Using Mycophenolate Mofetil for Systemic Sclerosis with Mild Lung Issues

Randomized Double-Blind Placebo-Controlled Clinical Trial to Assess the Efficacy of Mycophenolate Mofetil in Subclinical Interstitial Lung Disease Associated With Systemic Sclerosis: a Feasibility Study

This study is testing if a medication called mycophenolate mofetil can help people with systemic sclerosis and mild lung problems feel better over 96 weeks.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 35 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Centre hospitalier de l'Université de Montréal (CHUM) (other)
• Drugs / interventions: tocilizumab, Rituximab, tofacitinib, Cyclophosphamide, Prednisone
• Locations: 3 sites (Montreal, Quebec and 2 other locations)
• Trial ID: NCT05785065 on ClinicalTrials.gov

What this trial studies

This pilot study aims to evaluate the feasibility of a larger clinical trial assessing the effectiveness of mycophenolate mofetil (MMF) in patients with systemic sclerosis who have mild interstitial lung disease. Participants will be randomly assigned to receive either MMF or a placebo for a duration of 96 weeks. The study will take place over 12 months across three academic centers, focusing on recruitment rates and preliminary efficacy outcomes. The goal is to gather data that could support a future phase III trial.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older diagnosed with systemic sclerosis and mild interstitial lung disease as confirmed by imaging.

Not a fit: Patients with progressive pulmonary fibrosis or those who have advanced lung disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved treatment options for patients with systemic sclerosis and mild lung involvement.

How similar studies have performed: While studies on mycophenolate mofetil for systemic sclerosis exist, this specific approach targeting subclinical interstitial lung disease is relatively novel.

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

Where this trial is running

Montreal, Quebec and 2 other locations

• Centre hospitalier de l'Université de Montréal (CHUM) — Montreal, Quebec, Canada (RECRUITING)
• Jewish General Hospital - CIUSSS-COMTL — Montreal, Quebec, Canada (NOT_YET_RECRUITING)
• Institut Universitaire de Cardiologie et Pneumologie de Québec — Québec, Quebec, Canada (NOT_YET_RECRUITING)

Study contacts

• Principal investigator: Sabrina Hoa, MD — Centre hospitalier de l'Université de Montréal (CHUM)
• Study coordinator: Sabrina Hoa, MD MSc

Conditions: Systemic Sclerosis With Lung Involvement, Systemic Sclerosis, Interstitial Lung Disease, Systemic sclerosis, Mycophenolate mofetil, Interstitial lung disease