Clinical trial NCT05087589: Efficacy, Safety and Immunological Evaluation of Tofacitinib in the Treatment of Primary Sjogren's Syndrome
Official title: Efficacy, Safety and Immunological Evaluation of Tofacitinib in the Treatment of Primary Sjögren's Syndrome:a Prospective Observational Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Primary Sjögren's Syndrome
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2021-10-21
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study aims to explore the clinical and immunological efficacy of tofacitinib on primary Sjögren's Syndrome

Detailed description: The investigators designed a single center, open-label, prospective study. Adults with active primary Sjögren's Syndrome will be enrolled, meeting the American College of Rheumatology(ACR) \& European allance of associations for rheumatology(EULAR)(2016) diagnostic criteria . Tofacitinib 5 mg bd was administered for 6 months to explore its efficacy and safety. The improvement of clinical and laboratory indexes was evaluated. Changes of immune cell subsets and cytokines were monitored.

Eligibility criteria: Inclusion Criteria:

1. Male or female \>18 years of age at screening visits
2. Patients meet the American-European Consensus Group 2002 classification criteria
3. The patient must be informed in writing of the consent to participate in the trial and the patient is expected to be able to comply with the requirements of the study follow-up plan and other protocols.
4. Dosing of antimalarials, prednisone or equivalent, cholinergic stimulants, and topical cyclosporine required to be stable for at least 4 weeks before screening and during study; maximum doses allowed:

   * Hydroxychloroquinone, 400 mg/day;
   * Prednisone, 10 mg/day

Exclusion Criteria:

Any subject meeting any of the following criteria should be excluded:

1. Laboratory abnormality:

   * Hb≤9 g/dl
   * Neutrophil \<1.0 x 109/l
   * lymphocyte\<0.5 x 109/l
2. Diagnosis of other autoimmune disease, or other sicca syndrome.
3. Use rituximab or other monoclonal antibodies within 6 months.
4. Received high doses of glucocorticoid (\>10 mg/d) within 1 month.
5. Serious complications: including heart failure (≥ New York Heart Association (NYHA) class III), renal insufficiency (creatinine clearance ≤ 30 ml/min), liver dysfunction (serum Alanine transaminase (ALT) or aspartate aminotransferase (AST) greater than three times the upper limit of normal, or total bilirubin greater than Normal upper limit)
6. Known allergies, hyperreactivity or intolerance of tofacitinib or its excipients.
7. Have a serious infection needing hospitalization (including but not limited to hepatitis, pneumonia, bacteremia, pyelonephritis, EB virus, tuberculosis infection), or use intravenous antibiotics to treat infection in 2 months before the enrollment.
8. Infection with HIV (HIV antibody positive serology) or hepatitis C (Hep C antibody positive serology). If seropositive, it is recommended to consult a doctor who has expertise in treating HIV or hepatitis C virus infection.
9. Any known history of malignancy in the past 5 years (except for non-melanoma skin cancer, non-melanoma skin cancer or cervical tumor without recurrence within 3 months after surgical cure prior to the first study preparation).
10. Uncontrolled mental or emotional disorders, including a history of drug and alcohol abuse over the past 3 years, may hinder the successful completion of the study.
11. Pregnant, lactating women (WCBP) are reluctant to use medically approved contraceptives during treatment and 12 months after treatment.

Primary outcomes:
measure: Immunological Responses
timeFrame: week 24
Secondary outcomes:
measure: Improvements in EULAR SS patient-reported index (ESSPRI), other clinical and immunological parameters
timeFrame: week 24
measure: Safety and tolerability of tofacitinib as assessed by incidence of adverse events reported and observed
timeFrame: up tp 24 weeks
Other outcomes:
Locations:
city: Beijing
country: China
facility: Department of Rheumatology and Immunology, Peking University People's Hospital
state: Beijing
Study officials:
affiliation: Peking University Institute of Rheuamotology and Immunology
name: Zhanguo Li
role: PRINCIPAL_INVESTIGATOR
References: