Comparing Tofacitinib and Cyclophosphamide for Skin Thickening in Systemic Sclerosis

A Study Evaluating the Effectiveness of Tofacitinib in Systemic Sclerosis

This study is testing whether a new medication called tofacitinib can help reduce skin thickening in adults with early diffuse systemic sclerosis better than the standard treatment with cyclophosphamide.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 44 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh (Academic / other)
• Drugs / interventions: prednisone, tofacitinib, cyclophosphamide
• Locations: 1 site (Dhaka, Shahbag)
• Trial ID: NCT06044844 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the effectiveness of tofacitinib, administered at a dose of 5mg twice daily, compared to cyclophosphamide, given at 500mg/m2 per month, in treating early diffuse cutaneous systemic sclerosis (dcSSc). The study will focus on measuring changes in skin thickening, as assessed by the modified Rodnan skin score (mRSS), in participants diagnosed with dcSSc. Eligible participants will be adults aged 18 to 70 years with a disease duration of 60 months or less and a baseline mRSS score between 10 and 45. The trial will also allow for the use of certain medications like oral corticosteroids at stable doses.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 to 70 years diagnosed with early diffuse cutaneous systemic sclerosis and a modified Rodnan skin score between 10 and 45.

Not a fit: Patients with severe pulmonary disease or those requiring high doses of oral corticosteroids may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for patients with early diffuse cutaneous systemic sclerosis.

How similar studies have performed: While this approach is being evaluated in this trial, previous studies have shown varying results with similar treatments in systemic sclerosis, indicating a need for further investigation.

Eligibility criteria

* Inclusion criteria:

  1. Diagnosis of SSc, as classified using the 2013 American College of Rheumatology
  2. dcSSc as defined by 2001 LeRoy and Medsge
  3. Disease duration ≤ 60 months (defined as time from the first non-Raynaud phenomenon manifestation)
  4. mRSS units ≥ 10 and ≤ 45 at screening.
  5. Oral corticosteroids (≤ 10 mg/day of prednisone or equivalent) are permitted if the patient is on a stable dose regimen for ≥ 2 weeks prior to and including the baseline visit.
  6. Calcium channel blocker and PDFE-5 inhibitors for Raynaud's and digital ulcers are permitted to use as oral monotherapy
  7. Age ≥ 18 years and ≤ 70 years
  8. Ability to provide informed consent.

Exclusion Criteria:

Subjects with any of the following characteristics/conditions will not be included in the study:

1. Any infection at screening .
2. Oral corticosteroids \>10 mg/day of prednisone or equivalent.
3. Pulmonary disease with FVC ≤ 35% of predicted.
4. Subjects at risk for tuberculosis (TB).Specifically excluded from this study with a history of active TB within the last 3 years and current clinical, radiographic, or laboratory evidence of active TB.j
5. Latent TB at or within 30 days of screening.
6. Positive for hepatitis B surface antigen at or within 30 days of screening.
7. Positive for hepatitis C antigen at or within 30 days of screening.
8. Current or recent history of uncontrolled clinically significant renal, hepatic, hematologic, gastrointestinal, metabolic, endocrine, pulmonary, cardiac or neurologic disease.
9. History of diverticulitis or chronic, ulcerative lower GI disease such as Crohns disease, ulcerative colitis, or other symptomatic, lower GI conditions that might predispose a patient to perforations.
10. Pregnant or breastfeeding female subjects and female subjects of childbearing potential who are unwilling or unable to use a highly effective method of contraception as outlined in the protocol for the duration of the study and for at least 28 days after discontinuation of study drug.
11. History of any malignancy in the last 5 years with the exception of adequately treated or excised basal cell or squamous cell or cervical cancer in situ.
12. History of SSc Renal Crisis within the 6 months prior to baseline.
13. History of live/attenuated vaccine ≤ 6 weeks prior to baseline
14. Any of the following lab results at screening:

    * Hemoglobin \<9 g/dL or Hematocrit \<30%
    * White Blood Cell count \<3.0 x 109/L;
    * Absolute Neutrophil count \<1.2 x 109/L;
    * Platelet count \<100 x 109/L;
    * Absolute Lymphocyte count \<0.75 x 109/L.
    * ALT or AST \> 3 × the upper limit of normal (ULN) of normal at screening or any
    * Total bilirubin \> ULN at Screening.
    * Estimated glomerular filtration rate \[GFR\] \<40mL/min/1.73 m2

Where this trial is running

Dhaka, Shahbag

• Nabil Amin Khan — Dhaka, Shahbag, Bangladesh (Recruiting)

Study contacts

• Principal investigator: Nabil Khan, MBBS — resident
• Study coordinator: Nabil khan, MBBS

Conditions Efficacy of Tofacitinib in the Systemic Sclerosis