Pilot Trial of Ustekinumab for Primary Sjögren's Syndrome
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|ClinicalTrials.gov Identifier: NCT04093531|
Recruitment Status : Recruiting
First Posted : September 18, 2019
Last Update Posted : March 5, 2021
|Condition or disease||Intervention/treatment||Phase|
|Primary Sjögren Syndrome||Drug: Ustekinumab||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Up to 15 subjects will receive an infusion loading dose of 6 mg/kg of ustekinumab at baseline, and 90 mg of ustekinumab subcutaneously at week 4, week 12 and week 20. Subjects will be followed for 24 weeks.|
|Masking:||None (Open Label)|
|Official Title:||Pilot Trial of Ustekinumab for Primary Sjögren's Syndrome|
|Actual Study Start Date :||January 15, 2020|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||December 2021|
All subjects will receive an intravenous loading dose of 6 mg/kg at their baseline visit. 650mg acetaminophen and 60mg allegra will be given as premedication to the infusion. All patients will receive 90mg ustekinumab by a subcutaneous injection at all subsequent dosing visits. Subcutaneous injections do not require any premedication. Drug will be administered by qualified personnel.
Up to 15 subjects will receive an infusion loading dose of 6 mg/kg of ustekinumab at baseline, and 90 mg of ustekinumab subcutaneously at week 4, week 12 and week 20. Subjects will be followed for 24 weeks.
- Change in The European League Against Rheumatism (EULAR) Sjogren's Syndrome Patient Reported Index (ESSPRI) score from Baseline to Week 24 [ Time Frame: 24 weeks ]The primary endpoint is a well-established, patient reported questionnaire for use in PSS, the ESSPRI. The ESSPRI is composed of 3 scales - dryness, fatigue, and pain. Each scale is measured 0 - 10, 0 being no symptoms, 10 being maximal imaginable dryness, fatigue or pain. The total score is the mean score of the three scales. Baseline ESSPRI total score will be compared to week 24, end of treatment, ESSPRI total score.
- Change in the Short Form Health Survey (SF-36) Patient Reported Outcome Measure between day 0 and week 24 [ Time Frame: 24 weeks ]The SF-36 is a measure of health-related quality-of-life. It's a 36-item patient-reported questionnaire that covers 8 health domains. Scores for each domain range from 0 to 100, with a higher score defining a more favorable health state. Changes in total scores in each domain from BL to week 24 will be measured.
- Change in serum biomarkers of inflammation from baseline to week 24 [ Time Frame: 24 weeks ]To determine whether the standard dosing schedule for ustekinumab lowers serum biomarkers of inflammation in patients with PSS blood will be collected at baseline, week 12 and week 24 for the following mechanistic studies: Serum levels of TNFα, IL-6, IL17, IL17A, IL17F, IL22, IL12, IL23, BAFF, B and T cell, blood interferon signature. Means, medians, standard deviations, minimums and maximums will be computed for each continuous mechanistic endpoint.
- Number of participants with treatment-emergent adverse events as defined by ICH definition of AE. [ Time Frame: 24 weeks ]Safety of ustekinumab in PSS patients will be measured by incidence of serious and non-serious adverse events per ICH definitions from prior to treatment and after the first dose of study drug. Number of events and number (%) of participants experiencing events will be summarized by system organ class and preferred term for each. In addition, AEs will be summarized by maximum severity and relationship to study drug.
- Change in total score of the EULAR Sjogren's syndrome disease activity index (ESSDAI) from baseline to week 24. [ Time Frame: 24 weeks. ]The ESSDAI is a disease activity measurement that has 12 domains. The score of each domain is the product of the weight of the domain by the level of activity. Activity levels are 0 - 3, 0 being no activity, 3 being high activity. The total score is the sum of the score of all domains. Change in total score from BL to week 24 will be measured.
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): NCT04093531
|Contact: Dustina Holt, MPHfirstname.lastname@example.org|
|United States, New York|
|University of Rochester||Recruiting|
|Rochester, New York, United States, 14642|
|Contact: Alena Leger 585-275-1634|
|Principal Investigator:||Ummara Shah, MD||Assistant Professor of Medicine|