Systemic Sclerosis With Lung InvolvementPhase 2SSc-mILDNCT05785065
Mycophenolate Mofetil in Systemic Sclerosis With Subclinical Interstitial Lung Disease
A Phase 2 study testing Mycophenolate Mofetil in people with Systemic Sclerosis With Lung Involvement and Systemic Sclerosis, run by Centre hospitalier de l'Université de Montréal (CHUM) at 3 sites in Canada.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Sclerosis With Lung Involvement, Systemic Sclerosis, Interstitial Lung Disease
- What is being tested?
- Mycophenolate Mofetil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 35 participants
- Where
- 3 sitesCanada
- Study dates
- 29 Aug 2024to Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Able and willing to provide informed consent and adhere to study protocol;
- Women and men of all race/ethnicity, aged 18 years and older;
- SSc based on 2013 ACR-EULAR classification criteria;
- 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;
- Diagnosis of ILD within 7 years before screening;
- Forced vital capacity of 80% predicted and above, on pulmonary function tests obtained within 6 months before screening;
- Able to communicate in French or English;
Who cannot join
- 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;
- Use of medications with putative lung disease-modifying properties:
- 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
- Cyclophosphamide within one year prior to screening
- Rituximab within 6 months prior to screening
- Cell therapies (including stem cell transplantation) within one year prior to screening
Show all 20Show fewer
- Current use of other biological, targeted synthetic or investigational products with immunosuppressive effects (e.g. TNF inhibitors, abatacept, tofacitinib) at time of screening
- Any contraindication to MMF, including:
- Pregnancy and/or breastfeeding
- Female of childbearing potential not using reliable method of contraception
- Persistent leucopenia (white blood cell count <3.0 x103/μL)
- Persistent thrombocytopenia (platelet count <100 x103/μL)
- Persistent anemia (hemoglobin <100 g/L)
- 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
- Uncontrolled congestive heart failure
- Active infection (lung or elsewhere)
- Active solid or hematological malignancy (other than basal cell cancer of the skin or cervical carcinoma in situ removed entirely by biopsy)
- Active peptic ulcer disease
- Other serious concomitant medical illness, unreliability or drug abuse that might compromise the patient's ability to safely take MMF
- Use of drugs or products with significant interactions with MMF
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Unité d'innovation thérapeutique, CHUM contact
Canada3 sites, 3 recruiting
- Recruiting
Centre hospitalier de l'Université de Montréal (CHUM)
Montreal, Quebec, H2X 3E4
Unité d'innovation thérapeutique, CHUM, 5148908000, uit.eligibilite.chum@ssss.gouv.qc.ca
Sabrina Hoa, MD MSc
- Not yet recruiting
Jewish General Hospital - CIUSSS-COMTL
Montreal, Quebec, H3T 1E2
Marie Hudson, MD
Marie Hudson, MD
- Not yet recruiting
Institut Universitaire de Cardiologie et Pneumologie de Québec
Québec, Quebec
Geneviève Dion, MD, genevieve.dion.med@ssss.gouv.qc.ca
Geneviève Dion, MD
About this study
Official title: 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
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.
Read the detailed description
Background: Systemic sclerosis (SSc, scleroderma) is a rare but life-threatening systemic autoimmune disease characterized by microvasculopathy, serum autoantibodies, inflammation and fibrosis of the skin and internal organs. Early rapidly progressive SSc remains the most lethal autoimmune rheumatic disease, with over 60% mortality at 5 years in high-risk patients. Interstitial lung disease (ILD) is the leading cause of SSc-related mortality and affects over half of SSc patients. SSc-ILD is currently treated with immunosuppressive and anti-fibrotic drugs, with the first-line treatment being mycophenolate mofetil (MMF), although treatments have modest benefits when initiated in advanced stages of disease. Emerging data suggest that earlier treatment, when lung function is still normal despite evidence of ILD on computed tomography scan ("subclinical SSc-ILD"), may lead to improved outcomes, suggesting a window of treatment opportunity.
Research Aims: The goal of the proposed pilot RCT is to establish the feasibility of a phase III RCT that will assess the efficacy of MMF in subclinical SSc-ILD. Specifically, we aim to:
- Determine the rate of patient recruitment at three centers over one year, and identify barriers and solutions to recruitment;
- Determine the proportion of participants receiving the allocated treatment and with complete primary efficacy outcome data at 48 and 96 weeks; and
- Generate preliminary data on clinical efficacy outcomes that will contribute information to the analysis of the phase III trial through a Bayesian inference framework.
Methods: Participants will be adults with SSc, ILD diagnosed within the past 3 years and a normal forced vital capacity (≥ 80%). Participants will be recruited over 12 months at 3 academic centers affiliated to the Canadian Scleroderma Research Group. Eligible participants will be assigned using stratified randomization to receive either MMF (up to 2 grams daily) or placebo for 96 weeks. The primary feasibility outcome will be the rate of recruitment per site over 12 months. A Bayesian approach will be used to estimate the probability of reaching the target sample size based on observed recruitment rates, with decision rules to continue, adapt, or stop the trial. Data collected on the primary clinical efficacy outcome (annual rate of decline in forced vital capacity over 96 weeks) will be used to inform the analysis of the phase III trial (as an informative prior) through a Bayesian inference framework.
