Systemic sclerosis or scleroderma is an autoimmune condition that cause thickening and hardening of the skin, but can also affect internal organs. There are two major subsets of scleroderma: the limited cutaneous systemic sclerosis (lcSSc) that usually affects the skin of the face, neck, lower legs or lower arms, but can also lead to internal organ complications, and the diffuse cutaneous systemic sclerosis (dcSSc) that may affect blood circulation and internal organs, as well as the skin. To date there is no drug that has been definitively proven to cure or modify the course of scleroderma. However, there is emerging evidence that immunosuppression and specifically mycophenolate mofetil (MMF) may be beneficial in lcSSc. The MINIMISE-Pilot trial would be an important first step to evaluate the risk and potential benefit to this disease group. MMF as the intervention of choice is both appropriate and timely, as it has been routinely used in the management of dcSSc. The aim of this pilot trial is to explore whether the immunosuppressive agent MMF can slow down disease progression in patients with lcSSc compared to the current standard of care alone. This pilot trial will also provide critical information for the development of a future large trial that could potentially transform lcSSc patient management.
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The total number of lcSSc patients screened during the 12 month recruitment period, measured from information provided on the site Screening Logs
Timeframe: From first site activation up to a period of 12 months
The proportion of participants who provide consent during the 12 month recruitment period, measured from information provided on the site Screening Logs
Timeframe: From first site activation up to a period of 12 months
The proportion of participants who meet the eligibility criteria during the 12 month recruitment period, measured from information provided on the site Screening Logs
Timeframe: From first site activation up to a period of 12 months
Adherence to trial treatment by participants randomised to the MMF arm as assessed by the Participant Dosing Diaries.
Timeframe: From Baseline, up until a minimum of 48 weeks or a maximum of 96 weeks
Drug adherence rate from participants randomised to the MMF arm, measured by Pharmacy Accountability Logs
Timeframe: From first participant dispensing, through study completion up to 36 months
Adherence to the study protocol by participating sites as assessed by the number of protocol deviations reported using the Protocol Deviation Reports
Timeframe: From Screening, through study completion up to 36 months
The proportion of participants intolerant to MMF who discontinue trial treatment from Baseline for a minimum of 48 weeks or a maximum of 96 weeks as assessed by the Participant withdrawal Logs
Timeframe: From Baseline, up until a minimum of 48 weeks or a maximum of 96 weeks
The total number of participants randomised to MMF who reach the target dose of 2g a day, measured by medication intake information captured in the Participant Dosing Diaries
Timeframe: From Baseline through to study completion for a minimum of 48 weeks or a maximum of 96 weeks
The total number of participants randomised to MMF and Control who reach a clinical worsening of disease progression, measured by the modified Rodnan Skin Score (mRSS) from Baseline, every 6 months until Final trial visit.
Timeframe: From Baseline, every 6 months, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
The number of participant loss to follow- up as assessed by the Participant Withdrawal Logs
Timeframe: From Baseline, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks