Research Study for Patients with Cardiac Amyloids

Study Summary

The University of Chicago and Dr. Savitri Fedson are conducting a research study for patients who have been diagnosed with a condition called Transthyretin Amyloid Cardiomyopathy (ATTR-CM), also known as Familial Amyloid Cardiomyopathy (FAC) or Senile Systemic Amyloidosis (SSA). Your liver produces a protein called Transthyretin (TTR). TTR is an important protein that carries other substances, like hormones, through the body. In patients with Transthyretin Amyloid Cardiomyopathy, this liver protein (TTR) breaks up and may form fibers called amyloid. Amyloid deposits develop in the heart muscle and in other places in your body preventing your heart and other parts of your body from working effectively. Eventually, the amyloid deposits cause the symptoms of TTR Amyloid Cardiomyopathy.

Transthyretin Amyloid Cardiomyopathy develops due to either a gene that is not normal (FAC), or due to the effects of age on the TTR protein (SSA). Genes are inherited from your parents and help determine certain aspects of physical development and body function. In Familial Amyloid Cardiomyopathy(FAC), a gene is not normal and makes abnormal TTR; in Senile Sytemic Amyloidosis (SSA) the gene works normally, however, the TTR protein itself becomes unstable with age.

This study is to determine if a new experimental drug can stop the TTR protein from breaking up and forming amyloid. The study drug is experimental, which means it is not approved by the Food and Drug Administration (FDA).

You will be in the study for about 14 ½ months. You will be screened to see if you qualify for participation. If you qualify, you will receive the experimental study drug once per day for up to 12 months. Some of the procedures that may be done during the study include genetic testing pertaining to cardiac amyloids, analysis of your amyloid biopsy sample, physical exams, blood tests, urine tests, echocardiogram (test that uses sound waves to form an image of your heart), electrocardiogram (test that measures the electrical activity of your heart), walk test (a measure of your tolerance for light exercise), heart MRI (a test that produces very high quality images of the heart), chest x-rays, questionnaires, and follow-up telephone calls. Study visits in the clinic occur at screening, baseline, week 2, week 6, month 3, month 6 and month 12. At each study visit that you attend, you will receive a voucher for free parking.

Benefits

You may not benefit directly from this research. However, the medical knowledge obtained from this study may be of considerable benefit to help treat patients with cardiac amyloids in the future.

Participation Details

Initial selection criteria include:

• Must be age 40 years or older
• Must have a history of Familial Amyloid Cardiomyopathy (FAC) or Senile Systemic Amyloidosis (SSA)
• Must be available for clinic visits for the duration of the study
• Must meet study medical criteria as evaluated by Dr. Fedson

Study Director: Dr. Savitri Fedson
Department: Cardiology

For more information contact:
lbond@medicine.bsd.uchicago.edu
(773) 702-6385

IRB: #16332A