MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1 beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2. Clinical trials are currently examining its efficacy in other diseases associated with elevations of these cytokines, including NLRC4-associated disease and hidradenitis suppurativa. This study proposes to assess the effectiveness of MAS825 in a single patient with XLP-2, who has previously demonstrated response to blockade of IL-1 beta and IL-18. Given the lack of alternative pharmaceutical options for XLP-2, this represents the only known medication option that avoids the toxicity associated with high-dose corticosteroids and the morbidity associated with hematopoietic stem cell transplantation.
Age range
13 Years – 14 Years
Sex
MALE
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A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Impact of MAS825 on Physician Global Assessment Score
Timeframe: 5 years
Impact of MAS825 on Physician Severity Assessment of Disease Signs and Symptoms
Timeframe: 5 years
Patient/Parent Global Assessment of Disease Activity
Timeframe: 5 years