Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With Inzomelid (NCT04015076) | Clinical Trial Compass
CompletedPhase 1
Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With Inzomelid
Australia80 participantsStarted 2019-07-16
Plain-language 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.
Who can participate
Age range
18 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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);
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 w…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of treatment emergent advert events [Safety and Tolerability]
Timeframe: Day 1-8 for SAD, Day 1-16 for MAD
2
Peak Plasma Concentration (Cmax)-single dose
Timeframe: Day 1-3
3
Area under the plasma concentration versus time curve (AUC)- single dose
Timeframe: Day 1-3
4
Peak Plasma Concentration (Cmax)-multiple dose
Timeframe: Days 1-9
5
Area under the plasma concentration versus time curve (AUC)- multiple dose
Timeframe: Days 1-9
6
Peak Plasma Concentration (Cmax)-multiple dose
Timeframe: Days 1-16
7
Area under the plasma concentration versus time curve (AUC)- multiple dose