History of Changes for Study: NCT05812781

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

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Study Record Versions

Version	A	B	Submitted Date	Changes
1			April 11, 2023	None (earliest Version on record)
2			March 14, 2024	Recruitment Status, Study Status, Contacts/Locations and Study Design

Comparison Format:

Merged
Side-by-Side

Study Identification


Unique Protocol ID:	VTX2735-201
Brief Title:	A Study to Evaluate VTX2735 in Patients With Cryopyrin-associated Periodic Syndrome (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)
Secondary IDs:

Study Status


Record Verification:	March 2023
Overall Status:	Recruiting
Study Start:	March 18, 2023
Primary Completion:	October 2023 [Anticipated]
Study Completion:	December 2023 [Anticipated]

First Submitted:	March 28, 2023
First Submitted that Met QC Criteria:	April 11, 2023
First Posted:	April 14, 2023 [Actual]

Last Update Submitted that Met QC Criteria:	April 11, 2023
Last Update Posted:	April 14, 2023 [Actual]

Sponsor/Collaborators


Sponsor:	Zomagen Biosciences, Ltd
Responsible Party:	Sponsor
Collaborators:

Oversight


U.S. FDA-regulated Drug:	Yes
U.S. FDA-regulated Device:	No
Data Monitoring:	No

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.

Detailed Description:

Conditions


Conditions:	Cryopyrin Associated Periodic Syndrome
Keywords:	Cryopyrin-Associated Periodic Syndrome Ventyx Zomagen VTX2735 CAPS

Study Design


Study Type:	Interventional
Primary Purpose:	Treatment
Study Phase:	Phase 2
Interventional Study Model:	Sequential Assignment
Number of Arms:	2
Masking:	None (Open Label)
Allocation:	Non-Randomized
Enrollment:	10 [Anticipated]

Arms and Interventions

Arms	Assigned Interventions
Experimental: Cohort 1	Drug: VTX2735 Dose A
Experimental: Cohort 2	Drug: VTX2735 Dose B

Outcome Measures


Primary Outcome Measures:
1.	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 Outcome Measures:
1.	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 ] Assess the change from baseline in disease activity using DHAF2 and KSS.
2.	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 ] Proportion of patients achieving 30%, 50%, or 75% improvement from baseline in disease activity using DHAF2 and KSS.
3.	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 multi-system disease flare days as defined by KSS
4.	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 ] Number of single system disease flare days as defined by KSS
5.	Maximum severity of any symptom score on DHAF2 [ Time Frame: From Day 1 to completion of treatment with VTX2735, up to Day 28 ] Maximum single DHAF2 symptom score

Eligibility


Minimum Age:	18 Years
Maximum Age:
Sex:	All
Gender Based:
Accepts Healthy Volunteers:	No
Criteria:	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

Contacts/Locations


Study Officials:	Matt Cascino, MD Study Director Ventyx Biosciences, Inc
Locations:	United States, California
	Local Site # 222 [Recruiting] San Diego, California, United States, 92123 Contact:Contact: Local Site Contact 858-268-2368 research@allergyandasthma.com
	United States, Georgia
	Local Site # 223 [Recruiting] Columbus, Georgia, United States, 31904 Contact:Contact: Local Site Contact 205-209-4101 wodonnell@allervie.com

IPDSharing


Plan to Share IPD:	Undecided

References


Citations:
Links:
Available IPD/Information: