Safety, Tolerability, PK and PD of ALN PCSSC in Elevated LDL-C
Research type
Research Study
Full title
A Phase 1, Randomized, Single Blind, Placebo Controlled, Single Ascending Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Administered ALN PCSSC in Subjects with Elevated Low Density Lipoprotein Cholesterol
IRAS ID
164885
Contact name
Jorg Taubel
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Eudract number
2014-002461-29
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
We are conducting a First-Into-Human clinical trial with an investigational medication known as ALN-PCSSC, which is being developed for treatment of hypercholesterolemia (high level of blood cholesterol). ALN-PCSSC works by lowering the amount of PCSK9 (a liver enzyme that controls levels of 'bad cholesterol' known as LDL-C). The study will be carried out in 2 parts - Single Ascending Dose (SAD) and Multiple Dosing (MD). The study will include up to 28 volunteers (SAD) or up to 48 volunteers (MD) who are otherwise healthy, but who have a higher than normal blood level of LDL-C, and who may or may not be receiving statin medication, or any lipid lowering treatment.
In this study we will assess the safety of ALN-PCSSC and how well it is tolerated when it is given as a single increasing dose in the SAD part of the study; or two doses in the MD part of the study via injections under the skin (i.e. sub-cutaneously - SC). We will also measure how much ALN-PCSSC is found in the blood and urine after sc dosing (i.e. pharmacokinetics) and the levels of LDL-C and PCSK9 in the blood, to determine if the Study Drug has any impact on these levels (i.e. pharmacodynamics).
Volunteers will be involved in the study for 4 – 6 months including one or two in-patient periods of 3 days/2 nights and a series of outpatient visits including the final follow-up which will be scheduled 56 days (SAD) or 84 days (MD) after receiving the study medication. Standard safety assessments will be conducted during the study.
This study will be conducted at Richmond Pharmacology Ltd. and Covance Clinical Research Unit Ltd and is funded by Alnylam Pharmaceuticals Inc. (the sponsor for the study), which is a biopharmaceutical company based in the USA.
REC name
London - Brent Research Ethics Committee
REC reference
14/LO/1790
Date of REC Opinion
7 Nov 2014
REC opinion
Favourable Opinion