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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single Ascending Dose | Experimental | Once daily oral IZD334 or Placebo |
|
| Multiple Ascending Dose | Experimental | Once or twice daily oral IZD334 or Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IZD334 | Drug | Active Drug |
| |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Reduction of IL-1 production in stimulated whole blood | % reduction in IL-1 production in stimulated whole blood as measured by ELISA | Day 1-3 for SAD and Day 1-9 for MAD] |
| Reduction in CAPS symptom scores |
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Inclusion Criteria: (Healthy Volunteers)
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)
Exclusion Criteria: (CAPS Patients)
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| Name | Affiliation | Role |
|---|---|---|
| Jason Lickliter, MBBS, PhD | Nucleus Network | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nucleus Network | Melbourne | Victoria | Australia |
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| ID | Term |
|---|---|
| D056587 | Cryopyrin-Associated Periodic Syndromes |
| ID | Term |
|---|---|
| D056660 | Hereditary Autoinflammatory Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
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Healthy volunteer section is double blind.
| Placebos |
| Drug |
Placebo to Match |
|
Reduction in Physician Assessed CAPS scores based on 8 point questionnaire
| Day 1-15 |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D000094482 | Chronic Inducible Urticaria |
| D000080223 | Chronic Urticaria |
| D014581 | Urticaria |
| D017445 | Skin Diseases, Vascular |
| D000096703 | Cold Urticaria |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |