Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01437059
First received: September 19, 2011
Last updated: October 11, 2012
Last verified: October 2012
  Purpose

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).


Condition Intervention Phase
Elevated LDL-Cholesterol (LDL-C)
Drug: ALN-PCS02
Drug: Sterile Normal Saline (0.9% NaCl)
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Official 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)

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. [ Time Frame: Up to 28 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetics (PK) of ALN-PCS02 (Cmax, tmax, t1/2, AUC0-last, CL). [ Time Frame: Up to 180 days ] [ Designated as safety issue: Yes ]
  • Effect of ALN-PCS02 on Circulating PCSK9 Levels (Determination of % Lowering of PCSK9 to pretreatment/Baseline PCSK9 Level). [ Time Frame: Up to 28 days ] [ Designated as safety issue: No ]
  • Effect of ALN-PCS02 on Circulating LDL-c Levels (Determination of % Lowering of LDL-c to pretreatment/Baseline LDL-c Level). [ Time Frame: Up to 28 days ] [ Designated as safety issue: No ]

Enrollment: 32
Study Start Date: September 2011
Study Completion Date: September 2012
Primary Completion Date: March 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: ALN-PCS02 Drug: ALN-PCS02
Dose levels between 15 and 400 μg/kg by intravenous (IV) infusion
Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
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
  • Employee or family member of the sponsor or the clinical study site personnel
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01437059

Locations
United Kingdom
Clinical Site
Leeds, United Kingdom, LS2 9LH
Clinical Site
London, United Kingdom, SE1 1YR
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Amy Simon, MD Alnylam Pharmaceuticals
  More Information

No publications provided by Alnylam Pharmaceuticals

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01437059     History of Changes
Other Study ID Numbers: ALN-PCS02-001
Study First Received: September 19, 2011
Last Updated: October 11, 2012
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

ClinicalTrials.gov processed this record on April 14, 2014