24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients
Not yet recruiting
Brigham and Women's Hospital
Transthyretin is a protein produced in the liver that transports thyroid hormone and vitamin
A. A single substitution of an amino acid in the structure of TTR can result in a relatively
unstable protein, the breakdown products of which (predominantly monomers) aggregate
abnormally and produce proteinaceous deposits in nerves and the heart. These deposits are
known as amyloid and produce progressive nerve and heart damage. Amyloidosis due to a mutant
TTR is usually an autosomal dominant and hence is a familial condition. Wild-type TTR is also
capable of producing amyloid deposits which predominantly involves the heart (rather than the
nervous system) resulting in a progressive decrease in cardiac function with increasing signs
of heart failure. This study aims to determine whether subcutaneous injection of an antisense
oligonucleotide drug, known as inotersen, that has been specifically designed to reduce
production of the protein transthyretin by the liver, can slow or stop the progression of TTR
amyloid cardiomyopathy as compared to historical controls, using advanced echocardiography
and cardiac MRI. The study also aims to determine the tolerability and safety of this drug
when administered over a 24-month period to patients with TTR amyloid cardiomyopathy.
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