- Being tested
- Mycophenolate Mofetil, Placebo
- Keywords
- Systemic sclerosis, Mycophenolate mofetil, Interstitial lung disease
- Medical subject headings
- Scleroderma, Systemic, Lung Diseases, Interstitial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
The participant will receive 500 mg to 1000 mg twice daily of mycophenolate mofetil administered orally for 96 weeks.
The dose scheduling will be as follow:
Weeks 1 and 2: 500 mg twice a day Weeks 3 and 4: 750 mg twice a day Weeks 5 to 96: 1000 mg twice a day
PlaceboOther
The participant will receive 500 mg to 1000 mg twice daily of placebo administered orally for 96 weeks.
The dose scheduling will be as follow:
Weeks 1 and 2: 500 mg twice a day Weeks 3 and 4: 750 mg twice a day Weeks 5 to 96: 1000 mg twice a day
| Study group | Role | What they receive |
|---|---|---|
| Mycophenolate mofetil | Experimental | 2 to 4 capsules of mycophenolate mofetil twice daily. Receives: Drug: Mycophenolate Mofetil |
| Placebo | Placebo comparator | 2 to 4 capsules of placebo twice daily. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total number of potentially eligible patients identified per site
Measured: Over one year
Proportion of potentially eligible patients who provide consent per site
Measured: Over one year
Proportion of consented participants who meet the eligibility criteria per site
Measured: Over one year
Monthly rate of randomized participants per site
Measured: Over one year
Adherence to treatment as assessed by Participant Dosing Diaries
Measured: From the first dose to the last dose taken for each participant, up to 96 weeks
Drug adherence rate as assessed by Pharmacy Accountability Logs
Measured: From the first dose to the last dose taken for each participant, up to 96 weeks
Adherence to the study protocol as assessed by the number of protocol deviations
Measured: Over total study period (up to 96 weeks per participant)
Proportion of participants intolerant to the study drug who discontinue trial treatment
Measured: Over total study period (up to 96 weeks per participant)
Proportion of participants receiving the allocated treatment at 48 weeks
Measured: At 48 weeks
Proportion of participants receiving the allocated treatment at 96 weeks
Measured: At 96 weeks
Proportion of participants with complete primary efficacy outcome data at 48 weeks
Measured: At 48 weeks
Proportion of participants with complete primary efficacy outcome data at 96 weeks
Measured: At 96 weeks
Proportion of participants lost to follow-up
Measured: Over total study period (up to 96 weeks per participant)
Secondary outcomes (1)
Frequency of treatment-related adverse events
Incidence and severity of AEs, with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5.0 (NCI CTCAE v5.0)
Measured: Over total study period (up to 96 weeks per participant)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2023
- Study start29 Aug 2024
- Primary completionDec 2026 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Sclerosis With Lung InvolvementStudies of the same condition, open and under way.
- More trials run by Centre hospitalier de l'Université de Montréal (CHUM)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Patients will be randomized to treatment group or placebo group in a 1:1 ratio and stratified according to the presence of anti-topoisomerase I autoantibody.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- McGill University, University of Calgary, Jewish General Hospital, Canadian Institutes of Health Research (CIHR), Sclérodermie Québec, Institut universitaire de cardiologie et de pneumologie de Québec, University Laval
- Responsible party
- Sponsor
- Investigators
- Sabrina Hoa, MD, Principal investigator, Centre hospitalier de l'Université de Montréal (CHUM)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05785065
- Sponsor's protocol ID
- MP-02-2023-11180
- Acronym
- SSc-mILD
- Status last verified
- Sep 2026
- First submitted
- 7 Feb 2023
- Last updated
- 29 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05785065
- Acronym / sponsor ID
- SSc-mILD / MP-02-2023-11180
- Keywords
- Systemic sclerosis, Mycophenolate mofetil, Interstitial lung disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- Centre hospitalier de l'Université de Montréal (CHUM)University, hospital or other
- Collaborators
- McGill University, University of Calgary, Jewish General Hospital, Canadian Institutes of Health Research (CIHR), Sclérodermie Québec, Institut universitaire de cardiologie et de pneumologie de Québec, University Laval
- Study start
- 29 Aug 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 27 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Background: Systemic sclerosis (SSc, scleroderma) is a rare but life-threatening systemic autoimmune disease characterized by microvasculopathy, serum autoantibodies, inflammation and fibrosis of the skin and internal organs. Early rapidly progressive SSc remains the most lethal autoimmune rheumatic disease, with over 60% mortality at 5 years in high-risk patients. Interstitial lung disease (ILD) is the leading cause of SSc-related mortality and affects over half of SSc patients. SSc-ILD is currently treated with immunosuppressive and anti-fibrotic drugs, with the first-line treatment being mycophenolate mofetil (MMF), although treatments have modest benefits when initiated in advanced stages of disease. Emerging data suggest that earlier treatment, when lung function is still normal despite evidence of ILD on computed tomography scan ("subclinical SSc-ILD"), may lead to improved outcomes, suggesting a window of treatment opportunity.
