Cryopyrin Associated Periodic SyndromePhase 2ExploreNCT05812781
A Study to Evaluate VTX2735 in Patients With Cryopyrin-associated Periodic Syndrome
A Phase 2 study testing VTX2735 in people with Cryopyrin Associated Periodic Syndrome, run by Zomagen Biosciences Ltd. at 2 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cryopyrin Associated Periodic Syndrome
- What is being tested?
- VTX2735
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 2 sitesUnited States
- Study dates
- 18 Mar 2023to 6 Mar 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Local Site # 223
Columbus, Georgia, 31904
Local Site # 222
San Diego, California, 92123
About this study
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)
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.
- Conditions
- Cryopyrin Associated Periodic Syndrome
- Being tested
- VTX2735
- Keywords
- Cryopyrin-Associated Periodic Syndrome, Ventyx, Zomagen, VTX2735, CAPS
- Medical subject headings
- Cryopyrin-Associated Periodic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VTX2735Drug
Dose A
VTX2735Drug
Dose B
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Not described Receives: Drug: VTX2735 |
| Cohort 2 | Experimental | Not described Receives: Drug: VTX2735 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability of VTX2735
Incidence and severity of treatment-emergent adverse events (TEAE), serious adverse events (SAE), and discontinuation due to adverse events
Measured: From the initial administration of VTX2735 through study completion, up to 10 weeks
Secondary outcomes (5)
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.
Measured: 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
Proportion of patients achieving 30%, 50%, or 75% improvement from baseline in disease activity using DHAF2 and KSS.
Measured: From Day 1 to completion of treatment with VTX2735, up to Day 28
Number of days when the daily KSS is >3
Number of multi-system disease flare days as defined by KSS
Measured: From Day 1 to completion of treatment with VTX2735, up to Day 28
Number of days when any single DHAF2 symptom score is >3
Number of single system disease flare days as defined by KSS
Measured: From Day 1 to completion of treatment with VTX2735, up to Day 28
Maximum severity of any symptom score on DHAF2
Maximum single DHAF2 symptom score
Measured: From Day 1 to completion of treatment with VTX2735, up to Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2023
- Study start18 Mar 2023
- Primary completion6 Mar 2024
- Study completion6 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cryopyrin Associated Periodic SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zomagen Biosciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Henrik Sonnergren, MD, PhD, Study director, Ventyx Biosciences, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05812781
- Sponsor's protocol ID
- VTX2735-201
- Acronym
- Explore
- Status last verified
- Mar 2025
- First submitted
- 28 Mar 2023
- Last updated
- 10 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05812781
- Acronym / sponsor ID
- Explore / VTX2735-201
- Conditions
- Cryopyrin Associated Periodic Syndrome
- Keywords
- Cryopyrin-Associated Periodic Syndrome, Ventyx, Zomagen, VTX2735, CAPS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Zomagen Biosciences Ltd.Industry (pharma, biotech, devices)
- Study start
- 18 Mar 2023 (actual)
- Primary completion
- 6 Mar 2024 (actual)
- Study completion
- 6 Mar 2024 (actual)
- First posted
- 14 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: VTX2735 |
| Cohort 2 | Experimental | Drug: VTX2735 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (6 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VTX2735Dose A | Drug | Cohort 1 |
| VTX2735Dose B | Drug | Cohort 2 |
Primary outcome
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
5 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2025.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Henrik Sonnergren, MD, PhD study director
Ventyx Biosciences, Inc
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VTX2735 in people with Cryopyrin Associated Periodic Syndrome, run by Zomagen Biosciences Ltd. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability of VTX2735 (time frame: From the initial administration of VTX2735 through study completion, up to 10 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2025.