Autoinflammatory diseases including NLRC4-Gain of Function (GOF) also known as AIFEC (autoinflammation with infantile enterocolitis), X-linked Inhibitor of Apoptosis Protein (XIAP) deficiency, or Cell division control protein 42 homolog (CDC42) mutation.
Conditions
Brief summary
Occurrence of disease flare in patients with MAS825 treated patients compared with placebo during Period 2 assessed by Physician's Global Assessment and inflammatory markers
Detailed description
- Confirmation of serological markers of MAS825, - PGA and inflammatory markers at Day 29, end of Period 1 and Period 2, - Serological remission via inflammatory markers, - Glucocorticoid therapy ≤0.2mg/kg/day by end of period 1, - Time to first flare during period 2, - Physician Severity Assessment of Disease Signs and Symptoms scale, - Patient' / Parent's global assessment of disease activity (PPGA) scale
Interventions
DRUG-
DRUGPlacebo to MAS825 100 mg/1 mL Concentrate for solution for infusion / Solution for injection
DRUGMAS825
DRUGCICLOSPORIN
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Occurrence of disease flare in patients with MAS825 treated patients compared with placebo during Period 2 assessed by Physician's Global Assessment and inflammatory markers | — |
Secondary
| Measure | Time frame |
|---|---|
| - Confirmation of serological markers of MAS825, - PGA and inflammatory markers at Day 29, end of Period 1 and Period 2, - Serological remission via inflammatory markers, - Glucocorticoid therapy ≤0.2mg/kg/day by end of period 1, - Time to first flare during period 2, - Physician Severity Assessment of Disease Signs and Symptoms scale, - Patient' / Parent's global assessment of disease activity (PPGA) scale | — |
Countries
Czechia, France, Italy, Spain
Outcome results
None listed