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

Hypercholesterolemia Clinical Trial

Official 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 Subcutaneously Administered ALN PCSSC in Subjects With Elevated Low Density Lipoprotein Cholesterol

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02314442
• Other study ID #: ALN-PCSSC-001
• Status: Completed
• Phase: Phase 1
• First received: December 5, 2014
• Last updated: December 16, 2015
• Start date: December 2014
• Est. completion date: November 2015

Study information

• Verified date: December 2015
• Source: Alnylam Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 70
• Est. completion date: November 2015
• Est. primary completion date: May 2015
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 18 Years to 60 Years

Eligibility

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

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

Related Conditions & MeSH terms

• Hypercholesterolemia

Intervention

Drug:
• ALN-PCSSC
Single or multiple doses of ALN-PCSSC by subcutaneous (sc) injection
• Sterile Normal Saline (0.9% NaCl)
calculated volume to match active comparator

Locations

Country	Name	City	State
United Kingdom	Covance Clinical Research Unit	Leeds
United Kingdom	Richmond Pharmacology	London

Sponsors (2)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals	The Medicines Company

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes
Secondary	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	No
Secondary	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	No
Secondary	The effect of ALN-PCSSC on plasma levels of PCSK9		Through the sooner of final follow up visit or 180 days post final dose	No