The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04086602




Registration number
NCT04086602
Ethics application status
Date submitted
9/09/2019
Date registered
11/09/2019
Date last updated
2/03/2020

Titles & IDs
Public title
Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334
Scientific title
A Phase 1, Randomised, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Determine the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Food Effect of IZD334 in Healthy Adult Participants as Well as an Open-label Cohort to Confirm the Safety, Pharmacokinetics, and Pharmacodynamics in Adult Patients With Cryopyrin-Associated Periodic Syndromes
Secondary ID [1] 0 0
IZD334-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Healthy Volunteers 0 0
Cryopyrin Associated Periodic Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Skin 0 0 0 0
Dermatological conditions
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - IZD334
Treatment: Drugs - Placebos

Experimental: Single Ascending Dose - Once daily oral IZD334 or Placebo

Experimental: Multiple Ascending Dose - Once or twice daily oral IZD334 or Placebo


Treatment: Drugs: IZD334
Active Drug

Treatment: Drugs: Placebos
Placebo to Match

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment emergent adverse events [Safety and Tolerability] - Incidence, frequency and severity of treatment emergent adverse events.
Timepoint [1] 0 0
Day 1-8 for SAD
Primary outcome [2] 0 0
Incidence of treatment emergent adverse events [Safety and Tolerability] - Incidence, frequency and severity of treatment emergent adverse events.
Timepoint [2] 0 0
Day 1-16 for MAD
Primary outcome [3] 0 0
Peak plasma concentration (Cmax) single dose - Peak plasma concentration following single dose administration
Timepoint [3] 0 0
Day 1-3
Primary outcome [4] 0 0
Area under the plasma concentration versus time curve (AUC)- single dose - AUC following single dose administration
Timepoint [4] 0 0
Day 1-3
Primary outcome [5] 0 0
Peak Plasma Concentration (Cmax)-multiple dose - Peak plasma concentration following multiple dose administration
Timepoint [5] 0 0
Days 1-9
Primary outcome [6] 0 0
Area under the plasma concentration versus time curve (AUC)- multiple dose - AUC following multiple dose administration
Timepoint [6] 0 0
Days 1-9
Secondary outcome [1] 0 0
Reduction of IL-1 production in stimulated whole blood - % reduction in IL-1 production in stimulated whole blood as measured by ELISA
Timepoint [1] 0 0
Day 1-3 for SAD and Day 1-9 for MAD]
Secondary outcome [2] 0 0
Reduction in CAPS symptom scores - Reduction in Physician Assessed CAPS scores based on 8 point questionnaire
Timepoint [2] 0 0
Day 1-15

Eligibility
Key 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

(CAPS Patients)

*Patients with a confirmed diagnosis of CAPS (FCAS or MWS) aged 18 to 65 years (inclusive
at the time of informed consent)
Minimum age
18 Years
Maximum age
65 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key 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

(CAPS Patients)

- Live vaccinations within 3 months prior to Screening, for the duration of the study
and for up to 3 months following the last dose of study drug;

- Positive QuantiFERON test at the Screening visit or within 2 months prior to
Screening.

Study design
Purpose of the study
Other
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Nucleus Network - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Inflazome UK Ltd
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT04086602
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Jason Lickliter, MBBS, PhD
Address 0 0
Nucleus Network
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications