Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study in Healthy Volunteers and CAPS Patients

Overview

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.

Full Title of Study: “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”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Other
    • Masking: Double (Participant, Investigator)
  • Study Primary Completion Date: February 2020

Interventions

  • Drug: IZD334
    • Active Drug
  • Drug: Placebos
    • Placebo to Match

Arms, Groups and Cohorts

  • Experimental: Single Ascending Dose
    • Once daily oral IZD334 or Placebo
  • Experimental: Multiple Ascending Dose
    • Once or twice daily oral IZD334 or Placebo
  • Experimental: CAPS Patients
    • Twice daily oral IZD334

Clinical Trial Outcome Measures

Primary Measures

  • Incidence of treatment emergent adverse events [Safety and Tolerability]
    • Time Frame: Day 1-8 for SAD
    • Incidence, frequency and severity of treatment emergent adverse events.
  • Incidence of treatment emergent adverse events [Safety and Tolerability]
    • Time Frame: Day 1-16 for MAD
    • Incidence, frequency and severity of treatment emergent adverse events.
  • Peak plasma concentration (Cmax) single dose
    • Time Frame: Day 1-3
    • Peak plasma concentration following single dose administration
  • Area under the plasma concentration versus time curve (AUC)- single dose
    • Time Frame: Day 1-3
    • AUC following single dose administration
  • Peak Plasma Concentration (Cmax)-multiple dose
    • Time Frame: Days 1-9
    • Peak plasma concentration following multiple dose administration
  • Area under the plasma concentration versus time curve (AUC)- multiple dose
    • Time Frame: Days 1-9
    • AUC following multiple dose administration

Secondary Measures

  • Reduction of IL-1 production in stimulated whole blood
    • Time Frame: Day 1-3 for SAD and Day 1-9 for MAD]
    • % reduction in IL-1 production in stimulated whole blood as measured by ELISA
  • Reduction in CAPS symptom scores
    • Time Frame: Day 1-15
    • Reduction in Physician Assessed CAPS scores based on 8 point questionnaire

Participating in This Clinical Trial

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

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.

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 65 Years

Are Healthy Volunteers Accepted: Accepts Healthy Volunteers

Investigator Details

  • Lead Sponsor
    • Inflazome UK Ltd
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Jason Lickliter, MBBS, PhD, Principal Investigator, Nucleus Network
  • Overall Contact(s)
    • Jeff Thompson, PhD, MBA, +44(0)1223 751549, j.thompson@inflazome.com

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