None listed
Conditions
Brief summary
This study aims to evaluate the safety and effectiveness of SIR9900 in adults with VEXAS syndrome. Participants will be randomly assigned to receive either SIR9900 or a matching placebo in addition to their standard medical care. The study will compare whether treatment with SIR9900 improves disease activity and clinical outcomes while remaining safe and well tolerated. Information collected during the study will help determine whether SIR9900 may be a potential treatment option for people with VEXAS syndrome.
Interventions
Participants will be randomised to receive SIR9900 20 mg, SIR9900 30 mg, or matching placebo orally once daily, together with permitted background treatment. Double-blind treatment will continue for up to 24 weeks, after which eligible participants may enter an open-label treatment period through Week 48. Treatment adherence is assessed at scheduled study visits using study treatment records. For this study, standard care consists of protocol-permitted background glucocorticoid treatment managed
Participants will be randomised to receive SIR9900 20 mg, SIR9900 30 mg, or matching placebo orally once daily, together with permitted background treatment. Double-blind treatment will continue for up to 24 weeks, after which eligible participants may enter an open-label treatment period through Week 48. Treatment adherence is assessed at scheduled study visits using study treatment records. For this study, standard care consists of protocol-permitted background glucocorticoid treatment managed by the Investigator according to the participant’s clinical needs and the study protocol.
Sponsors
Sironax USA, Inc.
Study design
Allocation
Randomised controlled trial
Primary purpose
Treatment
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Adults aged 18 years or older. Genetically confirmed VEXAS syndrome. Active disease meeting the protocol-defined eligibility criteria. Stable background treatment, if applicable. Able and willing to provide written informed consent and comply with study procedures.
Exclusion criteria
Prohibited concomitant medications include hypomethylating agents; non-glucocorticoid anti-inflammatory and haematological support therapies; antiplatelet therapy other than permitted low-dose aspirin; other investigational therapies; CYP1A2 substrates with a narrow therapeutic window; strong CYP3A inhibitors or inducers; strong CYP2C8 inhibitors; and non-prescription or herbal products with clinically relevant interaction potential.
Outcome results
None listed