Mycophenolate Mofetil: 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
Placebo: 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 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. 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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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
Primary outcome measure(s)
Total number of potentially eligible patients identified per site — Over one year
Proportion of potentially eligible patients who provide consent per site — Over one year
Proportion of consented participants who meet the eligibility criteria per site — Over one year
Monthly rate of randomized participants per site — Over one year
Adherence to treatment as assessed by Participant Dosing Diaries — 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 — 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 — Over total study period (up to 96 weeks per participant)
Proportion of participants intolerant to the study drug who discontinue trial treatment — Over total study period (up to 96 weeks per participant)
Proportion of participants receiving the allocated treatment at 48 weeks — At 48 weeks
Proportion of participants receiving the allocated treatment at 96 weeks — At 96 weeks
Proportion of participants with complete primary efficacy outcome data at 48 weeks — At 48 weeks
Proportion of participants with complete primary efficacy outcome data at 96 weeks — At 96 weeks
Proportion of participants lost to follow-up — Over total study period (up to 96 weeks per participant)
Trial sites (3)
Facility
City
Region
Status
Centre hospitalier de l'Université de Montréal (CHUM)
Montreal
Quebec
Recruiting
Jewish General Hospital - CIUSSS-COMTL
Montreal
Quebec
Not Yet Recruiting
Institut Universitaire de Cardiologie et Pneumologie de Québec
Québec
Quebec
Not Yet Recruiting
More Centre hospitalier de l'Université de Montréal (CHUM) trials in Canada
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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