Pilot Trial of Ustekinumab for Primary Sjögren's Syndrome

Phase 1

Completed

• Conditions: Primary Sjögren Syndrome
• Interventions: Drug: Ustekinumab

• Registration Number: NCT04093531
• Lead Sponsor: University of Rochester

Brief Summary

This pilot study will make a preliminary determination of the safety of ustekinumab in patients with Primary Sjogren's Syndrome (PSS) and assess the response of systemic measures of inflammation (biomarkers).

Detailed Description

This is a single-center, open label, pilot trial of ustekinumab in patients with Primary Sjögren's Syndrome (PSS). 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.

Study Timeline

• Study First Submitted: 2019-08-05
• Study First Submitted QC: 2019-09-16
• Study First Posted: 2019-09-18
• Study Start: 2020-01-15
• Primary Completion: 2022-05-11
• Study Completion: 2022-05-11
• Results First Submitted: 2023-12-06
• Status Verified: 2024-06
• Results First Submitted QC: 2024-06-20
• Last Update Submitted: 2024-06-20
• Results First Posted: 2024-06-25
• Last Update Posted: 2024-06-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ustekinumab	Ustekinumab	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.

Primary Outcome Measures

Name	Time	Method
Change in The European League Against Rheumatism (EULAR) Sjogren's Syndrome Patient Reported Index (ESSPRI) Score From Baseline to Week 24	baseline to 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 which ranges from 0- 10. Baseline ESSPRI total score will be compared to week 24, end of treatment, ESSPRI total score.

Secondary Outcome Measures

Name	Time	Method
Change in the Short Form Health Survey (SF-36) Patient Reported Outcome Measure Between Day 0 and Week 24	baseline to 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 for physical function, energy/fatigue, pain and general health.
Change in Mean Serum Biomarkers of Inflammation From Baseline to Week 24	baseline to 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 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.
Total Score of the EULAR Sjogren's Syndrome Disease Activity Index (ESSDAI)	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. Lower numbers indicate less activity. The total score is the sum of the score of all domains. The total score ranges from 0-123. Mean scores at baseline and week 24 are reported.

Trial Locations

Locations (1)

University of Rochester; 🇺🇸 Rochester, New York, United States