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Comparison of treatment options for systemic sclerosis

Not Applicable
Conditions
Health Condition 1: M349- Systemic sclerosis, unspecified
Registration Number
CTRI/2019/02/017795
Lead Sponsor
ICMR grant applied
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Yet Recruiting
Sex
Not specified
Target Recruitment
0
Inclusion Criteria

1. Patients with a diagnosis of systemic sclerosis as per the 2013 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) classification criteria, irrespective of their gender, will be included in the study. They may have either diffuse cutaneous or limited cutaneous systemic sclerosis.

2. Age >=12 years

3. Patients with the following evidence of pulmonary involvement:

% predicted FVC <= 70% and/or

HRCT chest showing involvement of >= 20% of lung area with interstitial lung disease.

Exclusion Criteria

1. Patients with overlap syndrome.

2. Diagnosis of clinically significant resting pulmonary hypertension requiring treatment diagnosed on echocardiography (defined as tricuspid regurgitation velocity >= 4m/s, eqivalent to estimated pulmonary artery systolic pressure of 64 mm Hg) while evaluation of the patient during study.

3. Evidence of uncontrolled congestive heart failure, unstable ischemic heart disease, history of pulmonary embolism, or cardiac arrhythmia requiring chronic anticoagulation.

4. FVC <= 15% at baseline.

5. Patients who cannot perform spirometry for evaluation of pulmonary function tests despite sufficient counselling about the procedure.

6. More than 50% area of lung showing fibrosis on HRCT chest at baseline.

7. Hematologic abnormality at screening including:

•Leukopenia (white blood cells [WBC] < 4.0x103/µl).

•Thrombocytopenia (platelet count < 150.0x103/µl).

•Clinically significant anemia (hemoglobin < 7 g/dl).

Participants with an identified and correctable etiology will be eligible if repeat testing shows values greater than the above mentioned cut-off.

8. A diagnosis of chronic liver disease or abnormal baseline liver function tests (total bilirubin or liver enzymes, alanine aminotransferase and aspartate aminotransferase > 2.0 times the upper normal limit).

9. Serum creatinine >2.0mg/dl

10. Pregnancy and/or breast feeding

11. If of child bearing potential (a female participant < 55 years of age who has not been postmenopausal for >= 5 years or who has not had a hysterectomy and/or oophorectomy), failure to employ reliable means of contraception.

12. Prior use of oral or intravenous cyclophosphamide, MMF, azathioprine or other putative disease modifying medications in last 3 months (assessed at baseline).

13. Current use, or use within the 30 days prior to their baseline visit, of prednisone (or equivalent) in dose of 40 mg/day.

14. Active infection (Hepatitis B, Hepatitis C, tuberculosis, HIV) whose management would be compromised by immunosuppression.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
1. To measure absolute and percentage change in MRSS score from baseline to 2 years. <br/ ><br>2. To measure absolute and percentage change in pulmonary function tests (FVC) from baseline to 2 years.Timepoint: 2 years
Secondary Outcome Measures
NameTimeMethod
Change from baseline at 2 years, in skin histopathology findings.Timepoint: 2 years;Change from baseline in Dermatology life quality index (DLQI) scores at 2 years.Timepoint: 2 years;Change from baseline in Scleroderma Health Assessment Questionnaire (HAQ) scores at 2 years.Timepoint: 2 years;To assess the change in 6-minute walk test at the end of 2 years of treatment as compared to baseline.Timepoint: 2 years;To assess the changes from baseline in HRCT chest patterns and quantitative HRCT scores for interstitial lung disease at the end of 2 years of treatment.Timepoint: 2 years;To assess the changes in ultrasonography of skin at the end of 2 years of treatment as compared to baseline.Timepoint: 2 years;To assess the changes on Echo (pulmonary artery hypertension) at the end of 2 years of treatment as compared to baseline.Timepoint: 2 years;To measure absolute and percentage change in pulmonary function tests (DLCO) from baseline to 2 years.Timepoint: 2 years
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