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

Cryopyrin Associated Periodic Syndrome Clinical Trial

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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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05812781
• Other study ID #: VTX2735-201
• Status: Completed
• Phase: Phase 2
• Start date: March 18, 2023
• Est. completion date: March 6, 2024

Study information

• Verified date: March 2024
• Source: Ventyx Biosciences, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 7
• Est. completion date: March 6, 2024
• Est. primary completion date: March 6, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cryopyrin Associated Periodic Syndrome
• Cryopyrin-Associated Periodic Syndromes
• Syndrome

Intervention

Drug:
• VTX2735
Dose A
• VTX2735
Dose B

Locations

Country	Name	City	State
United States	Local Site # 223	Columbus	Georgia
United States	Local Site # 222	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Zomagen Biosciences, Ltd

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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