A Study of the Safety and Tolerability of INCB050465 in Pemphigus Vulgaris

Study Overview
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Brief Summary
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The purpose of this study is to assess the safety and tolerability of parsaclisib in participants with mild to moderate pemphigus vulgaris.

Official Title
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A Phase 2 Dose-Escalation Study of the Safety and Tolerability of INCB050465 in Participants With Pemphigus Vulgaris

Conditions
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Pemphigus Vulgaris

Intervention / Treatment
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* Drug: Parsaclisib


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Parsaclisib<br> | Drug: Parsaclisib<br>* Parsaclisib administered orally once daily at the cohort-specified dose level.<br>* Other names: INCB050465;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of treatment-emergent adverse events | Any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug. | Up to 20 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cmax of Parsaclisib | Maximum observed concentration. | Up to 6 weeks | 
| tmax of Parsaclisib | Time to maximum concentration. | Up to 6 weeks | 
| Cmin of Parsaclisib | Minimum observed concentration over the dose interval. | Up to 6 weeks | 
| AUC0-t of Parsaclisib | Area under the concentration-time curve from time = 0 to the last measurable concentration at time = t. | Up to 6 weeks | 
| CL/F of Parsaclisib | Apparent oral dose clearance. | Up to 6 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pemphigus vulgaris, phosphatidylinositol 3-kinase delta, immunoglobulin G autoantibodies