Hypercholesterolemia
Conditions
Keywords
LDL-C, RNAi therapeutic
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.
Interventions
DRUGALN-PCSSC
Single or multiple doses of ALN-PCSSC by subcutaneous (sc) injection
DRUGSterile Normal Saline (0.9% NaCl)
calculated volume to match active comparator
Sponsors
The Medicines Company
Alnylam Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
Yes
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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
Countries
United Kingdom
Outcome results
None listed