Mycophenolate Mofetil in Systemic Sclerosis With Subclinical Interstitial Lung Disease

Launched by CENTRE HOSPITALIER DE L'UNIVERSITÉ DE MONTRÉAL (CHUM) · Mar 14, 2023

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness of a medication called mycophenolate mofetil in people with systemic sclerosis (a condition that affects the skin and internal organs) who also have mild lung involvement. The researchers want to see if this medication can help improve lung function in these patients. Over the next year, participants aged 18 and older will be recruited from three academic centers. They will be randomly assigned to either receive the active medication or a placebo (a look-alike pill with no active ingredients) for 96 weeks.

To be eligible for this trial, participants must have been diagnosed with systemic sclerosis and have specific lung conditions that show mild fibrosis (scarring) affecting less than 20% of their lungs. They should also be able to understand and communicate in English or French. Participants can expect to take the medication or placebo for nearly two years while being monitored for any changes in their health. This trial is an important step in determining if mycophenolate mofetil can be a beneficial treatment for managing lung issues in patients with systemic sclerosis.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Able and willing to provide informed consent and adhere to study protocol;
• • 2. Women and men of all race/ethnicity, aged 18 years and older;
• • 3. SSc based on 2013 ACR-EULAR classification criteria;
• • 4. Presence of interstitial lung disease on HRCT scan, obtained within 12 months before screening, that shows fibrosis affecting less than 20% of the lungs, as confirmed by an expert radiologist;
• • 5. Diagnosis of ILD within 7 years before screening;
• • 6. Forced vital capacity of 80% predicted and above, on pulmonary function tests obtained within 6 months before screening;
• • 7. Able to communicate in French or English;
• Exclusion Criteria:
• • 1. Progressive pulmonary fibrosis, defined as at least two of three criteria (worsening symptoms, radiological progression, and physiological progression) occurring within the past year with no alternative explanation, as defined by the 2022 ATS/ERS/JRS/ALAT Clinical Practice Guideline;
• 2. Use of medications with putative lung disease-modifying properties:
• • 1. Current use of MMF, mycophenolic acid, azathioprine, calcineurin inhibitors (e.g. tacrolimus, cyclosporin A), tocilizumab, nintedanib, pirfenidone or corticosteroids (Prednisone equivalent dose \>10 mg/day) at time of screening
• • 2. Cyclophosphamide within one year prior to screening
• • 3. Rituximab within 6 months prior to screening
• • 4. Cell therapies (including stem cell transplantation) within one year prior to screening
• • 3. Current use of other biological, targeted synthetic or investigational products with immunosuppressive effects (e.g. TNF inhibitors, abatacept, tofacitinib) at time of screening
• 4. Any contraindication to MMF, including:
• • 1. Pregnancy and/or breastfeeding
• • 2. Female of childbearing potential not using reliable method of contraception
• • 3. Persistent leucopenia (white blood cell count \<3.0 x103/μL)
• • 4. Persistent thrombocytopenia (platelet count \<100 x103/μL)
• • 5. Persistent anemia (hemoglobin \<100 g/L)
• • 6. Baseline liver enzymes (alanine transaminase (ALT) or aspartate transaminase (AST)) or bilirubin \>1.5 times the upper limit of normal, other than due to Gilbert's disease
• • 7. Uncontrolled congestive heart failure
• • 8. Active infection (lung or elsewhere)
• • 9. Active solid or hematological malignancy (other than basal cell cancer of the skin or cervical carcinoma in situ removed entirely by biopsy)
• • 10. Active peptic ulcer disease
• • 11. Other serious concomitant medical illness, unreliability or drug abuse that might compromise the patient's ability to safely take MMF
• • 12. Use of drugs or products with significant interactions with MMF