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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

7 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-03-18

Study Completion Date

2024-03-06

Brief Summary

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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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Detailed Description

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Conditions

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Cryopyrin Associated Periodic Syndrome

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Group Type EXPERIMENTAL

VTX2735

Intervention Type DRUG

Dose A

Cohort 2

Group Type EXPERIMENTAL

VTX2735

Intervention Type DRUG

Dose B

Interventions

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VTX2735

Dose A

Intervention Type DRUG

VTX2735

Dose B

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No