ISIS 304801 in Patients With Familial Chylomicronemia Syndrome

• Research type

  Research Study

• Full title

  A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 304801 Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)

• IRAS ID

  159136

• Contact name

  Dr Steven Martin

• Contact email

  steven.martin@pbh-tr.nhs.uk

• Sponsor organisation

  Isis Pharmaceuticals, Inc.

• Eudract number

  2014-002421-35

• Clinicaltrials.gov Identifier

  NCT02211209

• Research summary

  ISIS 304801 is an antisense drug designed to reduce apoC-III protein production and lower triglycerides. ApoC-III regulates triglyceride metabolism in the blood and is an independent cardiovascular risk factor. People who have certain mutations in the gene for apoC-III that result in lower levels of apoC-III have lower levels of triglycerides and lower instances of cardiovascular disease. People with elevated levels of apoC-III have increased dyslipidemia associated with multiple metabolic abnormalities, such as insulin resistance and/or metabolic syndrome. In addition, people with elevated triglycerides are at increased risk for type 2 diabetes, and people with severely elevated triglycerides are at high risk for acute pancreatitis and other serious conditions.

  In this study, approximately 50 eligible patients worldwide will receive 300 mg of ISIS 304801 or placebo once per week for 26 weeks.

• REC name

  East Midlands - Leicester Central Research Ethics Committee

• REC reference

  14/EM/1141

• Date of REC Opinion

  27 Oct 2014

• REC opinion

  Further Information Favourable Opinion