Mycophenolate Mofetil for Systemic Sclerosis with Interstitial Lung Disease

(SSc-mILD Trial)

Recruiting at 4 trial locations

Overseen By Unité d'innovation thérapeutique, CHUM

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Centre hospitalier de l'Université de Montréal (CHUM)

Must not be taking: MMF, Corticosteroids, Cyclophosphamide, others

Disqualifiers: Pregnancy, Active infection, Cancer, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 96 weeks

35 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether mycophenolate mofetil, an immunosuppressant medication, can aid individuals with systemic sclerosis, a condition where the immune system attacks the body, causing thick skin and organ issues, particularly affecting the lungs. Researchers aim to determine if this medication is effective, compared to a placebo, in treating mild lung involvement over 96 weeks. Individuals diagnosed with systemic sclerosis and interstitial lung disease, a lung condition with scarring, but without severe lung issues, might be suitable candidates. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications that affect lung disease, such as mycophenolate mofetil, azathioprine, and some others, before joining. If you're on any of these, you may need to stop them before participating.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that mycophenolate mofetil is usually well-tolerated by people with systemic sclerosis and interstitial lung disease. Studies have found that it can slow the decline in lung function and may even improve quality of life. Observational data suggest that mycophenolate can help stabilize lung and skin problems in these patients. A meta-analysis, which combines data from multiple studies, supported that mycophenolate is a safe option for people with these conditions. Overall, these findings indicate that the treatment appears safe. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for systemic sclerosis with interstitial lung disease, which often include immunosuppressants like cyclophosphamide and azathioprine, mycophenolate mofetil stands out because it selectively targets and suppresses the proliferation of T and B lymphocytes, which are key players in the autoimmune process. This targeted action could potentially lead to fewer side effects compared to broader immunosuppressive therapies. Researchers are particularly excited about mycophenolate mofetil because it has shown promise in preserving lung function while offering a more tailored approach to managing the disease.

What evidence suggests that mycophenolate mofetil might be an effective treatment for systemic sclerosis with interstitial lung disease?

Research has shown that mycophenolate mofetil (MMF), which participants in this trial may receive, can help treat systemic sclerosis with interstitial lung disease (SSc-ILD). Studies have found that MMF can improve or maintain lung function, measured by forced vital capacity. It also slows disease progression and enhances patients' quality of life. Additionally, MMF can reduce the progression of lung disease and related health issues. Overall, MMF serves as a helpful treatment option for people with SSc-ILD. ^{2 4 6 7 8}

Who Is on the Research Team?

Sabrina Hoa, MD

Principal Investigator

Centre hospitalier de l'Université de Montréal (CHUM)

Are You a Good Fit for This Trial?

This trial is for adults over 18 with systemic sclerosis and mild lung involvement, diagnosed within the last 7 years. Participants must have a stable lung function and be able to consent and follow the study plan. They can't join if they've had worsening lung fibrosis in the past year, are on certain other medications, pregnant or not using contraception, have blood disorders or liver issues, heart failure, active infections or cancer.

Inclusion Criteria

My lung function test shows I breathe well, meeting the required standard.

I have been diagnosed with systemic sclerosis according to the 2013 criteria.

My lung scan shows less than 20% fibrosis, confirmed by a specialist.

Exclusion Criteria

I do not have any health conditions or situations that would make it unsafe for me to take MMF.

I am taking medication that could affect my lung condition.

My lung scarring has worsened in the last year without other causes.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either mycophenolate mofetil or placebo for 96 weeks

96 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Mycophenolate Mofetil
• Placebo

Trial Overview

The study tests Mycophenolate Mofetil (MMF) against a placebo in people with systemic sclerosis who also have subclinical interstitial lung disease. It's designed to see if it's feasible to do a larger study on MMF's effectiveness for these patients over a period of 96 weeks.

How Is the Trial Designed?

Group I: Mycophenolate mofetil Experimental Treatment 1 Intervention

2 to 4 capsules of mycophenolate mofetil twice daily.

Group II: Placebo Placebo Group 1 Intervention

2 to 4 capsules of placebo twice daily.

