R-2487 in Patients With Sjogren's Syndrome (SS) (R-2487-SS-01)

A Single and Repeat Dosing Study of the Safety, Drug Exposure and Clinical Activity of R-2487 in Patients With Sjogren's Syndrome (SS)

The goal of this study is to determine the safety and tolerability of orally taken probiotic (R-2487) in patients with Sjogren's Syndrome.

Patients will take an oral dosage of probiotic (R-2487) and physicians will assess and measure their Sjogren's Syndrome. Blood and fecal evaluations of inflammation and assessment of probiotic (R-2487) on fecal level will also be measured.

Study Overview

• Status: Not yet recruiting
• Conditions: Sjogren's Syndrome; Sjögren; Sjögren Syndrome, Unspecified
• Intervention / Treatment: Drug: R-2487

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Christian Freguia, PhD; Phone Number: 2159231818; Email: cfreguia@risetherapeutics.com
• Study Contact Backup: Name: Janet Stephens, PhD; Phone Number: 650-417-8556; Email: jstephens@risetherapeutics.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Diagnosis of SS according to American-European Consensus Group Criteria
• Able to provide informed consent
• Subjects receiving prednisone (10 mg or less/day) must be on a stable dose for more than 2 weeks
• All male and female subjects who are biologically capable of having children must agree to use medically acceptable method of birth control for the duration of the study. All female subjects who are biologically capable of having children must have a negative pregnancy test result before administration of investigational product.
• The use of probiotics prior to study enrollment is accepted; however, during the course of the study, the use of probiotics is forbidden.

Exclusion Criteria:

• No known active overlapping or associated other autoimmune disease
• Prior allogenic or autologous bone marrow or organ transplantation
• Subjects with prior irradiation to the head and neck, including radioactive iodine treatment for hyperthyroidism
• Subjects who have a present malignancy or previous malignancy within the last 5 years prior to screening (except documented history of cured non-metastatic squamous or basal cell skin carcinoma or cervical carcinoma in situ). Subjects who had a screening procedure that is suspicious for malignancy, and in whom the possibility of malignancy cannot be reasonably excluded following additional clinical, laboratory or other diagnostic evaluations.
• Subjects with positive results for human immunodeficiency virus (HIV), hepatitis A virus (HAV), hepatitis B virus (HBV), or hepatitis C virus (HCV)
• Subjects with active viral, bacterial, or fungal infection, or history of severe opportunistic infection within the preceding 3 months, or COVID-19 infection in the past 3 months
• Subjects with evidence of active or latent tuberculosis
• Active infection of the salivary or lacrimal glands
• Prior immunotherapy, biologics, or investigational therapy must have completed at least 5 half-lives or 30 days, whichever is longer, prior to the first dose of R-2487 DP
• Pregnant or breastfeeding women
• Current clinical findings of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, cardiac, endocrine, neurological, or cerebral disease with laboratory values as following:

Hemoglobin level < 9.0 g/dL

Absolute white blood cell (WBC) count of <3.0×109/L (<3000/mm3), or absolute neutrophil count of <1.2×109/L (<1200/mm3), or absolute lymphocyte count of <0.8×109/L (<800/mm3).

Thrombocytopenia, defined by platelet count <100×109/L (<100,000/mm3)

Chronic kidney disease defined as Estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m2, based on the age appropriate calculation.

Proteinuria ≥3+.

Total bilirubin (T-bili), aspartate aminotransferase (AST), alanine aminotransferase (ALT) more than 1.5 times upper limit of normal (ULN).

Previously diagnosed hepatic cirrhosis (Child Pugh A or higher) or previously diagnosed significant liver fibrosis (> F3)

• Any form of vaccination in the last 30 days, to include but not limited to influenza, COVID, shingles, tetanus, hepatitis, pneumonia, HPV, DPT, MMR, and polio.
• Subjects should not receive any of the following medications:

Rituximab or belimumab within 6 months prior to Day 1 Abatacept within 3 months prior to Day 1 Infliximab, Adalimumab, Certolizumab, Tocilizumab, Cyclosporine, or Mycophenolate mofetil within 2 months prior to Day 1 Etanercept, Anakinra, Immunoglobulin, or blood products within 28 days prior to Day 1

• Prior immunotherapy, including systemic corticosteroids, such prednisone, biologics, Janus kinase (JAK) inhibitors (such as tofacitinib, or upadacitinib), ozanimod, or investigational therapy must have completed at least 5 half-lives or 30 days, whichever is longer, prior to Day 0, unless otherwise specified. In the case of cell-depleting therapies, such as B or T cell depletion, cell counts must have recovered to acceptable or baseline levels (use of licensed agents for indications not listed in the package insert is permitted).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Open Label; Probiotic	Drug: R-2487; R-2487 DP; Other Names: Probiotic

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and severity of adverse events and their relationship to R-2487 (probiotic) administration	To assess the number of participants with treatment-related adverse events after taking R-2487 (probiotic)	Baseline through Week 4

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in disease activity through Clinical European League Against Rheumatism Sjögren's Syndrome Disease Activity Index (ClinESSDAI)	Change from Baseline in Disease Activity Score at Week 4 to assess the disease severity of Sjogren's syndrome using data without laboratory results	Baseline through Week 4
Change in Patient reported general health questionnaire Short Form Health Survey-36(SF-36)	Baseline Short Form Health Survey-36 (SF-36) Score over time. The Baseline Short Form Health Survey is the numerical sum of 36 patient reported outcome parameters of quality of life. The higher the score the more favorable the reflection of the quality of life.	Baseline through week 4

Collaborators and Investigators

• Sponsor: Rise Therapeutics LLC

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2024
• Primary Completion (Estimated): August 30, 2026
• Study Completion (Estimated): August 30, 2026

Study Registration Dates

• First Submitted: February 28, 2024
• First Submitted That Met QC Criteria: March 5, 2024
• First Posted (Actual): March 7, 2024

Study Record Updates

• Last Update Posted (Actual): April 12, 2024
• Last Update Submitted That Met QC Criteria: April 10, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Sjogren's Syndrome
• 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: R-2487-SS-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No