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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03780166
Recruitment Status : Not yet recruiting
First Posted : December 19, 2018
Last Update Posted : December 19, 2018
Information provided by (Responsible Party):
Incyte Corporation

Brief Summary:
The purpose of this study is to assess the safety and tolerability of INCB050465 in participants with mild to moderate pemphigus vulgaris.

Condition or disease Intervention/treatment Phase
Pemphigus Vulgaris Drug: INCB050465 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Dose-Escalation Study of the Safety and Tolerability of INCB050465 in Participants With Pemphigus Vulgaris
Estimated Study Start Date : February 2019
Estimated Primary Completion Date : August 2020
Estimated Study Completion Date : October 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Pemphigus

Arm Intervention/treatment
Experimental: INCB050465 Drug: INCB050465
INCB050465 administered orally once daily at the cohort-specified dose level.

Primary Outcome Measures :
  1. Number of treatment-emergent adverse events [ Time Frame: 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 Outcome Measures :
  1. Cmax of INCB050465 [ Time Frame: Up to 6 weeks ]
    Maximum observed concentration.

  2. tmax of INCB050465 [ Time Frame: Up to 6 weeks ]
    Time to maximum concentration.

  3. Cmin of INCB050465 [ Time Frame: Up to 6 weeks ]
    Minimum observed concentration over the dose interval.

  4. AUC0-t of INCB050465 [ Time Frame: Up to 6 weeks ]
    Area under the concentration-time curve from time = 0 to the last measurable concentration at time = t.

  5. CL/F of INCB050465 [ Time Frame: Up to 6 weeks ]
    Apparent oral dose clearance.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts 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 INCB050465 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03780166

Contact: Incyte Corporation Call Center (US) 1.855.463.3463
Contact: Incyte Corporation Call Center (ex-US) +800 00027423

Sponsors and Collaborators
Incyte Corporation
Study Director: Kathleen Butler, MD Incyte Corporation

Responsible Party: Incyte Corporation Identifier: NCT03780166     History of Changes
Other Study ID Numbers: INCB 50465-208
First Posted: December 19, 2018    Key Record Dates
Last Update Posted: December 19, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

Keywords provided by Incyte Corporation:
Pemphigus vulgaris
phosphatidylinositol 3-kinase delta
immunoglobulin G autoantibodies

Additional relevant MeSH terms:
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases