The purpose of this study is to evaluate clinical efficacy and safety of arumakimig (MAS825) compared to placebo in patients with Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome. In addition, the study will evaluate the long-term efficacy, safety and tolerability of arumakimig in this population.
This is a randomized, double-blind, placebo-controlled study with a 52-week duration evaluating the efficacy and safety of arumakimig in participants with VEXAS who are receiving glucocorticoids. Participants will be randomized 1:1 to either arumakimig or placebo. Following the double-blind period, participants may have the option to enter a 2-year (104-week) open-label extension (OLE) period, continuing until Week 156. A 16-week safety follow-up period must be completed after the OLE (up to Week 172) or after the double-blind period (up to Week 68).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
120
Arumakimig Injection
Placebo Injection
Background therapy with glucocorticoids. After the first 2 weeks of the study, participants may begin with glucocorticoid tapering depending on the disease status and the Investigator's judgement.
Number of participants achieving improvement of key VEXAS manifestations and oral glucocorticoid (GC) reduction at Week 52
Response is defined as meeting both criteria: A) Clinical domain: In participants with clinical disease activity in any domain at baseline, complete resolution of clinical manifestations related to active disease in at least one affected domain. In participants with no clinical disease activity in any domain at baseline, continued absence of clinical manifestations in all domains at Week 52. AND B) Protocol-defined glucocorticoid reduction through 52 weeks.
Time frame: From baseline up to Week 52
Number of participants achieving Overall Clinical Response (OCR) at Week 52
Achieving OCR at Week 52 is defined as meeting all the following criteria: * Absence of active inflammatory manifestations of VEXAS * Protocol-defined reduction in oral glucocorticoid. * Protocol-defined reduction in C reactive protein.
Time frame: From baseline up to Week 52
Number of participants achieving resolution of VEXAS manifestations
In participants with clinical disease activity in any domain at baseline, achieving complete resolution of clinical manifestations.
Time frame: From baseline up to Week 52
Number of participants with oral glucocorticoid reduction
Number of participants achieving protocol-defined glucocorticoid dose reduction through 52 weeks.
Time frame: From baseline up to Week 52
Total number of flare-free days over 52 weeks
Total cumulative number of days over 52 weeks meeting the protocol-defined criteria for a flare-free day.
Time frame: Up to 52 weeks
Number of participants achieving Hematologic Improvement - Erythroid (HI-E) during the double-blind treatment period
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Hematologic Improvement - Erythroid (HI-E) during the double-blind 52-week treatment period among participants with baseline hemoglobin within the protocol-defined range, according to modified International Working Group (IWG) and protocol-defined response criteria.
Time frame: From baseline up to Week 52
Number of participants achieving Hematologic Improvement - Platelets (HI-P) during the double-blind treatment period
Hematologic Improvement - Platelets (HI-P) during the double-blind 52-week treatment period among participants with baseline platelets within the protocol-defined range, according to modified International Working Group (IWG) and protocol-defined response criteria.
Time frame: From baseline up to Week 52
Number of participants without worsening disease according to a participant-reported questionnaire
Participants without protocol-defined worsening of disease according to a participant-reported questionnaire.
Time frame: From baseline up to Week 52
Time to death during the double-blind treatment period
Assessment of mortality through Week 52 in each treatment arm.
Time frame: Up to 52 weeks
Number of participants with adverse events (AEs) and serious adverse events (SAEs)
Number of participants with AEs and SAEs, including changes in vital signs, electrocardiograms and laboratory values qualifying and reported as AEs.
Time frame: Up to 172 weeks