A Study of INCB050465 in Primary Sjögren's Syndrome

Phase 2CompletedNCT03627065

Incyte Corporation10 enrolled

Overview

The purpose of this study is to assess the impact of parsaclisib on the signs and symptoms of Sjögren's syndrome (SS).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Primary Sjögren's Syndrome

Interventions

ParsaclisibDRUG

Parsaclisib administered orally once daily at the protocol-defined dose.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Primary SS diagnosed according to the revised American-European Consensus Group (AECG) criteria. * Minimum score of 2 on the SGUS score for parotid and submandibular glands. * EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) score ≥ 5. * Positive serum titers of anti-SS-A and/or anti-SS-B antibodies. * Symptomatic oral dryness score of at least 5 on patient questionnaire. * Willingness to avoid pregnancy or fathering children based on protocol-defined criteria. Exclusion Criteria: * Diagnosis of secondary SS according to the revised AECG criteria (eg, the presence of a previously diagnosed or a present diagnosis of rheumatoid arthritis, systemic lupus erythematosus, systemic sclerosis, mixed connective tissue disease, polymyositis, dermatomyositis, immunoglobulin G4-related disorder, sarcoidosis, or any other defined autoimmune rheumatologic disorder). * Concurrent conditions and history of other diseases per protocol-defined criteria. * Positive test results for tuberculosis (TB) from the QuantiFERON-TB Gold test or T-spot.TB test. * Positive serology test results for HIV antibody, hepatitis B surface antigen, hepatitis B surface antigen antibody, HBV core antibody, or HCV (HCV antibody with positive HCV-RNA). * Severely impaired liver function (Child-Pugh Class C). * Prior or ongoing therapy with protocol-defined drugs. * Receipt of any live vaccine in the 30 days before screening. * No major surgery within 30 days before screening. Inadequate recovery from toxicity and/or complications from a major surgery before starting therapy. * Current alcohol or drug use that, in the opinion of the investigator, will interfere with the participant's ability to comply with the dose regimen and study evaluations. * Laboratory values at screening outside the protocol-defined ranges.

Locations (4)

Advanced Pharma CR

Miami, Florida, United States

North Georgia Rheumatology Group

Lawrenceville, Georgia, United States

The Center for Rheumatology and Bone Research

Wheaton, Maryland, United States

Ramesh C. Gupta, MD

Memphis, Tennessee, United States

Outcomes

Primary Outcomes

Proportion of Participants With a 1 Point or Greater Change on the Salivary Gland Ultrasound (SGUS) Score for Parotid and Submandibular Glands

The SGUS is a combination of the scores of the 4 salivary glands (2 parotid and 2 submandibular) each gland is scored on a 5-point scale (0 to 4; 4 being more severe), with the maximum total score being 16.

Time frame: Week 4 and Week 12

Secondary Outcomes

Change From Baseline in Salivary CXCL13 Levels

To assess the impact of parsaclisib on salivary CXCL13.

Time frame: Baseline, Week 4 and Week 12

Number of Participants With Treatment-emergent Adverse Events

Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug.

Time frame: Up to 21 weeks

Change in Whole Salivary Flow

Defined as change in stimulated and unstimulated whole salivary flow from baseline

Time frame: Baseline, Weeks 4, 8, and 12

Change in EULAR Sjögren's Syndrome Disease Activity Index

Defined as change in ESSDAI. The ESSDAI is a physician-assessed disease activity index for primary Sjögren's syndrome developed by the EULAR consortium. It consists of 44 items in 12 organ-specific 'domains' contributing to disease activity (constitutional, lymphadenopathy, articular, muscular, cutaneous, glandular, pulmonary, renal, peripheral nervous system, central nervous system, hematological, biological). Each domain is assessed for activity level (i.e., no, low, moderate, high) and assigned a numerical score based on pre-determined weighting of each individual domain. An overall score is then calculated as the sum of all individual weighted domain scores. Overall score is calculated as sum of all individual weighted domain scores (ranges from 0 (best) to 123 (worst activity).

Data from ClinicalTrials.gov

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Change in EULAR Sjögren's Syndrome Patient Reported Index

Defined as Change in ESSPRI. EULAR Sjogren's Syndrome Patient-Reported Index (ESSPRI) Score is defined as the change in score between baseline (Week -1) and Week 12. The ESSPRI is a patient-reported, subjective symptom index for primary Sjögren's syndrome developed by the EULAR consortium. It consists of three questions covering the cardinal symptoms of Sjögren's syndrome: dryness, fatigue and pain (articular and/or muscular). Each domain scored on scale of 0-10 (0 =no symptom at all and 10 = worst symptom imaginable), and an overall score is calculated as the mean of the three individual domains where all domains carry the same weight.

