---
title: A Study to Evaluate VTX2735 in Patients With Cryopyrin-associated Periodic Syndrome
nct_id: NCT05812781
overall_status: COMPLETED
phase: PHASE2
sponsor: Zomagen Biosciences Ltd.
study_type: INTERVENTIONAL
primary_condition: Cryopyrin Associated Periodic Syndrome
countries: United States
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT05812781.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT05812781"
ct_last_update_post_date: 2025-03-10
last_seen_at: "2026-05-12T06:29:53.385Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# A Study to Evaluate VTX2735 in Patients With Cryopyrin-associated Periodic Syndrome

**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)

**NCT ID:** [NCT05812781](https://clinicaltrials.gov/study/NCT05812781)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE2
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 7
- **Lead Sponsor:** Zomagen Biosciences Ltd.
- **Conditions:** Cryopyrin Associated Periodic Syndrome
- **Start Date:** 2023-03-18
- **Completion Date:** 2024-03-06
- **CT.gov Last Update:** 2025-03-10

## 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.

## Eligibility

- **Minimum age:** 18 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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
```

## Arms

- **Cohort 1** (EXPERIMENTAL)
- **Cohort 2** (EXPERIMENTAL)

## Interventions

- **VTX2735** (DRUG) — Dose A
- **VTX2735** (DRUG) — Dose B

## Primary Outcomes

- **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

- **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)_
- **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)_
- **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 days when any single DHAF2 symptom score is >3** _(time frame: From Day 1 to completion of treatment with VTX2735, up to Day 28)_
- **Maximum severity of any symptom score on DHAF2** _(time frame: From Day 1 to completion of treatment with VTX2735, up to Day 28)_

## Locations (2)

- Local Site # 222, San Diego, California, United States
- Local Site # 223, Columbus, Georgia, United States

## Recent Field Changes (last 30 days)

- `locations.local site # 222|san diego|california|united states` — added _(2026-05-12)_
- `locations.local site # 223|columbus|georgia|united states` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT05812781.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT05812781*  
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