Research Aims: The goal of the proposed pilot RCT is to establish the feasibility of a phase III RCT that will assess the efficacy of MMF in subclinical SSc-ILD. Specifically, we aim to:
- Determine the rate of patient recruitment at three centers over one year, and identify barriers and solutions to recruitment;
- Determine the proportion of participants receiving the allocated treatment and with complete primary efficacy outcome data at 48 and 96 weeks; and
- Generate preliminary data on clinical efficacy outcomes that will contribute information to the analysis of the phase III trial through a Bayesian inference framework.
Methods: Participants will be adults with SSc, ILD diagnosed within the past 3 years and a normal forced vital capacity (≥ 80%). Participants will be recruited over 12 months at 3 academic centers affiliated to the Canadian Scleroderma Research Group. Eligible participants will be assigned using stratified randomization to receive either MMF (up to 2 grams daily) or placebo for 96 weeks. The primary feasibility outcome will be the rate of recruitment per site over 12 months. A Bayesian approach will be used to estimate the probability of reaching the target sample size based on observed recruitment rates, with decision rules to continue, adapt, or stop the trial. Data collected on the primary clinical efficacy outcome (annual rate of decline in forced vital capacity over 96 weeks) will be used to inform the analysis of the phase III trial (as an informative prior) through a Bayesian inference framework.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Patients will be randomized to treatment group or placebo group in a 1:1 ratio and stratified according to the presence of anti-topoisomerase I autoantibody.
| Arm | Type | Receives |
|---|---|---|
| Mycophenolate mofetil | Experimental | Drug: Mycophenolate Mofetil2 to 4 capsules of mycophenolate mofetil twice daily. |
| Placebo | Placebo comparator | Other: Placebo2 to 4 capsules of placebo twice daily. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Able and willing to provide informed consent and adhere to study protocol;
- Women and men of all race/ethnicity, aged 18 years and older;
- SSc based on 2013 ACR-EULAR classification criteria;
- 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;
- Diagnosis of ILD within 7 years before screening;
Key exclusion criteria (20 listed)
- 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;
- Use of medications with putative lung disease-modifying properties:
- Current use of other biological, targeted synthetic or investigational products with immunosuppressive effects (e.g. TNF inhibitors, abatacept, tofacitinib) at time of screening
- Any contraindication to MMF, including:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mycophenolate MofetilThe participant will receive 500 mg to 1000 mg twice daily of mycophenolate mofetil administered orally for 96 weeks. The dose scheduling will be as follow: Weeks 1 and 2: 500 mg twice a day Weeks 3 and 4: 750 mg twice a day Weeks 5 to 96: 1000 mg twice a day | Drug | Mycophenolate mofetil |
| PlaceboThe participant will receive 500 mg to 1000 mg twice daily of placebo administered orally for 96 weeks. The dose scheduling will be as follow: Weeks 1 and 2: 500 mg twice a day Weeks 3 and 4: 750 mg twice a day Weeks 5 to 96: 1000 mg twice a day | Other | Placebo |
Primary outcome
Total number of potentially eligible patients identified per site
Time frame: Over one year
Proportion of potentially eligible patients who provide consent per site
Time frame: Over one year
Proportion of consented participants who meet the eligibility criteria per site
Time frame: Over one year
Monthly rate of randomized participants per site
Time frame: Over one year
Adherence to treatment as assessed by Participant Dosing Diaries
Time frame: From the first dose to the last dose taken for each participant, up to 96 weeks
Drug adherence rate as assessed by Pharmacy Accountability Logs
Time frame: From the first dose to the last dose taken for each participant, up to 96 weeks
Adherence to the study protocol as assessed by the number of protocol deviations
Time frame: Over total study period (up to 96 weeks per participant)
Proportion of participants intolerant to the study drug who discontinue trial treatment
Time frame: Over total study period (up to 96 weeks per participant)
Proportion of participants receiving the allocated treatment at 48 weeks
Time frame: At 48 weeks
Proportion of participants receiving the allocated treatment at 96 weeks
Time frame: At 96 weeks
Proportion of participants with complete primary efficacy outcome data at 48 weeks
Time frame: At 48 weeks
Proportion of participants with complete primary efficacy outcome data at 96 weeks
Time frame: At 96 weeks
Proportion of participants lost to follow-up
Time frame: Over total study period (up to 96 weeks per participant)
1 secondary outcome
Frequency of treatment-related adverse events
Time frame: Over total study period (up to 96 weeks per participant)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Unité d'innovation thérapeutique, CHUM contact
Sabrina Hoa, MD principal investigator
Centre hospitalier de l'Université de Montréal (CHUM)
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mycophenolate Mofetil in people with Systemic Sclerosis With Lung Involvement and Systemic Sclerosis, run by Centre hospitalier de l'Université de Montréal (CHUM) at 3 sites in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sabrina Hoa, MD MSc (514-890-8000, sabrina.anh-tu.hoa.med@ssss.gouv.qc.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total number of potentially eligible patients identified per site (time frame: Over one year). The study lists 13 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2026.