A Phase 2/3 Randomized Double-Blind Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy

ID Number 13-1509

Principal Investigator(s)
Peter D Gorevic

Department(s) or Division(s)


The drug being researched is called ISIS 420915. ISIS 420915 is an investigational drug. An "investigational drug" is one that is not approved by regulatory authorities such as "US Food and Drug Administration (FDA)". The drug is not for sale in any country. The purpose of this study is to determine if ISIS 420915 can help people with mild or moderate Familial Amyloid Polyneuropathy (FAP). To be in this study, one must have FAP and be able to walk without help (Stage 1) or walk with using only one cane (Stage 2). Subjects may qualify to take part in this research study because they have Familial Amyloid Polyneuropathy (FAP). 

FAP is a disease that is caused when one has a mutation (accidental change of the cell) in a protein called transthyretin (TTR). Mutations in TTR cause it to clump and make amyloid deposits (deposits of abnormal material) in different organs in the body. The amyloid deposits build up in areas such as the nervous system, digestive (gastrointestinal) tract, and sometimes in the heart. This build up can affect organs so they do not work properly and can cause neuropathy (neuropathy is a collection of disorders that occurs when nerves of the peripheral nervous system - the part of the nervous system outside of the brain and spinal cord- are damaged) or heart disease. 

ISIS 420915 temporarily reduces the level of TTR in the blood. Reducing the amount of TTR in blood may reduce the amount of amyloid deposits that are building up in the body's tissues and may keep neuropathy from getting worse or slow down its progress. Funds for conducting this research are provided by Isis Pharmaceuticals Inc. also the manufacturer of this drug. The length of the study is approximately 96 - 102 weeks (approximately 2 years).

Contact Information
Garnette Mason
(212) 241-4824

Recruiting Patients: No