Clinical trial NCT05812781: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cryopyrin Associated Periodic Syndrome
Keywords: Cryopyrin-Associated Periodic Syndrome
Ventyx
Zomagen
VTX2735
CAPS
Lead sponsor: Zomagen Biosciences Ltd.
Sponsor class: INDUSTRY
First posted: 2023-04-14
Enrollment: 7
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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 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

Primary outcomes:
measure: Safety and Tolerability of VTX2735
timeFrame: From the initial administration of VTX2735 through study completion, up to 10 weeks
Secondary outcomes:
measure: Mean change in the mean key symptom score (KSS) derived from the Daily Health Assessment Form, Second Generation (DHAF2) from baseline
timeFrame: From Day 1 to completion of treatment with VTX2735, up to Day 28
measure: Achievement of 30%, 50%, or 75% improvement in mean KSS from baseline
timeFrame: From Day 1 to completion of treatment with VTX2735, up to Day 28
measure: Number of days when the daily KSS is >3
timeFrame: From Day 1 to completion of treatment with VTX2735, up to Day 28
measure: Number of days when any single DHAF2 symptom score is >3
timeFrame: From Day 1 to completion of treatment with VTX2735, up to Day 28
measure: Maximum severity of any symptom score on DHAF2
timeFrame: From Day 1 to completion of treatment with VTX2735, up to Day 28
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Local Site # 222
state: California
city: Columbus
country: United States
facility: Local Site # 223
state: Georgia
Study officials:
affiliation: Ventyx Biosciences, Inc
name: Henrik Sonnergren, MD, PhD
role: STUDY_DIRECTOR
References: