Efficacy of Tofacitinib on Skin Thickening in Diffuse Cutaneous Systemic Sclerosis: A Comparative Study With Methotrexate

NCT07472166 | Completed Phase 4 DMC

• 1 other identifier: 5230
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to evaluate the efficacy of Tofacitinib compared to Methotrexate (MTX) in reducing skin thickening in patients with diffuse cutaneous systemic sclerosis (dcSSc). Systemic sclerosis is a chronic autoimmune disease characterized by skin fibrosis and internal organ involvement. Despite existing treatments, many patients continue to experience progressive skin and joint symptoms. Tofacitinib, a Janus kinase (JAK) inhibitor, has shown potential benefit in reducing inflammation and fibrosis in autoimmune diseases. This randomized controlled study will compare the change in Modified Rodnan Skin Score (mRSS), Clinical Disease Activity Index (CDAI), and 12-Item Short Form Survey (SF-12) between the Tofacitinib and Methotrexate groups over the study period. The study will also monitor adverse events and laboratory parameters to assess drug safety and tolerability.

Conditions

Scleroderma, Systemic

Keywords

Methotrexate; Skin thickening; mRSS; Autoimmune disease; Tofacitinib

Outcome Measures

Primary Outcomes (1)

• Change in Modified Rodnan Skin Score (mRSS) from baseline to week 24 Scale range: 0 (no skin thickening) to 51 (most severe skin thickening) Higher score indicate worse skin thickening (worse outcome)

  Baseline and Week 24

Study Arms (2)

Tofacitinib group

EXPERIMENTAL

Drug: Tofacitinib

Methotrexate group

ACTIVE COMPARATOR

Drug: Methotrexate

Interventions

Tofacitinib DRUG

Tofacitinib: 5mg orally twice daily for 24 weeks

Tofacitinib group

Methotrexate DRUG

25mg orally once weekly for 24 weeks

Methotrexate group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Diagnosed with systemic sclerosis (SSc) according to the 2013 ACR/EULAR classification criteria 2. Classified as having diffuse cutaneous systemic sclerosis (dcSSc), as defined by LeRoy and Medsger 3. Disease duration of ≤ 36 months, calculated from the onset of the first non-Raynaud's phenomenon manifestation.
• \. Modified Rodnan Skin Score (mRSS) between 10 and 45 at the time of screening.
• \. Concurrent use of calcium channel blockers and phosphodiesterase-5 inhibitors for the treatment of Raynaud's phenomenon or digital ulcers was permitted.
• \. Subjects were aged 18 years and older

You may not qualify if:

• \. Presence of a rheumatic disease other than systemic sclerosis, except for fibromyalgia or scleroderma-associated myopathy.
• \. Evidence of any active infection, including active tuberculosis 3. Abnormal laboratory values at screening, including:
• Hemoglobin \<9 g/dL
• White blood cell count \<3.0 × 10⁹/L
• Absolute neutrophil count \<1.2 × 10⁹/L
• Platelet count \<100 × 10⁹/L
• Absolute lymphocyte count \<0.75 × 10⁹/L
• ALT or AST \>3× the upper limit of normal (ULN)
• Estimated glomerular filtration rate (eGFR) \<60 mL/min/1.73 m²
• \. Pulmonary function showing forced vital capacity (FVC) ≤ 50% predicted, or DLCO (uncorrected for hemoglobin) ≤ 40% predicted.
• \. History of major surgery (including joint surgery) within 8 weeks prior to the screening visit.
• \. Use of oral corticosteroids 7. Diagnosis of latent TB at or within 30 days of screening. 8. Positive hepatitis B surface antigen or anti HCV test at or within 30 days of screening.
• \. Pregnant or breastfeeding women, females of childbearing potential who were unwilling or unable to use highly effective contraception during the study 10. History or evidence of malignancy 11. Receipt of a live or attenuated vaccine within 6 weeks prior to baseline

Sponsors & Collaborators

• Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh: lead

Study Sites (1)

Department of Rheumatology, Bangabandhu Sheikh Mujib Medical University (BSMMU)

Dhaka, 1000, Bangladesh

Location

MeSH Terms

Conditions

Scleroderma, Systemic; Autoimmune Diseases

Interventions

tofacitinib; Methotrexate

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Aminopterin; Pterins; Pteridines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Md. Abu Shahin, FCPS, MD

  Department of Rheumatology, Bangabandhu Sheikh Mujib Medical University (BSMMU), Dhaka, Bangladesh

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator & MD (Rheumatology) Resident

Study Record Dates

• First Submitted: March 9, 2026
• First Posted: March 16, 2026
• Study Start: December 21, 2024
• Primary Completion: August 30, 2025
• Study Completion: August 30, 2025
• Last Updated: March 16, 2026

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

BA (1)