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

Study Overview
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Brief Summary
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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.

Official Title
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A Phase 2A, Single-Arm Study to Evaluate the Safety and Clinical Activity of VTX2735 in Participants With Cryopyrin-Associated Periodic Syndrome (CAPS)

Conditions
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Cryopyrin Associated Periodic Syndrome

Intervention / Treatment
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* Drug: VTX2735
* Drug: VTX2735


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1<br> | Drug: VTX2735<br>* Dose A<br>|
| Experimental: Cohort 2<br> | Drug: VTX2735<br>* Dose B<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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 | 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 | 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 | From Day 1 to completion of treatment with VTX2735, up to Day 28 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Zomagen, Ventyx, Cryopyrin-Associated Periodic Syndrome, VTX2735, CAPS