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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04015076




Registration number
NCT04015076
Ethics application status
Date submitted
8/07/2019
Date registered
10/07/2019
Date last updated
27/03/2020

Titles & IDs
Public title
Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With Inzomelid
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 Inzomelid in Healthy Adult Participants as Well as an Open Label Cohort to Confirm the Safety, Pharmacokinetics, and Pharmacodynamics of Inzomelid in Adult Patients With Cryopyrin-Associated Periodic Syndromes
Secondary ID [1] 0 0
IZD174-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Healthy 0 0
Condition category
Condition code

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

Experimental: Single Ascending Dose - Inzomelid or Placebo

Experimental: Multiple Ascending Dose - Inzomelid or Placebo

Experimental: Patients with CAPS - Inzomelid Open Label


Treatment: Drugs: Inzomelid
Active Drug

Treatment: Drugs: Placebo
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 advert events [Safety and Tolerability]
Timepoint [1] 0 0
Day 1-8 for SAD, Day 1-16 for MAD
Primary outcome [2] 0 0
Peak Plasma Concentration (Cmax)-single dose
Timepoint [2] 0 0
Day 1-3
Primary outcome [3] 0 0
Area under the plasma concentration versus time curve (AUC)- single dose
Timepoint [3] 0 0
Day 1-3
Primary outcome [4] 0 0
Peak Plasma Concentration (Cmax)-multiple dose
Timepoint [4] 0 0
Days 1-9
Primary outcome [5] 0 0
Area under the plasma concentration versus time curve (AUC)- multiple dose
Timepoint [5] 0 0
Days 1-9
Primary outcome [6] 0 0
Peak Plasma Concentration (Cmax)-multiple dose
Timepoint [6] 0 0
Days 1-16
Primary outcome [7] 0 0
Area under the plasma concentration versus time curve (AUC)- multiple dose
Timepoint [7] 0 0
Days 1-16
Secondary outcome [1] 0 0
Pharmacodynamic activity
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
Timepoint [2] 0 0
Days 1-15

Eligibility
Key inclusion criteria
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;

Inclusion Criteria- CAPS Patients:

* Patients with a confirmed diagnosis of CAPS (FCAS, NOMID, or MWS) aged 18 to 70 years (inclusive at the time of informed consent);
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Exclusion Criteria- Healthy Volunteers:

* 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;

Exclusion Criteria- 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.

Participants who have a positive QuantiFERON test with documentation of BCG vaccination, who are at low environmental risk for TB infection or reactivation, and have a negative chest X-ray can be included;

* Pregnant or lactating at Screening or planning to becomepregnant (self or partner) at any time during the study, including the follow-up period;

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
16/07/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/03/2020
Sample size
Target
Accrual to date
Final
80
Recruitment in Australia
Recruitment state(s)
QLD
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 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.
Trial website
https://clinicaltrials.gov/study/NCT04015076
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ben Snyder, MB, BS
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

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04015076