This is a first in human (FIH), single-centre, double blind, randomised, cross-over, SAD followed by a MAD study of Inzomelid. The study is designed to evaluate the safety, tolerability, PK, PD, and food effect of Inzomelid in healthy adult participants and to evaluate the safety, tolerability, PK, PD, and preliminary clinical efficacy of Inzomelid in adult patients with CAPS. Oversight will be provided by a safety monitoring committee (SMC) comprised of the Investigator, the Sponsor's Medical Monitor and an Independent Medical Monitor.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
OTHER
Masking
DOUBLE
Enrollment
80
Nucleus Network
Melbourne, Queensland, Australia
Incidence of treatment emergent advert events [Safety and Tolerability]
Incidence, frequency and severity of treatment emergent advert events
Time frame: Day 1-8 for SAD, Day 1-16 for MAD
Peak Plasma Concentration (Cmax)-single dose
Peak plasma concentration following single dose administration
Time frame: Day 1-3
Area under the plasma concentration versus time curve (AUC)- single dose
AUC following single dose administration
Time frame: Day 1-3
Peak Plasma Concentration (Cmax)-multiple dose
Peak plasma concentration following multiple dose administration
Time frame: Days 1-9
Area under the plasma concentration versus time curve (AUC)- multiple dose
AUC following multiple dose administration
Time frame: Days 1-9
Peak Plasma Concentration (Cmax)-multiple dose
Peak plasma concentration following multiple dose administration
Time frame: Days 1-16
Area under the plasma concentration versus time curve (AUC)- multiple dose
AUC following multiple dose administration
Time frame: Days 1-16
Pharmacodynamic activity
NLRP3 Inhibition in whole blood
Time frame: Day 1-3 for SAD and Day 1-9 for MAD
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Reduction in CAPS symptom scores
Reduction in Physician Assessed CAPS scores based on 8 point questionnaire
Time frame: Days 1-15