Cryopyrin Associated Periodic Syndrome, Healthy Volunteers
Conditions
Brief summary
This is a first in human (FIH), single-centre, double -blind, randomised, cross-over, SAD followed by a MAD study of IZD334 conducted in healthy adult participants as well as an open-label cohort in adult patients with CAPS. The study is designed to evaluate the safety, tolerability, PK, PD, and food effect of IZD334 in healthy adult participants, and to evaluate the safety, tolerability, PK, PD, and preliminary clinical efficacy of IZD334 in adult patients with CAPS.
Interventions
DRUGIZD334
Active Drug
DRUGPlacebos
Placebo to Match
Sponsors
Inflazome UK Ltd
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
DOUBLE (Subject, Investigator)
Masking description
Healthy volunteer section is double blind.
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
Yes
Inclusion criteria
(Healthy Volunteers) * Healthy male or female volunteers, aged 18 to 65 years (inclusive at the time of informed consent) * Participants must be in good general health, with no significant medical history, have no clinically significant abnormalities on physical examination at Screening and/or before administration of the initial dose of study drug * Participants must have a Body Mass Index (BMI) between ≥18.0 and ≤32.0 kg/m2 at Screening * Participants must have clinical laboratory values within normal range as specified by the testing laboratory, unless deemed not clinically significant by the Investigator or delegate Inclusion Criteria: (CAPS Patients) \*Patients with a confirmed diagnosis of CAPS (FCAS or MWS) aged 18 to 65 years (inclusive at the time of informed consent)
Exclusion criteria
(Healthy volunteer) * Pregnant or lactating at Screening or planning to become pregnant (self or partner) at any time during the study, including the follow-up period * Prior or ongoing medical conditions, medical history, physical findings, or laboratory abnormality that, in the Investigator's (or delegate's) opinion, could adversely affect the safety of the participant * Presence of any underlying physical or psychological medical condition that, in the opinion of the Investigator, would make it unlikely that the participant will comply with the protocol or complete the study per protocol * Blood donation or significant blood loss within 60 days prior to the first study drug administration
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Peak plasma concentration (Cmax) single dose | Day 1-3 | Peak plasma concentration following single dose administration |
| Peak Plasma Concentration (Cmax)-multiple dose | Days 1-9 | Peak plasma concentration following multiple dose administration |
| Area under the plasma concentration versus time curve (AUC)- multiple dose | Days 1-9 | AUC following multiple dose administration |
| Incidence of treatment emergent adverse events [Safety and Tolerability] | Day 1-8 for SAD | Incidence, frequency and severity of treatment emergent adverse events. |
| Area under the plasma concentration versus time curve (AUC)- single dose | Day 1-3 | AUC following single dose administration |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Reduction in CAPS symptom scores | Day 1-15 | Reduction in Physician Assessed CAPS scores based on 8 point questionnaire |
| Reduction of IL-1 production in stimulated whole blood | Day 1-3 for SAD and Day 1-9 for MAD] | % reduction in IL-1 production in stimulated whole blood as measured by ELISA |
Countries
Australia
Outcome results
None listed