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

Sponsor

Zomagen Biosciences, Ltd (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05812781

Collaborator

(none)

Enrollment

Locations

Arms

8.5

Anticipated Duration (Months)

Patients Per Site

0.6

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cryopyrin Associated Periodic Syndrome	Drug: VTX2735 Drug: VTX2735	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

10 participants

Allocation:

Non-Randomized

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Mar 18, 2023

Anticipated Primary Completion Date :

Oct 1, 2023

Anticipated Study Completion Date :

Dec 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort 1	Drug: VTX2735 Dose A
Experimental: Cohort 2	Drug: VTX2735 Dose B

Arm

Intervention/Treatment

Experimental: Cohort 1

Drug: VTX2735

Dose A

Experimental: Cohort 2

Drug: VTX2735

Dose B

Outcome Measures

Primary Outcome Measures

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

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.
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.
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
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
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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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