Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334

NCT04086602 | Completed Phase 1

• 1 other identifier: IZD334-001
• Study Type: interventional
• Target: 64
• 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 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.

Conditions

Healthy Volunteers; Cryopyrin Associated Periodic Syndrome

Outcome Measures

Primary Outcomes (6)

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

  Incidence, frequency and severity of treatment emergent adverse events.

  Day 1-8 for SAD

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

  Incidence, frequency and severity of treatment emergent adverse events.

  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

Secondary Outcomes (2)

• Reduction of IL-1 production in stimulated whole blood

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

• Reduction in CAPS symptom scores

  Day 1-15

Study Arms (2)

Single Ascending Dose

EXPERIMENTAL

Once daily oral IZD334 or Placebo

Drug: IZD334; Drug: Placebos

Multiple Ascending Dose

EXPERIMENTAL

Once or twice daily oral IZD334 or Placebo

Drug: IZD334; Drug: Placebos

Interventions

IZD334 DRUG

Active Drug

Multiple Ascending Dose; Single Ascending Dose

Placebos DRUG

Placebo to Match

Multiple Ascending Dose; Single Ascending Dose

Eligibility Criteria

• Age: 18 Years - 65 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
• 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
• \*Patients with a confirmed diagnosis of CAPS (FCAS or MWS) aged 18 to 65 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
• Blood donation or significant blood loss within 60 days prior to the first study drug administration
• 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.

Sponsors & Collaborators

• Inflazome UK Ltd: lead

Study Sites (1)

Nucleus Network

Melbourne, Victoria, Australia

Location

MeSH Terms

Conditions

Cryopyrin-Associated Periodic Syndromes

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Chronic Inducible Urticaria; Chronic Urticaria; Urticaria; Skin Diseases, Vascular; Cold Urticaria; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Jason Lickliter, MBBS, PhD

  Nucleus Network

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Healthy volunteer section is double blind.
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 9, 2019
• First Posted: September 11, 2019
• Study Start: September 13, 2019
• Primary Completion: February 4, 2020
• Study Completion: February 4, 2020
• Last Updated: March 2, 2020

Record last verified: 2020-02

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)