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

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

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.

Study Overview

• Status: Completed
• Conditions: Primary Sjögren's Syndrome (pSS)
• Intervention / Treatment: Drug: SA001 Low dose; Drug: SA001 Mid dose; Drug: SA001 High dose; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 28
• Phase: Phase 2

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 03722
    • Yonsei University Health System, Severance Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 19 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; 3 tablets b.i.d for 8 weeks
Experimental: Group 1; SA001 Low dose	Drug: SA001 Low dose; 3 tablets b.i.d for 8 weeks
Experimental: Group 2; SA001 Mid dose	Drug: SA001 Mid dose; 3 tablets b.i.d for 8 weeks
Experimental: Group 3; SA001 High dose	Drug: SA001 High dose; 3 tablets b.i.d for 8 weeks

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Samjin Pharmaceutical Co., Ltd.
• Investigators: Principal Investigator: Kyoung Yul Seo, Ph.D., Yonsei University Health System, Severance Hospital

Study record dates

Study Major Dates

• Study Start (Actual): May 29, 2020
• Primary Completion (Actual): February 25, 2022
• Study Completion (Actual): March 21, 2022

Study Registration Dates

• First Submitted: February 24, 2022
• First Submitted That Met QC Criteria: March 3, 2022
• First Posted (Actual): March 8, 2022

Study Record Updates

• Last Update Posted (Actual): February 17, 2023
• Last Update Submitted That Met QC Criteria: February 16, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Eye Diseases; Disease; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Arthritis; Stomatognathic Diseases; Mouth Diseases; Lacrimal Apparatus Diseases; Arthritis, Rheumatoid; Xerostomia; Salivary Gland Diseases; Dry Eye Syndromes; Syndrome; Sjogren's Syndrome
• Other Study ID Numbers: SA001_04

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No