Texas Children's Cancer and Hematology Center Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations (MASter-1) 

Description

This study is a Phase 2 trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in patients with NLRC4-GOF, XIAP deficiency, or CDC42 mutations.

Eligibility Criteria

• Must weigh at least 3 kg
• Ages Eligible for Study: Children, Adult, Older Adult
• Sexes Eligible for Study: All
• Cohort 1 specific inclusion criteria:
  • Patients with a genetic diagnosis of either NLRC4-GOF, XIAP deficiency, or CDC42 mutation
  • Clinical history and investigations consistent with autoinflammation and infantile enterocolitis (AIFEC/NLRC4-GOF), XIAP or CDC42. XIAP patients must have persistent disease or be resistant to escalating therapy.
  • At first treatment, evidence of active disease as assessed by inflammatory markers and PGA
• Cohort 2 specific inclusion criteria:
  • Patients with a genetic diagnosis of NLRC4-GOF, XIAP deficiency, or CDC42 mutations who are being treated with MAS825 in a Novartis Managed Access Program (MAP).

Detailed inclusion and exclusion criteria as listed on clinicaltrials.gov