Systemic SclerosisPhase 2MINIMISENCT04927390
Mycophenolate in Limited Cutaneous Systemic Sclerosis (MINIMISE-Pilot)
A Phase 2 study testing Mycophenolate Mofetil 500mg in people with Systemic Sclerosis and Limited Cutaneous Systemic Sclerosis, run by University College, London at 12 sites in United Kingdom.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Sclerosis, Limited Cutaneous Systemic Sclerosis
- What is being tested?
- Mycophenolate Mofetil 500mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 12 sitesUnited Kingdom
- Study dates
- 8 Dec 2021to 30 Apr 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants with lcSSc classified by the 2013 EULAR ACR criteria for limited cutaneous subset of SSc
- Participants with less than 7 years disease duration from first non-Raynaud's manifestation of SSc
- Participants aged 18 years or more (≥ 18 years) at screening visit
- If women of child bearing potential, the participant must have a negative pregnancy test at screening and baseline visits
- Negative viral screen for HIV, Hepatitis B and C
- Ability to provide full informed consent
- Registered with a GP practice in the UK
- Participants must be willing to attend for follow up visits (at site or remotely) and to comply with study-related procedures -
Who cannot join
- Having already developed a complication of SSc that requires initiation of MMF or an alternative major immunosuppressive drug for SSc such as methotrexate, cyclophosphamide or azathioprine
- Treatment with methotrexate, cyclosporine A, azathioprine, mycophenolate mofetil (MMF), rapamycin, colchicine, D-penicillamine, within ≤ 4 weeks prior to the baseline visit date
- Contraindication to MMF (e.g. active infection that would preclude MMF in judgement of investigator), or previous intolerance of MMF
- Any clinical condition which the investigator considers would make the patient unsuitable for the trial
- Pregnancy (or planned pregnancy during trial participation) and/or breastfeeding
- Women of child bearing potential and male participants with a partner of child bearing potential not willing to use adequate contraception as described in section 6.3.1.4 for the duration of trial treatment and within the time points specified following last trial treatment.
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- 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:
- Hospitalisation for treatment of infection within ≤ 8 weeks of screening visit date
- Use of parenteral (IV or IM) antibiotics (antibacterials, antivirals, anti-fungals, or anti-parasitic agents) within ≤ 4 weeks of screening visit date
- Receipt of a live-attenuated vaccine within ≤ 12 weeks of screening visit date
- Participants enrolled in any other interventional trial within ≤ 4 weeks of the screening visit date (co-enrolment in observational studies is acceptable)
- Current drug or alcohol abuse or dependence, or a history of drug or alcohol abuse or dependence within ≤ 52 weeks prior to screening visit date.
- Any of the following laboratory results at screening visit:
- Glomerular filtration rate (GFR) <60 ml/min/1.73m²
- Absolute neutrophil count (ANC) < 1.6 x 10^9/l
- ALT or AST > 2 x ULN
- Participants not willing or unable to attend on-site screening visit.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United Kingdom12 sites
Royal United Hospitals Bath Nhs Foundation Trust
Bath, BA1 3NG
Southmead Hospital - NORTH BRISTOL NHS TRUST
Bristol, BS10 5NB
Darlington Memorial Hospital - County Durham and Darlington NHS Foundation Trust
Darlington, DL3 6HX
Ninewells Hospital - NHS Tayside
Dundee, DD1 9SY
Chapel Allerton Hospital - LEEDS TEACHING HOSPITALS NHS TRUST
Leeds, LS9 7TF
Aintree University Hospital NHS Foundation Trust
Liverpool, L9 7AL
Royal Free Hospital - Royal Free NHS Foundation Trust
London, NW3 2QG
Manchester Royal Infirmary - Manchester University NHS Foundation Trust
Manchester, M13 9WL
Salford Hospital - Northern Care Alliance NHS Foundation Trust
Manchester, M6 8HD
Freeman Hospital - THE NEWCASTLE UPON TYNE HOSPITALS NHS FOUNDATION TRUST
Newcastle, NE7 7DN
Royal Hallamshire Hospital - SHEFFIELD TEACHING HOSPITALS NHS FOUNDATION TRUST
Sheffield, S5 7AU
The Royal Wolverhampton Nhs Trust
Wolverhampton, WV10 0QP
About this study
Official title: A Randomised Prospective Open Label Pilot Trial Comparing Mycophenolate Mofetil (MMF) With no Immunosuppression in Adults With Limited Cutaneous Systemic Sclerosis
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.
Read the detailed description
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.
- Being tested
- Mycophenolate Mofetil 500mg
- Keywords
- Limited Cutaneous Systemic Sclerosis, Mycophenolate Mofetil
- Medical subject headings
- Scleroderma, Systemic, Scleroderma, Limited
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mycophenolate Mofetil 500mgDrug
Mycophenolate Mofetil oral tablet twice daily for up to 96 weeks
| Study group | Role | What they receive |
|---|---|---|
| Mycophenolate Mofetil (MMF) Arm | Experimental | 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. Receives: Drug: Mycophenolate Mofetil 500mg |
| Control Arm | No intervention | Standard of Care (no immunosuppression) for systemic sclerosis related symptoms. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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 .
