A Phase 2 interventional study of Mycophenolate Mofetil 500mg in Systemic Sclerosis and Limited Cutaneous Systemic Sclerosis, sponsored by University College, London. Active, not recruiting at 12 sites in United Kingdom. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-07-27.
Sponsored by University College, London · Phase 2, Interventional, and Treatment
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.
The MINIMISE-Pilot trial aims to explore whether the immunosuppressive agent mycophenolate mofetil (MMF) at a target dose of 2g daily can slow down disease progression in patients with limited cutaneous systemic sclerosis (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.
This is an open label randomised prospective trial that will recruit 120 participants aged 18 and older with limited cutaneous systemic sclerosis across 13 sites in the UK. Following a screening visit, eligible participants will attend a baseline visit where they will be randomly allocated into one of two groups; MMF or Control. Those in the first group are given mycophenolate mofetil (MMF) taken daily by mouth for up to 96 weeks, in addition to their background Standard of Care medication for SSc related symptoms. Those in the second group will not receive any MMF but will remain on their standard of care medication alone.
Participants are expected to be followed up for a minimum of 48 weeks or a maximum of 96 weeks. The trial will involve five (5) clinic visits which are expected to be carried out at the same time of the participants' normal hospital appointment with their scleroderma specialist. Participants from both groups will have the same assessments. Participants are expected to return to the clinics at Week 24, 48, 72 and 96. However, participants allocated to the MMF group will have additional blood samples taken for safety monitoring every 2 weeks for the first 8 weeks, then every 4 weeks for the following 12 weeks. Thereafter, every 12 weeks up to their final visit.
All the participants will receive four (4) routine telephone calls in between their clinic visits.
688 studies on the registry are indexed under Scleroderma, Systemic; 223 are open to participants now.
This study's planned enrollment of 120 is above the median of 34 across 493 interventional studies indexed under Scleroderma, Systemic.
Browse Scleroderma, Systemic studies →University College, London is the lead sponsor of 632 studies on the registry; 145 are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 2 (33%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Active chronic infection such as COVID-19, tuberculosis, pneumocystis, cytomegalovirus, herpes simplex virus, herpes zoster and atypical mycobacteria.
Suitability for enrolment once the participant has recovered from infection will be based on Investigator judgment.
Infection history:
i. Hospitalisation for treatment of infection within ≤ 8 weeks of screening visit date
ii. Use of parenteral (IV or IM) antibiotics (antibacterials, antivirals, anti-fungals, or anti-parasitic agents) within ≤ 4 weeks of screening visit date
Any of the following laboratory results at screening visit:
Participants will receive mycophenolate mofetil (MMF) for up to 96 weeks, in addition to their background Standard of Care medication for systemic sclerosis related symptoms. They will receive 500mg twice daily over the first 4 weeks following their randomisation, and if tolerated the dose will be increased to a target dose of 1g twice daily starting from week 5 until their Final visit.
Drug: Mycophenolate Mofetil 500mg
Standard of Care (no immunosuppression) for systemic sclerosis related symptoms.
Mycophenolate Mofetil oral tablet twice daily for up to 96 weeks
The total number of lcSSc patients screened during the 12 month recruitment period, measured from information provided on the site Screening Logs
The total number of lcSSc patients screened will be captured on detailed screening logs
Time frame: 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
Participant informed consent will be captured in detailed screening logs
Time frame: 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
Information on participants who meet the eligibility criteria will be captured on detailed screening logs
Time frame: 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.
Participant self-reported adherence using participant dosing diaries to be completed daily. Diaries will be provided to all participants randomised to MMF to record their daily trial medication intake, in addition to any dose modifications and dose interruptions for the duration of their time in the study.
Time frame: 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
Pharmacy dispensing accountability logs will record the number of pills dispensed at each visit and the number of pills returned by the participant.(i.e. pill count) The number of pills taken is calculated by subtracting the count of the number of pills remaining from the total number of pills dispensed. The drug adherence rate is then calculated by dividing the number of pills taken by number of days elapsed since the last dispense.
Time frame: 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
Sites to report deviations and sponsor protocol compliance reviews during central, remote and on-site monitoring to be reported in Protocol Deviation Reports.