Time frame: Weeks 4, 8, and 12

Change in Symptom Scores for Dryness

Defined as change in symptom scores for dryness of eyes, mouth, and vagina. The dryness questionnaire will ask participants to rate the dryness of eyes, mouth, or vagina (female participants only) with 24-hour recall using an 11-point numerical rating system ranging from 0 (no dryness) to 10

Time frame: Weeks 4, 8, and 12.

Patient Global Impression of Change Questionnaire

Defined as proportions of participants in each PGIC category. The PGIC asks a single question regarding how the patient is feeling since beginning new therapy. The questionnaire uses a 7-point scale (from 1 to 7), ranging from "very much improved, 1" to "very much worse, 7

Time frame: Baseline, Weeks 4, 8, and 12

Change in PROMIS Fatigue Short Form

Defined as change in PROMIS Fatigue short form. The PROMIS fatigue short form includes 7 items with a rating scale of 1 to 5, 1 being no fatigue and 5 being severe fatigue. The recall period is 7 days.

Time frame: Weeks 4, 8, and 12

Female Participants Only : Change in Female Sexual Function Index

Defined as change in FSFI. The FSFI is a brief, self-report measure of female sexual function (female participants only). The questionnaire contains 19 item sexual functioning questionnaire to rate sexual function between 2.0 and 36.0, where 2.0 is low sexual function and 36.0 is high sexual function. Difference in FSFI scores are reported.

Time frame: Weeks 4, 8, and 12

Change in European Quality of Life 5 Dimensions Questionnaire

Defined as change in EQ-5D. The EQ-5D is a standardized measure of health status. It consists of 5 questions, each with a 5-item rating scale plus a visual analog scale rating from 1 to 100 for overall health status. Higher score represents more severe symptoms.

Time frame: Weeks 4, 8, and 12

Percentage Change in Whole Salivary Flow

Defined as change and percent change in stimulated and unstimulated whole salivary flow from baseline

Time frame: Baseline, Weeks 4, 8, and 12

Percentage Change in EULAR Sjögren's Syndrome Disease Activity Index

Defined as percent change in ESSDAI. The ESSDAI is a physician-assessed disease activity index for primary Sjögren's syndrome developed by the EULAR consortium. It consists of 44 items in 12 organ-specific 'domains' contributing to disease activity (constitutional, lymphadenopathy, articular, muscular, cutaneous, glandular, pulmonary, renal, peripheral nervous system, central nervous system, hematological, biological). Each domain is assessed for activity level (i.e., no, low, moderate, high) and assigned a numerical score based on pre-determined weighting of each individual domain. An overall score is then calculated as the sum of all individual weighted domain scores. Overall score is calculated as sum of all individual weighted domain scores (ranges from 0 (best) to 123 (worst activity).

Time frame: Week 12

Percentage Change in EULAR Sjögren's Syndrome Patient Reported Index

Defined as percentage change in ESSPRI. EULAR Sjogren's Syndrome Patient-Reported Index (ESSPRI) Score is defined as the change in score between baseline (Week -1) and Week 12. The ESSPRI is a patient-reported, subjective symptom index for primary Sjögren's syndrome developed by the EULAR consortium. It consists of three questions covering the cardinal symptoms of Sjögren's syndrome: dryness, fatigue and pain (articular and/or muscular). Each domain scored on scale of 0-10 (0 =no symptom at all and 10 = worst symptom imaginable), and an overall score is calculated as the mean of the three individual domains where all domains carry the same weight.

Time frame: Weeks 4, 8, and 12

Percentage Change in Symptom Scores for Dryness

Defined as percent change in symptom scores for dryness of eyes, mouth, and vagina. The dryness questionnaire will ask participants to rate the dryness of eyes, mouth, or vagina (female participants only) with 24-hour recall using an 11-point numerical rating system ranging from 0 (no dryness) to 10

Time frame: Weeks 4, 8, and 12.

Percentage Change in PROMIS Fatigue Short Form

Defined as percent change in PROMIS Fatigue short form. The PROMIS fatigue short form includes 7 items with a rating scale of 1 to 5, 1 being no fatigue and 5 being severe fatigue. The recall period is 7 days.

Time frame: Weeks 4, 8, and 12

Female Participants Only : Percentage Change in Female Sexual Function Index

Defined as percent change in FSFI. The FSFI is a brief, self-report measure of female sexual function (female participants only). The questionnaire contains 19 item sexual functioning questionnaire to rate sexual function between 2.0 and 36.0, where 2.0 is low sexual function and 36.0 is high sexual function. Difference in FSFI scores are reported.

Time frame: Weeks 4, 8, and 12

Percentage Change in European Quality of Life 5 Dimensions Questionnaire

Defined as percent change in EQ-5D. The EQ-5D is a standardized measure of health status. It consists of 5 questions, each with a 5-item rating scale plus a visual analog scale rating from 1 to 100 for overall health status. Higher score represents more severe symptoms.

Time frame: Weeks 4, 8, and 12