Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
Status:
Recruiting
Trial end date:
2022-07-30
Target enrollment:
Participant gender:
Summary
This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with
monogenic, interleukin-18 (IL 18) driven autoinflammation due to Nucleotide-binding
oligomerization domain, leucine-rich repeat and caspase recruiting domain (CARD domain)
containing 4 (NLRC4) - Macrophage activation syndrome (MAS) mutation (NLRC4-MAS mutation) or
X-linked inhibitor of apoptosis (XIAP) deficiency. Because of the likelihood for pathogenic
IL-18 in certain monogenic diseases, patients known to harbor deleterious mutations in
NLRC4-MAS or XIAP and who have a history of ongoing inflammation will be enrolled if they
have ferritin ≥ 500 ng/mL or persistent C reactive protein (CRP) elevation ≥ 2 times the
upper limit of normal (ULN) and the patients should have a Modified Autoinflammatory Disease
Activity Index (mAIDAI) ≥ 4.