Back to resultsSafety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334
Primary Purpose
Healthy Volunteers, Cryopyrin Associated Periodic Syndrome
Status
Completed
Phase
Phase 1
Locations
Australia
Study Type
Interventional
Intervention
IZD334
Placebos
Sponsored by
About this trial
This is an interventional other trial for Healthy Volunteers
Eligibility 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
- 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.
Sites / Locations
- Nucleus Network
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Single Ascending Dose
Multiple Ascending Dose
Arm Description
Once daily oral IZD334 or Placebo
Once or twice daily oral IZD334 or Placebo
Outcomes
Primary Outcome Measures
Incidence of treatment emergent adverse events [Safety and Tolerability]
Incidence, frequency and severity of treatment emergent adverse events.
Incidence of treatment emergent adverse events [Safety and Tolerability]
Incidence, frequency and severity of treatment emergent adverse events.
Peak plasma concentration (Cmax) single dose
Peak plasma concentration following single dose administration
Area under the plasma concentration versus time curve (AUC)- single dose
AUC following single dose administration
Peak Plasma Concentration (Cmax)-multiple dose
Peak plasma concentration following multiple dose administration
Area under the plasma concentration versus time curve (AUC)- multiple dose
AUC following multiple dose administration
Secondary Outcome Measures
Reduction of IL-1 production in stimulated whole blood
% reduction in IL-1 production in stimulated whole blood as measured by ELISA
Reduction in CAPS symptom scores
Reduction in Physician Assessed CAPS scores based on 8 point questionnaire
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04086602
Brief Title
Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334
Official 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 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
Interventional
2. Study Status
Record Verification Date
February 2020
Overall Recruitment Status
Completed
Study Start Date
September 13, 2019 (Actual)
Primary Completion Date
February 4, 2020 (Actual)
Study Completion Date
February 4, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Inflazome UK Ltd
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Healthy Volunteers, Cryopyrin Associated Periodic Syndrome
7. Study Design
Primary Purpose
Other
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Masking Description
Healthy volunteer section is double blind.
Allocation
Randomized
Enrollment
64 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Single Ascending Dose
Arm Type
Experimental
Arm Description
Once daily oral IZD334 or Placebo
Arm Title
Multiple Ascending Dose
Arm Type
Experimental
Arm Description
Once or twice daily oral IZD334 or Placebo
Intervention Type
Drug
Intervention Name(s)
IZD334
Intervention Description
Active Drug
Intervention Type
Drug
Intervention Name(s)
Placebos
Intervention Description
Placebo to Match
Primary Outcome Measure Information:
Title
Incidence of treatment emergent adverse events [Safety and Tolerability]
Description
Incidence, frequency and severity of treatment emergent adverse events.
Time Frame
Day 1-8 for SAD
Title
Incidence of treatment emergent adverse events [Safety and Tolerability]
Description
Incidence, frequency and severity of treatment emergent adverse events.
Time Frame
Day 1-16 for MAD
Title
Peak plasma concentration (Cmax) single dose
Description
Peak plasma concentration following single dose administration
Time Frame
Day 1-3
Title
Area under the plasma concentration versus time curve (AUC)- single dose
Description
AUC following single dose administration
Time Frame
Day 1-3
Title
Peak Plasma Concentration (Cmax)-multiple dose
Description
Peak plasma concentration following multiple dose administration
Time Frame
Days 1-9
Title
Area under the plasma concentration versus time curve (AUC)- multiple dose
Description
AUC following multiple dose administration
Time Frame
Days 1-9
Secondary Outcome Measure Information:
Title
Reduction of IL-1 production in stimulated whole blood
Description
% reduction in IL-1 production in stimulated whole blood as measured by ELISA
Time Frame
Day 1-3 for SAD and Day 1-9 for MAD]
Title
Reduction in CAPS symptom scores
Description
Reduction in Physician Assessed CAPS scores based on 8 point questionnaire
Time Frame
Day 1-15
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility 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
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jason Lickliter, MBBS, PhD
Organizational Affiliation
Nucleus Network
Official's Role
Principal Investigator
Facility Information:
Facility Name
Nucleus Network
City
Melbourne
State/Province
Victoria
Country
Australia
12. IPD Sharing Statement
Plan to Share IPD
No
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Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334
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