Time frame: 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
The number of participants who withdraw from trial treatment ONLY will be recorded using detailed Participant Withdrawal Logs
Time frame: 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
Self-reported: Participant Dosing Diaries will be provided to all participants randomised to MMF to record dose escalation information and daily intake of the IMP .
Time frame: 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.
Validated physical examination method for estimating skin induration. It is scored on a 0 (normal) to 3+ (severe induration) ordinal scales over 17 body areas, with a maximum score of 51 and is used to categorise severity of SSc. Minimally clinically significant difference in mRSS is 3-5 points. The measurement of time to clinically important disease progression will demonstrate a clinically important advantage of active treatment compared with no immunosuppression if a beneficial effect is found.
Time frame: 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
The number of participant loss to follow -up in each group (MMF or control) will be recorded using detailed Participant Withdrawal Logs
Time frame: From Baseline, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 from Baseline for a minimum of 48 weeks or a maximum of 96 weeks
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 from Baseline for a minimum of 48 weeks or a maximum of 96 weeks
Time frame: From Baseline, through study completion for a minimum of 48 weeks or a maximum of 96 weeks
Number of reported deaths Baseline
Participants self report, review of hospital records
Time frame: From Baseline , through study completion, up to 36 months
Changes in functional ability from Baseline for a minimum of 48 weeks or a maximum of 96 weeks as measured by the Scleroderma Assessment Questionnaire
The Scleroderma Assessment Questionnaire is a self-assessed measure ranging from 0-3 (where 0 = without difficulty and 3 = unable to do) for several questions including vascular, respiratory, gastrointestinal, musculoskeletal, and overall disease status with 23 questions divided into 4 groups.
Time frame: From Baseline, every 6 months, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
Quality of Life as measured by the EQ5-5D-5L Questionnaire from Baseline for a minimum of 48 weeks or a maximum of 96 weeks
EQ-5D- 5L is a participant self -reported questionnaire which evaluates the generic quality of life at the time of completion. It comprises of one question for each of the five dimensions that include mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Respondents are asked to choose the statement in each dimension that best describes their health status. Their responses are coded as a number (1, 2, or 3) that corresponds to the respective level of severity: 1 indicates no problems, 2 some problems, and 3 extreme problems
Time frame: From Baseline, every 6 months, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
Changes in pain and disability from Baseline for a minimum of 48 weeks or a maximum of 96 weeks as measured by the Patient Global Questionnaire.
Self-reported questionnaire assessed by a single question to reflect the participants perspective on their health overall. Rating scale used with a 0-100 response, where 0= has no effect at all, 100= worst possible effect.
Time frame: From Baseline, every 6 months, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
Changes in pain and disability from Baseline for a minimum of 48 weeks or a maximum of 96 weeks as measured by the Physician's Global Questionnaire.
Physician-reported questionnaire assessed by a single question to reflect the participants perspective on their health overall. Rating scale used with a 0-100 response, where 0= has no effect at all, 100= worst possible effect.
Time frame: From Baseline, every 6 months, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks]
Scleroderma skin activity and skin-related health related quality of life from Baseline for a minimum of 48 weeks or a maximum of 96 weeks as measured by PASTUL-SSPRO score
Self-reported questionnaire specifies a grading of skin (normal (0), mild (1), moderate (2), severely (3) thickened) at eight sites corresponding to mRSS with maximum score assigned to each site (PASTUL), and that assesses health-related quality of life (HRQOL) related to skin involvement in SSc. It has 18 items representing 4 HRQOL scales: physical effects, emotional effects, physical function, and social effects. All items are scored from 0 (better) to 6 (worse) (SSPRO).
Time frame: From Baseline, every 6 months, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
Subgroup analyses of antinuclear antibody, ACA+ versus ACA-
Standard diagnostic laboratory assays for antinuclear antibodies
Time frame: over 36 months
IcSSc disease duration at baseline
IcSSc disease duration at baseline up to 4 years versus four years or more
Time frame: Baseline
Plan to share: Undecided — No plan to share IPD has been made at this time
This study is active, not recruiting, as verified in Jul 2023. You cannot join it, but the record below documents what was studied.
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CREST Syndrome
University College, London