A Study to Evaluate VTX2735 in Patients With Cryopyrin-associated Periodic Syndrome - Clinical Trial for Cryopyrin Associated Periodic Syndrome | NCT05812781

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Primary Purpose

Status

Recruiting

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

VTX2735

About this trial

This is an interventional treatment trial for Cryopyrin Associated Periodic Syndrome focused on measuring Cryopyrin-Associated Periodic Syndrome, Ventyx, Zomagen, VTX2735, CAPS

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Local Site # 222Recruiting
Local Site # 223Recruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Cohort 1

Cohort 2

Arm Description

Outcomes

Primary Outcome Measures

Safety and Tolerability of VTX2735

Incidence and severity of treatment-emergent adverse events (TEAE), serious adverse events (SAE), and discontinuation due to adverse events

Secondary Outcome Measures

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.

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.

Number of days when the daily KSS is >3

Number of multi-system disease flare days as defined by KSS

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

Number of single system disease flare days as defined by KSS

Maximum severity of any symptom score on DHAF2

Maximum single DHAF2 symptom score

Full Information

NCT ID

NCT05812781

First Posted

March 28, 2023

Last Updated

April 11, 2023

Sponsor

Zomagen Biosciences, Ltd

1. Study Identification

Unique Protocol Identification Number

NCT05812781

Brief Title

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

Acronym

Explore

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)

Study Type

Interventional

2. Study Status

Record Verification Date

March 2023

Overall Recruitment Status

Recruiting

Study Start Date

March 18, 2023 (Actual)

Primary Completion Date

October 2023 (Anticipated)

Study Completion Date

December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Zomagen Biosciences, Ltd

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

No

Data Monitoring Committee

No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cryopyrin Associated Periodic Syndrome

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Sequential Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Cohort 1

Arm Type

Experimental

Arm Title

Cohort 2

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

VTX2735

Intervention Description

Dose A

Intervention Type

Drug

Intervention Name(s)

VTX2735

Intervention Description

Dose B

Primary Outcome Measure Information:

Title

Safety and Tolerability of VTX2735

Description

Incidence and severity of treatment-emergent adverse events (TEAE), serious adverse events (SAE), and discontinuation due to adverse events

Time Frame

From the initial administration of VTX2735 through study completion, up to 10 weeks

Secondary Outcome Measure Information:

Title

Mean change in the mean key symptom score (KSS) derived from the Daily Health Assessment Form, Second Generation (DHAF2) from baseline

Description

Assess the change from baseline in disease activity using DHAF2 and KSS.

Time Frame