A Study of the Safety and Tolerability of INCB050465 in Pemphigus Vulgaris
Primary Purpose
Pemphigus Vulgaris
Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Parsaclisib
Sponsored by
About this trial
This is an interventional treatment trial for Pemphigus Vulgaris focused on measuring Pemphigus vulgaris, phosphatidylinositol 3-kinase delta, immunoglobulin G autoantibodies
Eligibility Criteria
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.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Parsaclisib
Arm Description
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Cmax of Parsaclisib
Maximum observed concentration.
tmax of Parsaclisib
Time to maximum concentration.
Cmin of Parsaclisib
Minimum observed concentration over the dose interval.
AUC0-t of Parsaclisib
Area under the concentration-time curve from time = 0 to the last measurable concentration at time = t.
CL/F of Parsaclisib
Apparent oral dose clearance.
Full Information
NCT ID
NCT03780166
First Posted
December 17, 2018
Last Updated
September 11, 2019
Sponsor
Incyte Corporation
1. Study Identification
Unique Protocol Identification Number
NCT03780166
Brief Title
A Study of the Safety and Tolerability of INCB050465 in Pemphigus Vulgaris
Official Title
A Phase 2 Dose-Escalation Study of the Safety and Tolerability of INCB050465 in Participants With Pemphigus Vulgaris
Study Type
Interventional
2. Study Status
Record Verification Date
September 2019
Overall Recruitment Status
Withdrawn
Why Stopped
The study was withdrawn due to insufficient interest in study participation due to recent approval in this rare condition.
Study Start Date
March 2019 (Anticipated)
Primary Completion Date
November 2020 (Anticipated)
Study Completion Date
November 2020 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Incyte Corporation
4. Oversight
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
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to assess the safety and tolerability of parsaclisib in participants with mild to moderate pemphigus vulgaris.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pemphigus Vulgaris
Keywords
Pemphigus vulgaris, phosphatidylinositol 3-kinase delta, immunoglobulin G autoantibodies
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Parsaclisib
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Parsaclisib
Other Intervention Name(s)
INCB050465
Intervention Description
Parsaclisib administered orally once daily at the cohort-specified dose level.
Primary Outcome Measure Information:
Title
Number of treatment-emergent adverse events
Description
Any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug.
Time Frame
Up to 20 weeks
Secondary Outcome Measure Information:
Title
Cmax of Parsaclisib
Description
Maximum observed concentration.
Time Frame
Up to 6 weeks
Title
tmax of Parsaclisib
Description
Time to maximum concentration.
Time Frame
Up to 6 weeks
Title
Cmin of Parsaclisib
Description
Minimum observed concentration over the dose interval.
Time Frame
Up to 6 weeks
Title
AUC0-t of Parsaclisib
Description
Area under the concentration-time curve from time = 0 to the last measurable concentration at time = t.
Time Frame
Up to 6 weeks
Title
CL/F of Parsaclisib
Description
Apparent oral dose clearance.
Time Frame
Up to 6 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kathleen Butler, MD
Organizational Affiliation
Incyte Corporation
Official's Role
Study Director
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study of the Safety and Tolerability of INCB050465 in Pemphigus Vulgaris
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