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Completed

NCT Number: NCT05812781

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

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.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Local Site # 222, San Diego, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

VTX2735

Drug

Dose A

Primary outcomes

  1. 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

Secondary outcomes

  1. 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.

  2. 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.

  3. 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

  4. 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

  5. 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

Sponsors and collaborators

Lead sponsor

Zomagen Biosciences Ltd.

Industry

Registry information

Official study title

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

Acronym: Explore

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
Apr 14, 2023
Registry last updated
Mar 10, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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