1. Amyloid documented by biopsy.
2. Documented V30M TTR mutation.
3. Peripheral and/or autonomic neuropathy with a Karnofsky Performance Status ≥50.
4. Patient is 18-75 years old.
5. If female, patient is post-menopausal, surgically sterilized, or willing to use an
acceptable method of birth control. If male with a female partner of childbearing
potential, willing to use an acceptable method of birth control for the duration of
the study. For both females and males, birth control must be used for at least 3
months after the last dose of study medication.
6. Patient is, in the opinion of the investigator, willing and able to comply with the
study medication regimen and all other study requirements.
1. Chronic use of non-steroidal anti-inflammatory drugs (NSAIDs).
2. Primary amyloidosis.
3. If female, patient is pregnant or breast feeding.
4. Prior liver transplantation.
5. No recordable sensory threshold for vibration perception in both feet, as measured by
6. Positive results for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus
(HCV), and/or human immunodeficiency virus (HIV).
7. Renal insufficiency or liver function test abnormalities.
8. New York Heart Association (NYHA) Functional Classification ?III.
9. Other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV
treated with retroviral medications, thyroid disorders, alcohol abuse, and chronic
10. Co-morbidity anticipated to limit survival to less than 18 months.
11. Patient received an investigational drug/device and/or participated in another
clinical investigational study within 60 days before Baseline.