Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With Inzomelid

NCT04015076 | Completed Phase 1

• 1 other identifier: IZD174-001
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

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.

Conditions

Healthy

Outcome Measures

Primary Outcomes (7)

• Incidence of treatment emergent advert events [Safety and Tolerability]

  Incidence, frequency and severity of treatment emergent advert events

  Day 1-8 for SAD, Day 1-16 for MAD

• Peak Plasma Concentration (Cmax)-single dose

  Peak plasma concentration following single dose administration

  Day 1-3

• Area under the plasma concentration versus time curve (AUC)- single dose

  AUC following single dose administration

  Day 1-3

• Peak Plasma Concentration (Cmax)-multiple dose

  Peak plasma concentration following multiple dose administration

  Days 1-9

• Area under the plasma concentration versus time curve (AUC)- multiple dose

  AUC following multiple dose administration

  Days 1-9

• Peak Plasma Concentration (Cmax)-multiple dose

  Peak plasma concentration following multiple dose administration

  Days 1-16

• Area under the plasma concentration versus time curve (AUC)- multiple dose

  AUC following multiple dose administration

  Days 1-16

Secondary Outcomes (2)

• Pharmacodynamic activity

  Day 1-3 for SAD and Day 1-9 for MAD

• Reduction in CAPS symptom scores

  Days 1-15

Study Arms (3)

Single Ascending Dose

EXPERIMENTAL

Inzomelid or Placebo

Drug: Inzomelid; Drug: Placebo

Multiple Ascending Dose

EXPERIMENTAL

Inzomelid or Placebo

Drug: Inzomelid; Drug: Placebo

Patients with CAPS

EXPERIMENTAL

Inzomelid Open Label

Drug: Inzomelid

Interventions

Inzomelid DRUG

Active Drug

Multiple Ascending Dose; Patients with CAPS; Single Ascending Dose

Placebo DRUG

Placebo to Match

Multiple Ascending Dose; Single Ascending Dose

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;
• \* Patients with a confirmed diagnosis of CAPS (FCAS, NOMID, or MWS) aged 18 to 70 years (inclusive at the time of informed consent);

You may not qualify if:

• 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;
• 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;

Sponsors & Collaborators

• Inflazome UK Ltd: lead

Study Sites (1)

Nucleus Network

Melbourne, Queensland, Australia

Location

Study Officials

• Ben Snyder, MB, BS

  Nucleus Network

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 8, 2019
• First Posted: July 10, 2019
• Study Start: July 16, 2019
• Primary Completion: March 23, 2020
• Study Completion: March 23, 2020
• Last Updated: March 27, 2020

Record last verified: 2020-02

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)