Eligibility NCT00630864 Transthyretin-associated Amyloidosis With Polyneuropathy Inclusion Criteria age 18 Years to 75 Years Yes
No
Patient has amyloid documented by biopsy (in accordance with institutional site standard of care). Yes
No
Patient has documentation of one of the following targeted TTR mutations: Ser77Tyr, Thr60Ala, Tyr114Cys, Leu58His, Glu89Gln, Ser77Phe, Thr49Ala, Ile107Val, Val30Ala, Gly47Ala, Gly47Glu, Leu55Arg, Lys70Asn, Ile84Thr, Ile107Met. Patients with mutations other than those listed may be enrolled only after approval by the Sponsor. Yes
No
Patient has peripheral and/or autonomic neuropathy and/or cardiomyopathy with a Karnofsky Performance Status >= 50. Yes
No
If female, patient is post-menopausal, surgically sterilized, or willing to use two acceptable methods of birth control (i.e., a hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom with spermicide) throughout the study and for 3 months from the end of the study. (A condom alone is not considered an acceptable method of birth control.) If male with a female partner of childbearing potential, willing to use two acceptable methods of birth control for the duration of the study. For both females and males, acceptable birth control must be used for at least 3 months after the last dose of study medication. Yes
No
Patient is, in the opinion of the investigator, willing and able to comply with the study medication regimen and all other study requirements. Yes
No
Exclusion Criteria Chronic use of non-steroidal anti-inflammatory drugs (NSAIDs), defined as > 3-4 times/month (ibuprofen and nimesulide will be permitted). Yes
No
Patient has primary or secondary amyloidosis. Yes
No
Patient has TTR-associated amyloidosis with V30M mutation. Yes
No
If female, patient is pregnant or breast feeding. Yes
No
Patient has received prior liver transplantation. Yes
No
Patient is expected to undergo liver transplantation within 12 months after enrollment. Yes
No
Patient with positive results for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (HCV), and/or human immunodeficiency virus (HIV). Yes
No
Patient has renal insufficiency (creatinine clearance < 30 ml/min). Yes
No
Patient has liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) > 2 times upper limit of normal (ULN) that in the medical judgment of the investigator are due to reduced liver function or active liver disease. Yes
No
Patient has a New York Heart Association (NYHA) Functional Classification >= III. Yes
No
Patient has other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV treated with retroviral medications, thyroid disorders, alcohol abuse, and chronic inflammatory diseases). Yes
No
Patient has prior non-amyloid cardiac disease such as: myocardial infarction due to obstructive coronary artery disease, active non-amyloid cardiomyopathy (e.g., symptomatic left ventricular dysfunction from any cause other than amyloid, patients with a primary diagnosis of symptomatic valvular heart disease) Yes
No
Patient has a co-morbidity anticipated to limit survival to < 12 months. Yes
No
Patient has received an investigational drug/device and/or participated in another clinical investigational study within 60 days before Baseline (Day 0). Yes
No
