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

• ClinicalTrials.gov Identifier: NCT05812781
• Recruitment Status: Completed
• First Posted: April 14, 2023
• Last Update Posted: March 18, 2024
• Sponsor: Zomagen Biosciences, Ltd
• Information provided by (Responsible Party): Ventyx Biosciences, Inc ( Zomagen Biosciences, Ltd )

Tracking Information

• First Submitted Date: March 28, 2023
• First Posted Date: April 14, 2023
• Last Update Posted Date: March 18, 2024
• Actual Study Start Date: March 18, 2023
• Actual Primary Completion Date: March 6, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 11, 2023)

Safety and Tolerability of VTX2735 [ Time Frame: 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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: April 11, 2023)

• Mean change in the mean key symptom score (KSS) derived from the Daily Health Assessment Form, Second Generation (DHAF2) from baseline [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: From Day 1 to completion of treatment with VTX2735, up to Day 28 ]
  Maximum single DHAF2 symptom score

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study to Evaluate VTX2735 in Patients With Cryopyrin-associated Periodic Syndrome
• 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)

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.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Non-Randomized; Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Cryopyrin Associated Periodic Syndrome

Intervention

• Drug: VTX2735
  Dose A
• Drug: VTX2735
  Dose B

Study Arms

• Experimental: Cohort 1
  Intervention: Drug: VTX2735
• Experimental: Cohort 2
  Intervention: Drug: VTX2735

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 14, 2024): 7
• Original Estimated Enrollment (submitted: April 11, 2023): 10
• Actual Study Completion Date: March 6, 2024
• Actual Primary Completion Date: March 6, 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT05812781
• Other Study ID Numbers: VTX2735-201
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: Ventyx Biosciences, Inc ( Zomagen Biosciences, Ltd )
• Original Responsible Party: Same as current
• Current Study Sponsor: Zomagen Biosciences, Ltd
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Matt Cascino, MD; Ventyx Biosciences, Inc

• PRS Account: Ventyx Biosciences, Inc
• Verification Date: March 2024