA
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