Mycophenolate Mofetil in Systemic Sclerosis With Subclinical Interstitial Lung Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorCentre hospitalier de l'Université de Montréal (CHUM)
About 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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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;
Disqualifiers
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;
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
Trial design
Design model
Parallel
Treatments tested in this trial
Mycophenolate Mofetil
DrugThe 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
Other interventionThe 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
Treatment groups
35 Participants
are divided into 2 treatment groupsGroup A: Mycophenolate mofetilExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Total number of potentially eligible patients identified per site
Time frame
Over one year2
Proportion of potentially eligible patients who provide consent per site
Time frame
Over one year3
Proportion of consented participants who meet the eligibility criteria per site
Time frame
Over one year4
Monthly rate of randomized participants per site
Time frame
Over one yearSecondary 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)
Time frame
Over total study period (up to 96 weeks per participant)Other outcomes
1
Annual rate of decline in percent (%) predicted forced vital capacity (FVC) over 48 weeks
Time frame
Over 48 weeks2
Annual rate of decline in percent (%) predicted forced vital capacity (FVC) over 96 weeks
Time frame
Over 96 weeks3
Proportion of participants having clinically meaningful progression
Clinically meaningful progression defined by the Outcome Measures in Rheumatology (OMERACT) for connective tissue disease-associated interstitial lung diseases (CTD-ILD) (defined as a ≥10% relative decline in FVC from baseline, or a ≥5% to \<10% relative decline in FVC associated with a ≥15% relative decline in diffusion capacity \[DLCO\])
Time frame
Over total study period (up to 96 weeks per participant)4
Time to clinically meaningful progression
Clinically meaningful progression as defined by Outcome Measures in Rheumatology (OMERACT) for connective tissue disease- associated interstitial lung diseases (CTD-ILD) (defined as a ≥10% relative decline in FVC from baseline, or a ≥5% to \<10% relative decline in FVC associated with a ≥15% relative decline in diffusion capacity \[DLCO\])
Time frame
Over total study period (up to 96 weeks per participant)Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Centre hospitalier de l'Université de Montréal (CHUM)
Lead sponsor
McGill University
Collaborator
University of Calgary
Collaborator
Jewish General Hospital
Collaborator
Canadian Institutes of Health Research (CIHR)
Collaborator
Sclérodermie Québec
Collaborator
Institut universitaire de cardiologie et de pneumologie de Québec, University Laval
Collaborator
Source ClinicalTrials.gov