Mycophenolate Mofetil in Systemic Sclerosis With Subclinical Interstitial Lung Disease

NCT05785065 | Recruiting Phase 2

• 1 other identifier: MP-02-2023-11180
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Systemic Sclerosis With Lung Involvement; Systemic Sclerosis; Interstitial Lung Disease

Keywords

Systemic sclerosis; Mycophenolate mofetil; Interstitial lung disease

Outcome Measures

Primary Outcomes (13)

• 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)

Secondary Outcomes (1)

• Frequency of treatment-related adverse events

  Over total study period (up to 96 weeks per participant)

Other Outcomes (40)

• Annual rate of decline in percent (%) predicted forced vital capacity (FVC) over 48 weeks

  Over 48 weeks

• Annual rate of decline in percent (%) predicted forced vital capacity (FVC) over 96 weeks

  Over 96 weeks

• Proportion of participants having clinically meaningful progression

  Over total study period (up to 96 weeks per participant)

Study Arms (2)

Mycophenolate mofetil

EXPERIMENTAL

2 to 4 capsules of mycophenolate mofetil twice daily.

Drug: Mycophenolate Mofetil

Placebo

PLACEBO COMPARATOR

2 to 4 capsules of placebo twice daily.

Other: Placebo

Interventions

Mycophenolate Mofetil DRUG

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

Mycophenolate mofetil

Placebo OTHER

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

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Centre hospitalier de l'Université de Montréal (CHUM): lead
• 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

Study Sites (3)

Centre hospitalier de l'Université de Montréal (CHUM)

Montreal, Quebec, H2X 3E4, Canada

RECRUITING

Jewish General Hospital - CIUSSS-COMTL

Montreal, Quebec, H3T 1E2, Canada

NOT YET RECRUITING

Institut Universitaire de Cardiologie et Pneumologie de Québec

Québec, Quebec, Canada

NOT YET RECRUITING

MeSH Terms

Conditions

Scleroderma, Systemic; Lung Diseases, Interstitial

Interventions

Mycophenolic Acid

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Study Officials

• Sabrina Hoa, MD

  Centre hospitalier de l'Université de Montréal (CHUM)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sabrina Hoa, MD MSc

CONTACT

514-890-8000; sabrina.anh-tu.hoa.med@ssss.gouv.qc.ca

Unité d'innovation thérapeutique, CHUM

CONTACT

uit.eligibilite.chum@ssss.gouv.qc.ca

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The participant, the physician, the study investigators and the research personnel will be blinded to treatment allocation, whereas the dispensing pharmacy will not.
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: February 7, 2023
• First Posted: March 27, 2023
• Study Start: August 29, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2028
• Last Updated: April 11, 2025

Record last verified: 2025-04

Locations

CA (3)