Pemphigus Vulgaris
Conditions
Keywords
Pemphigus vulgaris, phosphatidylinositol 3-kinase delta, immunoglobulin G autoantibodies
Brief summary
The purpose of this study is to assess the safety and tolerability of parsaclisib in participants with mild to moderate pemphigus vulgaris.
Interventions
DRUGParsaclisib
Parsaclisib administered orally once daily at the cohort-specified dose level.
Sponsors
Incyte Corporation
Study design
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Clinically documented and confirmed diagnosis of pemphigus vulgaris: minimum of 6 months of pemphigus vulgaris diagnosis; positive for anti-desmoglein (DSG)1 or DSG3; Pemphigus Disease Area Index score of 8 to 45 points; active skin, scalp, or mucosal lesions. * Disease progression after treatment with standard therapies that are known to confer clinical benefit, or intolerant to treatment; there is no limit to the number of prior treatment regimens. * Willingness to avoid pregnancy or fathering children. * If required, willing to receive Pneumocystis jirovecii pneumonia prophylaxis during the study period.
Exclusion criteria
* Pregnant or breast-feeding female. * Participants with pemphigus vulgaris who are treatment-naive. * Use of protocol-specified medications within defined periods before baseline. * Evidence or history of clinically significant infection or protocol-defined medical conditions * Laboratory values outside the protocol-defined range at screening. * Known or suspected allergy to parsaclisib or any component of the study drug. * Known history of clinically significant drug or alcohol abuse in the last year before baseline. * Inability or unlikeliness of the participant to comply with the dose schedule and study evaluations, in the opinion of the investigator. * Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of treatment-emergent adverse events | Up to 20 weeks | Any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cmax of Parsaclisib | Up to 6 weeks | Maximum observed concentration. |
| tmax of Parsaclisib | Up to 6 weeks | Time to maximum concentration. |
| Cmin of Parsaclisib | Up to 6 weeks | Minimum observed concentration over the dose interval. |
| AUC0-t of Parsaclisib | Up to 6 weeks | Area under the concentration-time curve from time = 0 to the last measurable concentration at time = t. |
| CL/F of Parsaclisib | Up to 6 weeks | Apparent oral dose clearance. |
Outcome results
None listed