NCT02314442

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-PCSSC in subjects with elevated LDL-C.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
70

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Dec 2014

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2014

Completed
4 days until next milestone

First Submitted

Initial submission to the registry

December 5, 2014

Completed
6 days until next milestone

First Posted

Study publicly available on registry

December 11, 2014

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2015

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2015

Completed
Last Updated

December 17, 2015

Status Verified

December 1, 2015

Enrollment Period

5 months

First QC Date

December 5, 2014

Last Update Submit

December 16, 2015

Conditions

Hypercholesterolemia

Keywords

LDL-CRNAi therapeutic

Outcome Measures

Primary Outcomes (1)

  • The safety of ALN-PCSSC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation

    Part 1 (SAD phase): up to 180 days post dose; Part 2 (MD) phase: up to 180 days post final dose

Secondary Outcomes (3)

  • The pharmacokinetics (PK) of ALN-PCSSC (Cmax, tmax, AUC, t1/2)

    Part 1 (SAD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 42, 56 days post-dose; Part 2 (MD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 29, 35, 42, 56, 84 days post-dose

  • The effect of ALN-PCSSC on serum levels of LDL-C

    Through the sooner of final follow up visit or 180 days post final dose

  • The effect of ALN-PCSSC on plasma levels of PCSK9

    Through the sooner of final follow up visit or 180 days post final dose

Study Arms (2)

ALN-PCSSC

ACTIVE COMPARATOR
Drug: ALN-PCSSC

Sterile Normal Saline (0.9% NaCl)

PLACEBO COMPARATOR
Drug: Sterile Normal Saline (0.9% NaCl)

Interventions

ALN-PCSSCDRUG

Single or multiple doses of ALN-PCSSC by subcutaneous (sc) injection

ALN-PCSSC
Sterile Normal Saline (0.9% NaCl)DRUG

calculated volume to match active comparator

Sterile Normal Saline (0.9% NaCl)

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • Adequate complete blood counts, liver and renal function
  • Female subjects must be of non-childbearing potential; e.g. post-menopausal or pre-menopausal with surgical sterilization
  • Male subjects agree to use appropriate contraception
  • Willing to provide written informed consent and willing to comply with study requirements.
  • Non-smokers and non-nicotine users for at least 90 days before screening
  • On stable statin co-medication \[for designated multiple dose cohorts only\]

You may not qualify if:

  • Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk
  • Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
  • History of multiple drug allergies or intolerance to subcutaneous injection
  • Received any medication or nutraceutical to alter serum lipids within 30 days before screening (non-statin cohorts only)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Covance Clinical Research Unit

Leeds, United Kingdom

Location

Richmond Pharmacology

London, United Kingdom

Location

Related Publications (1)

  • Fitzgerald K, White S, Borodovsky A, Bettencourt BR, Strahs A, Clausen V, Wijngaard P, Horton JD, Taubel J, Brooks A, Fernando C, Kauffman RS, Kallend D, Vaishnaw A, Simon A. A Highly Durable RNAi Therapeutic Inhibitor of PCSK9. N Engl J Med. 2017 Jan 5;376(1):41-51. doi: 10.1056/NEJMoa1609243. Epub 2016 Nov 13.

    PMID: 27959715DERIVED

MeSH Terms

Conditions

Hypercholesterolemia

Interventions

ALN-PCS

Condition Hierarchy (Ancestors)

HyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Robert Kauffman, MD

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 5, 2014

First Posted

December 11, 2014

Study Start

December 1, 2014

Primary Completion

May 1, 2015

Study Completion

November 1, 2015

Last Updated

December 17, 2015

Record last verified: 2015-12

Locations

GB(2)