Mycophenolate Mofetil is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Cellcept for:

• Prevention of acute organ rejection in kidney, liver, and heart transplant patients
• Treatment of autoimmune diseases such as lupus nephritis and rheumatoid arthritis

Approved in United States as Cellcept for:

• Prophylaxis of organ rejection in patients receiving allogeneic renal, cardiac, or hepatic transplants
• Treatment of autoimmune diseases such as lupus nephritis and rheumatoid arthritis

Approved in Canada as Cellcept for:

• Prevention of acute organ rejection in kidney, liver, and heart transplant patients
• Treatment of autoimmune diseases such as lupus nephritis and rheumatoid arthritis

Approved in Japan as Cellcept for:

• Prevention of acute organ rejection in kidney, liver, and heart transplant patients
• Treatment of autoimmune diseases such as lupus nephritis and rheumatoid arthritis

Who Is Running the Clinical Trial?

Centre hospitalier de l'Université de Montréal (CHUM)

Lead Sponsor

Trials

389

Recruited

143,000+

St. Joseph's Healthcare Hamilton

Collaborator

Trials

203

Recruited

26,900+

McGill University

Collaborator

Trials

421

Recruited

1,017,000+

University of Calgary

Collaborator

Trials

827

Recruited

902,000+

Canadian Institutes of Health Research (CIHR)

Collaborator

Trials

1,417

Recruited

26,550,000+

Sclérodermie Québec

Collaborator

Trials

1

Recruited

40+

Institut universitaire de cardiologie et de pneumologie de Québec, University Laval

Collaborator

Trials

26

Recruited

6,300+

Jewish General Hospital

Collaborator

Trials

144

Recruited

283,000+

Published Research Related to This Trial

In a study of patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD), mycophenolate mofetil (MMF) treatment led to stable pulmonary function over 36 months, while azathioprine (AZA) did not show significant improvement in lung function.

Patients treated with MMF experienced fewer early adverse events leading to treatment discontinuation compared to those on AZA, suggesting that MMF may be a safer option for managing SSc-ILD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mycophenolate mofetil is an effective and safe option for the management of systemic sclerosis-associated interstitial lung disease: results from the Australian Scleroderma Cohort Study. Owen, C., Ngian, GS., Elford, K., et al.[2017]

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/31813058/

Effect of mycophenolate mofetil (MMF) on systemic ...

In this pilot study, MMF did not result in significant improvement in lung function in SSc-ILD with minimally impaired lung function, but was effective in ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8922665/

Mycophenolate in scleroderma-associated interstitial lung ...

Mycophenolate mofetil/mycophenolate sodium was efficacious in improving /stabilizing forced vital capacity irrespective of the baseline high-resolution ...

3.

atsjournals.org

atsjournals.org/doi/full/10.1513/AnnalsATS.202301-054OC

Mycophenolate in Patients with Systemic Sclerosis ...

Mycophenolate use in patients with SSc-ILD is associated with statistically significant improvements in disease progression and quality-of-life measures ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT02896205?cond=Lung%20Diseases,%20Interstitial&viewType=Table&term=Mycophenolate%20Mofetil&rank=3

Study to Compare the Efficacy of Mycophenolate Mofetil in ...

Treatment with immunosuppression drugs helps in retarding the progression of interstitial lung disease (ILD) and improves the morbidity and mortality among ...

5.

sciencedirect.com

sciencedirect.com/science/article/abs/pii/S2212534524001722

Advancements in the treatment of interstitial lung disease ...

This review examines the treatment of ILD in patients with SSc with the approval of MMF in Japan based on the latest American Thoracic Society guideline.

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC5444590/

Mycophenolate mofetil for scleroderma-related interstitial lung ...

MMF is well tolerated and slows the rate of decline in lung function in SSc-ILD patients, even at doses lower at 3 g/day.

7.

journals.plos.org

journals.plos.org/plosone/article?id=10.1371/journal.pone.0124205

Safety and Effectiveness of Mycophenolate in Systemic ...

Observational data suggests mycophenolate may be effective in improving or stabilizing interstitial lung disease, and skin involvement.

8.

onlinelibrary.wiley.com

onlinelibrary.wiley.com/doi/10.1155/2012/143637

Effect and Safety of Mycophenolate Mofetil or Sodium in ...

Meta-analysis data suggest that MMF is a safe therapeutic modality which was associated with functional stabilization in patients with SSc-ILD.