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)

Alnylam Pharmaceuticals1 site in 1 country32 target enrollmentSeptember 2011

ConditionsElevated LDL-Cholesterol (LDL-C)

InterventionsALN-PCS02 Sterile Normal Saline (0.9% NaCl)

DrugsALN-PCS02 Sterile Normal Saline (0.9% NaCl)

Overview

Phase: Phase 1
Intervention: ALN-PCS02
Conditions: Elevated LDL-Cholesterol (LDL-C)
Sponsor: Alnylam Pharmaceuticals
Enrollment: 32
Locations: 1
Primary Endpoint: The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Status: Completed
Last Updated: 13 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-PCS02 in subjects with Elevated LDL-Cholesterol (LDL-C).

Registry

clinicaltrials.gov

Start Date

September 2011

End Date

September 2012

Last Updated

13 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Alnylam Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Elevated LDL-C of \>3.0 mmol/L and \<5.7 mmol/L
•Fasting triglyceride concentration ≤2.8 mmol/L
•Body weight \>60.0 kg; body mass index (BMI) between 19.00 kg/m2 and \<35.00 kg/m2
•Adequate blood counts, liver and renal function
•May not received any lipid lowering drug/agent within the 30 days prior to the screening
•Non-smokers for at least 3 months
•Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control
•Males agree to use appropriate contraception
•Willing and able to comply with protocol-required visit schedule and visit requirements and provide written informed consent

Exclusion Criteria

•Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
•Multiple drug allergies or know sensitivity to oligonucleotide
•History of drug abuse and/or alcohol abuse
•Receiving an investigational agent within 3 months prior to study drug administration
•Subjects with safety laboratory test results deemed clinical significant by the Investigator;
•Received prescription drugs within 4 weeks of first dosing
•Subjects who have donated more than 500 mL of blood within the 3 months prior to ALN-PCS02 or placebo administration;
•Received megadose vitamin therapy or dietary supplements within 4 weeks prior to screening
•Subjects who have used prescription drugs within 4 weeks of first dosing
•Considered unfit for the study by the Principal Investigator

Arms & Interventions

ALN-PCS02

Intervention: ALN-PCS02

Sterile Normal Saline (0.9% NaCl)

Intervention: Sterile Normal Saline (0.9% NaCl)

Outcomes

Primary Outcomes

The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.

Time Frame: Up to 28 days

Secondary Outcomes

Pharmacokinetics (PK) of ALN-PCS02 (Cmax, tmax, t1/2, AUC0-last, CL).(Up to 180 days)
Effect of ALN-PCS02 on Circulating PCSK9 Levels (Determination of % Lowering of PCSK9 to pretreatment/Baseline PCSK9 Level).(Up to 28 days)
Effect of ALN-PCS02 on Circulating LDL-c Levels (Determination of % Lowering of LDL-c to pretreatment/Baseline LDL-c Level).(Up to 28 days)