A Study of INCB050465 in Primary Sjögren's Syndrome

• Sponsor: Incyte Corporation (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03627065
• Collaborator: (none)
• Enrollment: 10
• Locations: 4
• Arm: 1
• Actual Duration (Months): 10.1
• Patients Per Site: 2.5
• Patients Per Site Per Month: 0.2

Study Details

Study Description

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

Study Design

• Study Type: Interventional
• 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: Feb 28, 2019
• Actual Primary Completion Date: Dec 7, 2019
• Actual Study Completion Date: Jan 2, 2020

Arms and Interventions

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

Outcome Measures

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 [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 [Baseline, Week 4 and Week 12]

   To assess the impact of parsaclisib on salivary CXCL13.

2. Number of Participants With Treatment-emergent Adverse Events [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Advanced Pharma CR	Miami	Florida	United States	33147
2	North Georgia Rheumatology Group	Lawrenceville	Georgia	United States	30046
3	The Center for Rheumatology and Bone Research	Wheaton	Maryland	United States	20902
4	Ramesh C. Gupta, MD	Memphis	Tennessee	United States	38119-5208

Sponsors and Collaborators

• Incyte Corporation

Investigators

• Study Director: Kathleen Butler, MD, Incyte Corporation

Study Documents (Full-Text)

• Study Protocol - Jun 12, 2018
• Statistical Analysis Plan - Feb 25, 2019

More Information

Publications

None provided.

• Responsible Party: Incyte Corporation
• ClinicalTrials.gov Identifier: NCT03627065
• Other Study ID Numbers: INCB 50465-207; Parsaclisib
• First Posted: Aug 13, 2018
• Last Update Posted: Aug 13, 2021
• Last Verified: Aug 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• 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: Sjogren's Syndrome; Syndrome

Study Results

Participant Flow

Recruitment Details	Approximately 12 participants were planned. Ten participants were enrolled and analyzed for efficacy and safety.
Pre-assignment Detail	Ten participants were enrolled and treated. Seven participants completed treatment. Three participants discontinued treatment and withdrew from the study.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day.
Period Title: Overall Study
STARTED	10
COMPLETED	7
NOT COMPLETED	3

Baseline Characteristics

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Overall Participants	10
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	54.1 (13.47)
Sex: Female, Male (Count of Participants)
Female	9 90%
Male	1 10%
Race/Ethnicity, Customized (Count of Participants)
Hispanic or Latino	1 10%
Not Hispanic or Latino	9 90%
Race/Ethnicity, Customized (Count of Participants)
White/Caucasian	4 40%
Black/African-American	6 60%

Outcome Measures

1. Primary Outcome

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

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Week 4	0 0%
Week 12	0 0%

2. Secondary Outcome

Title	Change From Baseline in Salivary CXCL13 Levels
Description	To assess the impact of parsaclisib on salivary CXCL13.
Time Frame	Baseline, Week 4 and Week 12

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug, and detectable levels of CXCL13.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	2
Week 4	0.96 (0.1)
Week 12	1.98 (1.4)

3. Secondary Outcome

Title	Number of Participants With Treatment-emergent Adverse Events
Description	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

Outcome Measure Data

Analysis Population Description

The full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Count of Participants [Participants]	4 40%

4. Secondary Outcome

Title	Change in Whole Salivary Flow
Description	Defined as change in stimulated and unstimulated whole salivary flow from baseline
Time Frame	Baseline, Weeks 4, 8, and 12

Outcome Measure Data

Analysis Population Description

The full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Mean change in stimulated salivary flow Week 4	0.07 (0.152)
Mean change in stimulated salivary flow Week 8	-0.05 (0.129)
Mean change in stimulated salivary flow Week 12	-0.05 (0.144)
Mean change in unstimulated salivary flow Week 4	0.03 (0.046)
Mean change in unstimulated salivary flow Week 8	0.07 (0.109)
Mean change in unstimulated salivary flow Week 12	0.04 (0.082)

5. Secondary Outcome

Title	Change in EULAR Sjögren's Syndrome Disease Activity Index
Description	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).
Time Frame	Week 12

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Mean Change at Week 4	-0.3 (2.92)
Mean Change at Week 8	-2.0 (4.12)
Mean Change at Week 12	-5.3 (4.15)

6. Secondary Outcome

Title	Change in EULAR Sjögren's Syndrome Patient Reported Index
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	9
Mean Change at Week 4	-1.9 (2.26)
Mean Change at Week 8	-2.0 (1.93)
Mean Change at Week 12	-1.8 (1.98)

7. Secondary Outcome

Title	Change in Symptom Scores for Dryness
Description	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.

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Change in Symptom Score for eye dryness at Week 4	-2.6 (2.96)
Change in Symptom Score for eye dryness at Week 8	-2.4 (2.07)
Change in Symptom Score for eye dryness at Week 12	-2.4 (2.64)
Change in Symptom Score for mouth dryness at Week 4	-2.8 (2.64)
Change in Symptom Score for mouth dryness at Week 8	-2.1 (2.41)
Change in Symptom Score for mouth dryness at Week 12	-3.6 (2.99)
Change in Symptom Score for vaginal dryness at Week 4	-1.3 (3.20)
Change in Symptom Score for vaginal dryness at Week 8	-2.3 (3.50)
Change in Symptom Score for vaginal dryness at Week 12	-1.3 (2.94)

