A Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of SIR9900 in Patients with VEXAS Syndrome (SIVEX)

Condition studied

VEXAS Syndrome

Investigational medicinal product(s)

Eligibility

Primary endpoint

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.

Endpoint detail

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