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A Study of INCB050465 in Primary Sjögren's Syndrome

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ClinicalTrials.gov Identifier: NCT03627065
Recruitment Status : Completed
First Posted : August 13, 2018
Results First Posted : March 24, 2021
Last Update Posted : March 24, 2021
Sponsor:
Information provided by (Responsible Party):
Incyte Corporation

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

Condition or disease Intervention/treatment Phase
Primary Sjögren's Syndrome Drug: Parsaclisib Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Phase 2 Study of INCB050465 in Participants With Primary Sjögren's Syndrome
Actual Study Start Date : February 28, 2019
Actual Primary Completion Date : December 7, 2019
Actual Study Completion Date : January 2, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Parsaclisib Drug: Parsaclisib
Parsaclisib administered orally once daily at the protocol-defined dose.
Other Name: INCB050465




Primary Outcome Measures :
  1. Proportion of Participants With a 1 Point or Greater Change on the Salivary Gland Ultrasound (SGUS) Score for Parotid and Submandibular Glands [ Time Frame: Week 4 and Week 12 ]
    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.


Secondary Outcome Measures :
  1. Change From Baseline in Salivary CXCL13 Levels [ Time Frame: Baseline, Week 4 and Week 12 ]
    To assess the impact of parsaclisib on salivary CXCL13.

  2. Number of Participants With Treatment-emergent Adverse Events [ Time Frame: Up to 21 weeks ]
    Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug.

  3. Change in Whole Salivary Flow [ Time Frame: Baseline, Weeks 4, 8, and 12 ]
    Defined as change in stimulated and unstimulated whole salivary flow from baseline

  4. Change in EULAR Sjögren's Syndrome Disease Activity Index [ Time Frame: Week 12 ]
    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).

  5. Change in EULAR Sjögren's Syndrome Patient Reported Index [ Time Frame: Weeks 4, 8, and 12 ]
    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.

  6. Change in Symptom Scores for Dryness [ Time Frame: Weeks 4, 8, and 12. ]
    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

  7. Patient Global Impression of Change Questionnaire [ Time Frame: Baseline, Weeks 4, 8, and 12 ]
    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

  8. Change in PROMIS Fatigue Short Form [ Time Frame: Weeks 4, 8, and 12 ]
    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.

  9. Female Participants Only : Change in Female Sexual Function Index [ Time Frame: Weeks 4, 8, and 12 ]
    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.

  10. Change in European Quality of Life 5 Dimensions Questionnaire [ Time Frame: Weeks 4, 8, and 12 ]
    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.

  11. Percentage Change in Whole Salivary Flow [ Time Frame: Baseline, Weeks 4, 8, and 12 ]
    Defined as change and percent change in stimulated and unstimulated whole salivary flow from baseline

  12. Percentage Change in EULAR Sjögren's Syndrome Disease Activity Index [ Time Frame: Week 12 ]
    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).

  13. Percentage Change in EULAR Sjögren's Syndrome Patient Reported Index [ Time Frame: Weeks 4, 8, and 12 ]
    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.

  14. Percentage Change in Symptom Scores for Dryness [ Time Frame: Weeks 4, 8, and 12. ]
    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

  15. Percentage Change in PROMIS Fatigue Short Form [ Time Frame: Weeks 4, 8, and 12 ]
    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.

  16. Female Participants Only : Percentage Change in Female Sexual Function Index [ Time Frame: Weeks 4, 8, and 12 ]
    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.

  17. Percentage Change in European Quality of Life 5 Dimensions Questionnaire [ Time Frame: Weeks 4, 8, and 12 ]
    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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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 ClinicalTrials.gov identifier (NCT number): NCT03627065


Locations
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United States, Florida
Advanced Pharma CR
Miami, Florida, United States, 33147
United States, Georgia
North Georgia Rheumatology Group
Lawrenceville, Georgia, United States, 30046
United States, Maryland
The Center for Rheumatology and Bone Research
Wheaton, Maryland, United States, 20902
United States, Tennessee
Ramesh C. Gupta, MD
Memphis, Tennessee, United States, 38119-5208
Sponsors and Collaborators
Incyte Corporation
Investigators
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Study Director: Kathleen Butler, MD Incyte Corporation
  Study Documents (Full-Text)

Documents provided by Incyte Corporation:
Statistical Analysis Plan  [PDF] February 25, 2019
Study Protocol  [PDF] June 12, 2018

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Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT03627065    
Other Study ID Numbers: INCB 50465-207
Parsaclisib ( Other Identifier: Incyte Corporation )
First Posted: August 13, 2018    Key Record Dates
Results First Posted: March 24, 2021
Last Update Posted: March 24, 2021
Last Verified: March 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Incyte Corporation:
Primary Sjögren's syndrome
PI3Kδ inhibitor
Additional relevant MeSH terms:
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Sjogren's Syndrome
Syndrome
Disease
Pathologic Processes
Arthritis, Rheumatoid
Arthritis
Joint Diseases
Musculoskeletal Diseases
Rheumatic Diseases
Xerostomia
Salivary Gland Diseases
Mouth Diseases
Stomatognathic Diseases
Dry Eye Syndromes
Lacrimal Apparatus Diseases
Eye Diseases
Connective Tissue Diseases
Autoimmune Diseases
Immune System Diseases