Study to assess the safety and tolerability of ALN-PCS02

• Research type

  Research Study

• Full title

  A Phase 1, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-PCS02 in Subjects with Elevated LDL-Cholesterol (LDL-C).

• IRAS ID

  81074

• Sponsor organisation

  Alnylam Pharmaceuticals, Inc.

• Eudract number

  2011-000581-36

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  ALN-PCS02 is a new investigational drug that is being developed to treat hypercholesterolemia (elevated LDL-C). ALN-PCS02 is a form of RNA (ribonucleic acid) packaged inside tiny, fat containing particles (??lipid nanoparticles?). The form of RNA in ALN-PCS02 is known as small interfering RNA (siRNA). This molecule interferes with the action of RNA to reduce the amount of a protein called PCSK9 and the concentration of LDL-C circulating in the blood.The purpose of this study is to examine the safety, tolerability and the way the body handles a single dose of ALN-PCS02 and to determine whether it decreases LDL-cholesterol in the blood by lowering levels of PCSK9.Up to 32 subjects (men and women aged 18-65) with high levels of LDL-C are expected to take part. Volunteers are being admitted to the study unit for 8 days and will be asked to return to the unit for several follow-up outpatient visits after discharge.

• REC name

  South Central - Berkshire B Research Ethics Committee

• REC reference

  11/IE/0072

• Date of REC Opinion

  5 Jul 2011

• REC opinion

  Further Information Favourable Opinion