VEXAS Syndrome
Conditions
Brief summary
Part A, Dose Ranging Study: Incidence and severity of Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and clinically significant laboratory abnormalities; Proportion of participants with Early Treatment Failure defined as the inability to taper GC to ≤10 mg/day, early termination before OCR or the occurrence of a protocol-defined flare. Part B, Efficacy and Safety Study: Proportion of participants achieving OCR up to end of week 24.
Detailed description
Mean plasma conc. of SIR9900 at week 1, 12, 24, 48, 52; CRP, ESR, Ferritin up to end of week 52 Proportion of pts with OCR up to end of week 24: Incidence and severity of AEs and SAEs, death; Clinically significant abnormalities, vital signs, laboratory tests, 12-lead ECGs; Cumulative GC dose; Proportion of pts with each best response category; Nr of flare-free days with GC dose ≤10 mg/day; Erythroid (HI-E) at any time among pts with baseline hemoglobin <10 g/dL and Platelets (HI-P) at any t
Interventions
DRUGSIR9900 10mg
DRUGSir9900 Placebo
Sponsors
Sironax USA Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A, Dose Ranging Study: Incidence and severity of Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and clinically significant laboratory abnormalities; Proportion of participants with Early Treatment Failure defined as the inability to taper GC to ≤10 mg/day, early termination before OCR or the occurrence of a protocol-defined flare. Part B, Efficacy and Safety Study: Proportion of participants achieving OCR up to end of week 24. | — |
Secondary
| Measure | Time frame |
|---|---|
| Mean plasma conc. of SIR9900 at week 1, 12, 24, 48, 52; CRP, ESR, Ferritin up to end of week 52 Proportion of pts with OCR up to end of week 24: Incidence and severity of AEs and SAEs, death; Clinically significant abnormalities, vital signs, laboratory tests, 12-lead ECGs; Cumulative GC dose; Proportion of pts with each best response category; Nr of flare-free days with GC dose ≤10 mg/day; Erythroid (HI-E) at any time among pts with baseline hemoglobin <10 g/dL and Platelets (HI-P) at any t | — |
Outcome results
None listed