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

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

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

Study Overview

• Status: Withdrawn
• Conditions: Pemphigus Vulgaris
• Intervention / Treatment: Drug: Parsaclisib

• Study Type: Interventional
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Interventional Model: SEQUENTIAL
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Parsaclisib	Drug: Parsaclisib; Parsaclisib administered orally once daily at the cohort-specified dose level.; Other Names: INCB050465

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Incyte Corporation

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): March 1, 2019
• Primary Completion (ANTICIPATED): November 1, 2020
• Study Completion (ANTICIPATED): November 1, 2020

Study Registration Dates

• First Submitted: December 17, 2018
• First Submitted That Met QC Criteria: December 17, 2018
• First Posted (ACTUAL): December 19, 2018

Study Record Updates

• Last Update Posted (ACTUAL): September 13, 2019
• Last Update Submitted That Met QC Criteria: September 11, 2019
• Last Verified: September 1, 2019

More Information

Terms related to this study

• Keywords: Pemphigus vulgaris; phosphatidylinositol 3-kinase delta; immunoglobulin G autoantibodies
• Additional Relevant MeSH Terms: Skin Diseases; Immune System Diseases; Autoimmune Diseases; Skin Diseases, Vesiculobullous; Pemphigus
• Other Study ID Numbers: INCB 50465-208; Parsaclisib (Other Identifier: Incyte Corporation)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes