A Study to Evaluate VTX2735 in Patients With Cryopyrin-associated Periodic Syndrome

Phase 2

Completed

Brief Summary: 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.

Recruitment & Eligibility

Inclusion Criteria

At least 18 years of age
Diagnosis of CAPS and FCAS subtype and mild clinical phenotype
At least one flare during screening/washout
Women must not be of childbearing potential or must agree to use two methods of highly effective contraception during the study and for 30 days after the last dose of the study product
Men with a partner who is of childbearing potential must agree to use condoms plus another highly effective form of birth control during the study and for 90 days after the last dose of study product

Exclusion Criteria

Unwilling to comply with washout of anti-IL-1 therapy and tolerate symptoms of disease flare
Moderate or severe CAPS manifestations or significant damage or any CAPS feature that presents a contraindication to washout of anti-IL-1 therapy
Has a history of chronic or recurrent infectious disease
Has a known immune deficiency or is immunocompromised
Has hepatitis B or hepatitis C infection, human immunodeficiency virus (HIV), acquired immunodeficiency syndrome (AIDS), or active tuberculosis (TB)
Has another clinically important medical disorder that would compromise safety

Arm && Interventions

Group	Intervention	Description
Cohort 1	VTX2735	-
Cohort 2	VTX2735	-

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability of VTX2735	From the initial administration of VTX2735 through study completion, up to 10 weeks	Incidence and severity of treatment-emergent adverse events (TEAE), serious adverse events (SAE), and discontinuation due to adverse events

Secondary Outcome Measures

Name	Time	Method
Number of days when any single DHAF2 symptom score is >3	From Day 1 to completion of treatment with VTX2735, up to Day 28	Number of single system disease flare days as defined by KSS
Number of days when the daily KSS is >3	From Day 1 to completion of treatment with VTX2735, up to Day 28	Number of multi-system disease flare days as defined by KSS
Mean change in the mean key symptom score (KSS) derived from the Daily Health Assessment Form, Second Generation (DHAF2) from baseline	From Day 1 to completion of treatment with VTX2735, up to Day 28	Assess the change from baseline in disease activity using DHAF2 and KSS.
Maximum severity of any symptom score on DHAF2	From Day 1 to completion of treatment with VTX2735, up to Day 28	Maximum single DHAF2 symptom score
Achievement of 30%, 50%, or 75% improvement in mean KSS from baseline	From Day 1 to completion of treatment with VTX2735, up to Day 28	Proportion of patients achieving 30%, 50%, or 75% improvement from baseline in disease activity using DHAF2 and KSS.