A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C)

Phase 1

Completed

• Conditions: Hypercholesterolemia
• Interventions: Drug: Sterile Normal Saline (0.9% NaCl); Drug: ALN-PCSSC

• Registration Number: NCT02314442
• Lead Sponsor: Alnylam Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-12
• Study First Submitted: 2014-12-05
• Study First Submitted QC: 2014-12-09
• Study First Posted: 2014-12-11
• Primary Completion: 2015-05
• Study Completion: 2015-11
• Status Verified: 2015-12
• Last Update Submitted: 2015-12-16
• Last Update Posted: 2015-12-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

• 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]

Exclusion Criteria

• 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)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Sterile Normal Saline (0.9% NaCl)	Sterile Normal Saline (0.9% NaCl)	-
ALN-PCSSC	ALN-PCSSC	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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 plasma levels of PCSK9	Through the sooner of final follow up visit or 180 days post final 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

Trial Locations

Locations (2)

Covance Clinical Research Unit; 🇬🇧 Leeds, United Kingdom

Richmond Pharmacology; 🇬🇧 London, United Kingdom