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

NCT05812781 | Completed Phase 2 FDA Drug

• 1 other identifier: VTX2735-201
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 2

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.

Conditions

Cryopyrin Associated Periodic Syndrome

Keywords

Cryopyrin-Associated Periodic Syndrome; Ventyx; Zomagen; VTX2735; CAPS

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• 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

• 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

• Number of days when the daily KSS is >3

  From Day 1 to completion of treatment with VTX2735, up to Day 28

• Number of days when any single DHAF2 symptom score is >3

  From Day 1 to completion of treatment with VTX2735, up to Day 28

• Maximum severity of any symptom score on DHAF2

  From Day 1 to completion of treatment with VTX2735, up to Day 28

Study Arms (2)

Cohort 1

EXPERIMENTAL

Drug: VTX2735

Cohort 2

EXPERIMENTAL

Drug: VTX2735

Interventions

VTX2735 DRUG

Dose A

Cohort 1

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Zomagen Biosciences Ltd.: lead

Study Sites (2)

Local Site # 222

San Diego, California, 92123, United States

Location

Local Site # 223

Columbus, Georgia, 31904, United States

Location

MeSH Terms

Conditions

Cryopyrin-Associated Periodic Syndromes

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Chronic Inducible Urticaria; Chronic Urticaria; Urticaria; Skin Diseases, Vascular; Cold Urticaria; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Henrik Sonnergren, MD, PhD

  Ventyx Biosciences, Inc

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2023
• First Posted: April 14, 2023
• Study Start: March 18, 2023
• Primary Completion: March 6, 2024
• Study Completion: March 6, 2024
• Last Updated: March 10, 2025

Record last verified: 2025-03

Locations

US (2)