A first in human, single dose trial of PCSK9 inhibitor NNC0385-0434
Research type
Research Study
Full title
A first in human dose trial investigating safety, tolerability and pharmacokinetics of single doses of NNC0385-0434 in healthy subjects and patients with hypercholesterolaemia
IRAS ID
265192
Contact name
Pablo ForteSoto
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2019-001746-18
Duration of Study in the UK
0 years, 6 months, 2 days
Research summary
This is a first human dose (FHD), Phase I, single-centre, randomised, double-blinded within cohorts, placebo-controlled, single ascending dose and sequential group trial in 24 healthy male volunteers and 15 male patients suffering from hypercholesteremia.
The main purpose of the trial/study is to investigate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and cholesterol lowering effect of NNC0385-0434 when applied under the skin by injection (subcutaneous administration), in both healthy volunteers and in patients with high cholesterol (hypercholesteremia) that are being treated with another type of cholesterol-lowering drug (statins). PK refers to how the drug is taken up into and distrubuted throughout the body; broken down and finally removed from the body, whereas PD refers to the effect of the drug on the body and how it works to bring about this effect.
The study will comprise of up to three dose-increasing cohorts (groups), each with 8 healthy participants that are randomised (randomly allocated to groups) so that for every three receiving NNC0385-0434, one participant will receive placebo (a substance that has no therapeutic effect and is used as a control in testing new drugs). Following these increasing dose cohorts, an additional cohort of 15 patients, suffering from hypercholesterolemia and currently being treated with statin therapy, will start. This cohort will be randomised so that for each two patients receiving NNC0385-0434, one will receive placebo.
The total trial duration for the individual participant will be approximately 16 weeks. The trial includes a screening period of up to 6 weeks before randomisation (Day 1), a stay at the clinic for 9 days after the injection, and a 10-week post-treatment period.
As this is the first study with NNC0385-0434 in humans, no human safety data is available yet. Based on the nonclinical safety data, no clinically significant undesirable effects are expected.
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
19/SC/0251
Date of REC Opinion
15 Aug 2019
REC opinion
Further Information Favourable Opinion