This is a study to understand if taking VTX2735 is safe and effective in participants diagnosed with Cryopyrin-Associated Period Syndrome (CAPS). Approximately 10 patients will take VTX2735 Dose A or VTX2735 Dose B. The study consists of a screening/washout period of up to 28 weeks, a 2 week treatment period, a treatment withdrawal period of up to 2 weeks, another 2 week treatment period, and a 4 week follow up period. The maximum length of treatment is 4 weeks.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
7
Local Site # 222
San Diego, California, United States
Local Site # 223
Columbus, Georgia, United States
Safety and Tolerability of VTX2735
Incidence and severity of treatment-emergent adverse events (TEAE), serious adverse events (SAE), and discontinuation due to adverse events
Time frame: From the initial administration of VTX2735 through study completion, up to 10 weeks
Mean change in the mean key symptom score (KSS) derived from the Daily Health Assessment Form, Second Generation (DHAF2) from baseline
Assess the change from baseline in disease activity using DHAF2 and KSS.
Time frame: From Day 1 to completion of treatment with VTX2735, up to Day 28
Achievement of 30%, 50%, or 75% improvement in mean KSS from baseline
Proportion of patients achieving 30%, 50%, or 75% improvement from baseline in disease activity using DHAF2 and KSS.
Time frame: From Day 1 to completion of treatment with VTX2735, up to Day 28
Number of days when the daily KSS is >3
Number of multi-system disease flare days as defined by KSS
Time frame: From Day 1 to completion of treatment with VTX2735, up to Day 28
Number of days when any single DHAF2 symptom score is >3
Number of single system disease flare days as defined by KSS
Time frame: From Day 1 to completion of treatment with VTX2735, up to Day 28
Maximum severity of any symptom score on DHAF2
Maximum single DHAF2 symptom score
Time frame: From Day 1 to completion of treatment with VTX2735, up to Day 28
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.