Cryopyrin Associated Periodic Syndrome
Conditions
Keywords
Cryopyrin-Associated Periodic Syndrome, Ventyx, Zomagen, VTX2735, CAPS
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.
Interventions
DRUGVTX2735
Dose A
Sponsors
Zomagen Biosciences Ltd.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and Tolerability of VTX2735 | 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
| Measure | Time frame | Description |
|---|---|---|
| Achievement of 30%, 50%, or 75% improvement in mean KSS from baseline | 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. |
| Mean change in the mean key symptom score (KSS) derived from the Daily Health Assessment Form, Second Generation (DHAF2) from baseline | 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. |
| Number of days when the daily KSS is >3 | From Day 1 to completion of treatment with VTX2735, up to Day 28 | Number of multi-system disease flare days as defined by KSS |
| Number of days when any single DHAF2 symptom score is >3 | From Day 1 to completion of treatment with VTX2735, up to Day 28 | Number of single system disease flare days as defined by KSS |
| Maximum severity of any symptom score on DHAF2 | From Day 1 to completion of treatment with VTX2735, up to Day 28 | Maximum single DHAF2 symptom score |
Countries
United States
Outcome results
None listed