Mycophenolate Mofetil for Systemic Sclerosis with Interstitial Lung Disease

(SSc-mILD Trial)

Recruiting at 3 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

Trial Summary

What is the purpose of this trial?

The goal of this pilot study is to assess the feasibility of a larger study on the efficacy of mycophenolate mofetil in people diagnosed with systemic sclerosis with mild lung involvement. Participants will be recruited over 12 months at 3 academic centers and assigned randomly to receive either mycophenolate mofetil or placebo, a look-alike substance that contains no active drug, for 96 weeks.

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.

What data supports the effectiveness of the drug Mycophenolate Mofetil for treating systemic sclerosis with interstitial lung disease?

Research shows that Mycophenolate Mofetil (MMF) can improve lung function in people with scleroderma-related interstitial lung disease, and it may be safer and better tolerated than another drug called cyclophosphamide. ^{1 2 3 4 5}

Is Mycophenolate Mofetil safe for humans?

Mycophenolate Mofetil (also known as Cellcept or Myfortic) has been studied for safety in people with systemic sclerosis-related interstitial lung disease, and adverse events (unwanted effects) occurred at similar rates in both the treatment and placebo groups, suggesting it is generally safe for humans. ^{2 3 4 6 7}

How does the drug Mycophenolate Mofetil differ from other treatments for systemic sclerosis-related interstitial lung disease?

Mycophenolate Mofetil (MMF) is unique because it is considered safer and better tolerated than cyclophosphamide, another drug used for this condition, and it may provide longer-lasting improvements. Unlike cyclophosphamide, which can have significant toxicity, MMF offers a more favorable safety profile for long-term use. ^{2 3 4 5 7}

Research Team

Sabrina Hoa, MD

Principal Investigator

Centre hospitalier de l'Université de Montréal (CHUM)

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Mycophenolate in scleroderma-associated interstitial lung disease: Real-world data from rheumatology and pulmonology clinics in South Asia. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Mycophenolate Mofetil Versus Placebo for Systemic Sclerosis-Related Interstitial Lung Disease: An Analysis of Scleroderma Lung Studies I and II. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Effect of mycophenolate mofetil on pulmonary function in scleroderma-associated interstitial lung disease. [2022]

4. China pubmed.ncbi.nlm.nih.gov

[Randomized control multi-center clinical study of mycophenolate mofetil and cyclophosphamide in the treatment of connective tissue disease related interstitial lung disease]. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Mycophenolate mofetil versus oral cyclophosphamide in scleroderma-related interstitial lung disease (SLS II): a randomised controlled, double-blind, parallel group trial. [2022]

6. Germany pubmed.ncbi.nlm.nih.gov

Effect of mycophenolate mofetil (MMF) on systemic sclerosis-related interstitial lung disease with mildly impaired lung function: a double-blind, placebo-controlled, randomized trial. [2021]

7. Italy 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. [2017]