Clinical Study to Investigate the Efficacy of Orally Administered SA001 in Patients With Primary Sjögren's Syndrome

Study Overview
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Brief Summary
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This is a phase2a, multicenter, double-blind, placebo control, randomized study to investigate the efficacy and safety of SA001 in subjects with pSS. A total of 28 subjects (including dropout rate of 30%) will be randomized in a 1:1:1:1 ratio to receive 3 different doses of SA001 or placebo everyday for 8 weeks. Screening visit will be performed within 1 to 2 weeks(run-in period) prior to dosing after signing the informed consent form (ICF). During the run-in period, if necessary, subjects will apply artificial tears in the symptomatic eyes according to the dosage of artificial tears. Only subjects who have completed the run-in period and who are determined to be suitable for the study eligibility(inclusion/exclusion) criteria as a result of the screening evaluations are randomized to one of the four groups. Subjects will receive investigational product start on Day 0 for 8 weeks during the active treatment period. Subjects will visit to the study site on 4 and 8 weeks after starting dosing investigational product. Subjects will be in this study approximately 12weeks, which includes run-in period of 1 to 2weeks and a safety follow-up period of 2weeks.

Official Title
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Multicenter Double-blinded Randomized Phase 2a Clinical Trial to Evaluate the Efficacy of SA001 for Dry Eye and Mouth Dryness in the Patients With Primary Sjögren's Syndrome

Conditions
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Primary Sjögren's Syndrome (pSS)

Intervention / Treatment
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* Drug: SA001 Low dose
* Drug: SA001 Mid dose
* Drug: SA001 High dose
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age 19 through 80 years Patient who diagnosed with Primary Sjögren's Syndrome(has a score of ≥ 4 when the weights from 5 criteria items are summed) ① Labial salivary gland with focal lymphocytic sialadenitis and focus score of ≥ 1 foci/4 mm2 (weight: 3) ② Anti-SSA/Ro-positive (weight: 3) ③ Ocular Staining Score ≥ 5 (or van Bijsterveld score ≥ 4) in at least one eye (weight: 1) ④ Schirmer's test ≤ 5 mm/5 min in at least one eye (weight: 1) ⑤ Unstimulated whole saliva flow rate ≤ 0.1 mL/min (weight: 1) Standard Patient Evaluation of Eye Dryness(SPEED) score ≥ 5 Exclusion Criteria: Secondary Sjögren's Syndrome patient Severe blepharitis caused by Meibomian gland dysfunction Administration of eye drops that may affect efficacy assessment in the past 2 weeks prior to baseline (Steroid, glaucoma, allergy, antibiotic, eye drop gel, Cyclosporine, Diquafosol tetrasodium, etc) New administration or changing the dosage of systemic steroid drugs or immunosuppressant that may affect immune function in the past 4 weeks prior to baseline Administration of oral medication of Tetracycline, Isotretinoin in the past 4 weeks prior to baseline New administration or changing the dosage of Hydroxychloroquine medication in the past 12 weeks prior to baseline Planning to undergo eye surgery(including eyesight correction surgery) during the study period Wearing contact lenses during the study period History of ophthalmic surgery and trauma in the past 6 months prior to signing the ICF Participation in an investigational drug or device trial within 3 months prior to signing the ICF Hypersensitivity to the ingredients of this drug Genetic problems such as galactose intolerance, Lapp lactase deficiency, or glucose-galactose malabsorption A woman who has a positive Serum hCG test at the screening visit (Visit 1), or who does not agree to use at least one effective contraceptive method that is medically acceptable Take oral contraceptives during the study period Pregnant or lactating women A history of drug or alcohol abuse Any condition that, in the opinion of the investigator, would inappropriate to participate in the clinical trial

Ages Eligible for Study
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Minimum Age: 19 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Group 1<br>SA001 Low dose | Drug: SA001 Low dose<br>* 3 tablets b.i.d for 8 weeks<br>|
| Experimental: Group 2<br>SA001 Mid dose | Drug: SA001 Mid dose<br>* 3 tablets b.i.d for 8 weeks<br>|
| Experimental: Group 3<br>SA001 High dose | Drug: SA001 High dose<br>* 3 tablets b.i.d for 8 weeks<br>|
| Placebo Comparator: Placebo<br>Placebo | Drug: Placebo<br>* 3 tablets b.i.d for 8 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Tear Break-Up Time(TBUT) from baseline to Day 28 and Day 56 |  | Baseline(Day0), Day 28 and Day 56 | 
| Change in Ocular Staining Score(OSS) from baseline to Day 28 and Day 56 |  | Baseline(Day0), Day 28 and Day 56 | 
| Change in Schirmer Test score from baseline to Day 28 and Day 56 |  | Baseline(Day0), Day 28 and Day 56 | 
| Change in Standard Patient Evaluation of Eye Dryness questionnaire(SPEED) score from baseline to Day 28 and Day 56 |  | Baseline(Day0), Day 28 and Day 56 | 
| Change in Unstimulated whole saliva flow rate from baseline to Day 56 |  | Baseline(Day0) and Day 56 | 
| Change in The Xerostomia Inventory(XI) score from baseline to Day 56 |  | Baseline(Day0) and Day 56 | 
| Change in Anti-SSA/Ro levels from baseline to Day 56 |  | Baseline(Day0) and Day 56 | 
| Change in the average number of daily use of the artificial tears from baseline to Day 28 and Day 56 | Artificial tears may be applied if necessary. | Baseline(Day0), Day 28 and Day 56 |