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

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

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 Overview

• Status: Completed
• Conditions: Cryopyrin Associated Periodic Syndrome
• Intervention / Treatment: Drug: VTX2735; Drug: VTX2735

• Study Type: Interventional
• Enrollment (Actual): 7
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • San Diego, California, United States, 92123
      • Local Site # 222
  • Georgia
    • Columbus, Georgia, United States, 31904
      • Local Site # 223

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Zomagen Biosciences, Ltd
• Investigators: Study Director: Matt Cascino, MD, Ventyx Biosciences, Inc

Study record dates

Study Major Dates

• Study Start (Actual): March 18, 2023
• Primary Completion (Actual): March 6, 2024
• Study Completion (Actual): March 6, 2024

Study Registration Dates

• First Submitted: March 28, 2023
• First Submitted That Met QC Criteria: April 11, 2023
• First Posted (Actual): April 14, 2023

Study Record Updates

• Last Update Posted (Actual): March 18, 2024
• Last Update Submitted That Met QC Criteria: March 14, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: CAPS; Ventyx; Cryopyrin-Associated Periodic Syndrome; Zomagen; VTX2735
• Additional Relevant MeSH Terms: Pathologic Processes; Skin Diseases; Immune System Diseases; Hypersensitivity, Immediate; Disease Attributes; Disease; Genetic Diseases, Inborn; Hereditary Autoinflammatory Diseases; Skin Diseases, Genetic; Skin Diseases, Vascular; Hypersensitivity; Urticaria; Chronic Disease; Chronic Inducible Urticaria; Chronic Urticaria; Cold Urticaria; Syndrome; Cryopyrin-Associated Periodic Syndromes
• Other Study ID Numbers: VTX2735-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No