8. Secondary Outcome

Title	Patient Global Impression of Change Questionnaire
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Very Much Worse	0 0%
Worse	0 0%
Somewhat Worse	0 0%
No Change	9 90%
Somewhat Improved	0 0%
Improved	0 0%
Very much Improved	0 0%
Very Much Worse	0 0%
Worse	0 0%
Somewhat Worse	0 0%
No Change	3 30%
Somewhat Improved	2 20%
Improved	4 40%
Very much Improved	0 0%
Very Much Worse	0 0%
Worse	0 0%
Somewhat Worse	0 0%
No Change	2 20%
Somewhat Improved	2 20%
Improved	3 30%
Very much Improved	0 0%
Very Much Worse	0 0%
Worse	0 0%
Somewhat Worse	0 0%
No Change	2 20%
Somewhat Improved	2 20%
Improved	3 30%
Very much Improved	0 0%

9. Secondary Outcome

Title	Change in PROMIS Fatigue Short Form
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Change in Score at Week 4	-2.6 (4.03)
Change in Score at Week 8	-1.9 (2.79)
Change in Score at Week 12	-1.7 (2.87)

10. Secondary Outcome

Title	Female Participants Only : Change in Female Sexual Function Index
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all female participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	9
Change in Functional Index at Week 4	1.8 (3.19)
Change in Functional Index at Week 8	2.8 (4.76)
Change in Functional Index at Week 12	1.3 (1.83)

11. Secondary Outcome

Title	Change in European Quality of Life 5 Dimensions Questionnaire
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Change in EQ5D at week 4	7.2 (17.34)
Change in EQ5D at week 8	0.7 (28.05)
Change in EQ5D at week 12	-6.4 (22.49)

12. Secondary Outcome

Title	Percentage Change in Whole Salivary Flow
Description	Defined as change and percent change in stimulated and unstimulated whole salivary flow from baseline
Time Frame	Baseline, Weeks 4, 8, and 12

Outcome Measure Data

Analysis Population Description

The full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Percent change in stimulated salivary flow Week 4	17.57 (36.225)
Percent change in stimulated salivary flow Week 8	-10.59 (32.874)
Percent change in stimulated salivary flow Week 12	-16.07 (41.754)
Percent change in unstimulated salivary flow Week 4	228.10 (282.634)
Percent change in unstimulated salivary flow Week 8	307.56 (601.557)
Percent change in unstimulated salivary flow Week 12	281.12 (541.333)

13. Secondary Outcome

Title	Percentage Change in EULAR Sjögren's Syndrome Disease Activity Index
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Percent Change at Week 4	1.8 (28.13)
Percent Change at Week 8	-5.2 (24.09)
Percent Change at Week 12	-32.1 (25.70)

14. Secondary Outcome

Title	Percentage Change in EULAR Sjögren's Syndrome Patient Reported Index
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Percent Change at Week 4	-24.4 (40.36)
Percent Change at Week 8	-29.0 (28.27)
Percent Change at Week 12	-25.8 (30.52)

15. Secondary Outcome

Title	Percentage Change in Symptom Scores for Dryness
Description	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.

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Percent change in Symptom Score for eye dryness at Week 4	-33.2 (35.29)
Percent change in Symptom Score for eye dryness at Week 8	-39.3 (23.89)
Percent change in Symptom Score for eye dryness at Week 12	-40.9 (39.09)
Percent change in Symptom Score for mouth dryness at Week 4	-37.7 (37.56)
Percent change in Symptom Score for mouth dryness at Week 8	-28.0 (32.97)
Percent change in Symptom Score for mouth dryness at Week 12	-50.9 (45.88)
Percent change in Symptom Score for vaginal dryness at Week 4	-24.0 (49.18)
Percent change in Symptom Score for vaginal dryness at Week 8	-54.2 (51.59)
Percent change in Symptom Score for vaginal dryness at Week 12	-49.0 (42.54)

16. Secondary Outcome

Title	Percentage Change in PROMIS Fatigue Short Form
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Percent change in Score at Week 4	-11.1 (21.87)
Percent change in Score at Week 8	-7.5 (13.17)
Percent change in Score at Week 12	-6.4 (13.16)

17. Secondary Outcome

Title	Female Participants Only : Percentage Change in Female Sexual Function Index
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all female participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	9
Percent change in Functional Index at Week 4	39.0 (94.61)
Percent change in Functional Index at Week 8	8.7 (31.63)
Percent change in Functional Index at Week 12	23.3 (30.08)

18. Secondary Outcome

Title	Percentage Change in European Quality of Life 5 Dimensions Questionnaire
Description	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

Outcome Measure Data

Analysis Population Description

Full analysis set included all participants enrolled in the study who received at least 1 dose of study drug.

Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day for up to 12 weeks.
Measure Participants	10
Percent Change in EQ5D at week 4	21.1 (55.37)
Percent Change in EQ5D at week 8	11.9 (62.91)
Percent Change in EQ5D at week 12	-8.9 (49.54)

Adverse Events

Time Frame	Up to 21 weeks
Arm/Group Title	Parsaclisib
Arm/Group Description	parsaclisib was administered orally once a day.
All Cause Mortality
	Parsaclisib
	Affected / at Risk (%)	# Events
Total	0/10 (0%)
Serious Adverse Events
	Parsaclisib
	Affected / at Risk (%)	# Events
Total	0/10 (0%)
Other (Not Including Serious) Adverse Events
	Parsaclisib
	Affected / at Risk (%)	# Events
Total	4/10 (40%)
Gastrointestinal disorders
Dental caries	1/10 (10%)	1
Gastroesophageal reflux disease	1/10 (10%)	1
Nausea	1/10 (10%)	1
Toothache	1/10 (10%)	1
Infections and infestations
Oral candidiasis	1/10 (10%)	2
Injury, poisoning and procedural complications
Fall	1/10 (10%)	1
Musculoskeletal and connective tissue disorders
Arthralgia	1/10 (10%)	1
Musculoskeletal chest pain	1/10 (10%)	1
Skin and subcutaneous tissue disorders
Neurodermatitis	1/10 (10%)	1
Pruritus	1/10 (10%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

Clinical Study Agreement