Measured: 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.
Measured: 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
Measured: From Baseline, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
Secondary outcomes (7)
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
Measured: 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
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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).
Measured: From Baseline, every 6 months, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2021
- Study start8 Dec 2021
- Primary completion30 Nov 2023 (estimated)
- Study completion30 Apr 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- More trials run by University College, LondonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Eligible participants will be randomised 1:1 to receive Mycophenolate Mofetil plus Standard of Care (MMF Group) or Standard of Care alone (Control Group).
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University College, London
- Sponsor type
- University, hospital or other
- Collaborators
- Versus Arthritis
- Responsible party
- Sponsor
- Investigators
- Christopher Denton, Principal investigator, University College, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04927390
- Sponsor's protocol ID
- CTU/2017/306
- Other IDs
- 2019-004139-21, 2019
- Acronym
- MINIMISE
- Status last verified
- Jul 2023
- First submitted
- 19 May 2021
- Last updated
- 27 Jul 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: No plan to share IPD has been made at this time
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomised Prospective Open Label Pilot Trial Comparing Mycophenolate Mofetil (MMF) With no Immunosuppression in Adults With Limited Cutaneous Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT04927390
- Acronym / sponsor ID
- MINIMISE / CTU/2017/306
- Keywords
- Limited Cutaneous Systemic Sclerosis, Mycophenolate Mofetil
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- University College, LondonUniversity, hospital or other
- Collaborators
- Versus Arthritis
- Study start
- 8 Dec 2021 (actual)
- Primary completion
- 30 Nov 2023 (estimated)
- Study completion
- 30 Apr 2024 (estimated)
- First posted
- 16 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Eligible participants will be randomised 1:1 to receive Mycophenolate Mofetil plus Standard of Care (MMF Group) or Standard of Care alone (Control Group).
| Arm | Type | Receives |
|---|---|---|
| Mycophenolate Mofetil (MMF) Arm | Experimental | Drug: Mycophenolate Mofetil 500mgParticipants 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. |
| Control Arm | No intervention | –Standard of Care (no immunosuppression) for systemic sclerosis related symptoms. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Participants with lcSSc classified by the 2013 EULAR ACR criteria for limited cutaneous subset of SSc
- Participants with less than 7 years disease duration from first non-Raynaud's manifestation of SSc
- Participants aged 18 years or more (≥ 18 years) at screening visit
- If women of child bearing potential, the participant must have a negative pregnancy test at screening and baseline visits
- Negative viral screen for HIV, Hepatitis B and C
Key exclusion criteria (19 listed)
- Having already developed a complication of SSc that requires initiation of MMF or an alternative major immunosuppressive drug for SSc such as methotrexate, cyclophosphamide or azathioprine
- Treatment with methotrexate, cyclosporine A, azathioprine, mycophenolate mofetil (MMF), rapamycin, colchicine, D-penicillamine, within ≤ 4 weeks prior to the baseline visit date
- Contraindication to MMF (e.g. active infection that would preclude MMF in judgement of investigator), or previous intolerance of MMF
- Any clinical condition which the investigator considers would make the patient unsuitable for the trial
- Pregnancy (or planned pregnancy during trial participation) and/or breastfeeding
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mycophenolate Mofetil 500mgMycophenolate Mofetil oral tablet twice daily for up to 96 weeks | Drug | Mycophenolate Mofetil (MMF) Arm |
Primary outcome
The total number of lcSSc patients screened during the 12 month recruitment period, measured from information provided on the site Screening Logs
Time frame: From first site activation up to a period of 12 months
The total number of lcSSc patients screened will be captured on detailed screening logs
The proportion of participants who provide consent during the 12 month recruitment period, measured from information provided on the site Screening Logs
Time frame: From first site activation up to a period of 12 months
Participant informed consent will be captured in detailed screening logs
The proportion of participants who meet the eligibility criteria during the 12 month recruitment period, measured from information provided on the site Screening Logs
Time frame: From first site activation up to a period of 12 months
Information on participants who meet the eligibility criteria will be captured on detailed screening logs
Adherence to trial treatment by participants randomised to the MMF arm as assessed by the Participant Dosing Diaries.
Time frame: From Baseline, up until a minimum of 48 weeks or a maximum of 96 weeks
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.
Drug adherence rate from participants randomised to the MMF arm, measured by Pharmacy Accountability Logs
Time frame: From first participant dispensing, through study completion up to 36 months
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.
Adherence to the study protocol by participating sites as assessed by the number of protocol deviations reported using the Protocol Deviation Reports
Time frame: From Screening, through study completion up to 36 months
Sites to report deviations and sponsor protocol compliance reviews during central, remote and on-site monitoring to be reported in Protocol Deviation Reports.
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
Time frame: From Baseline, up until a minimum of 48 weeks or a maximum of 96 weeks
The number of participants who withdraw from trial treatment ONLY will be recorded using detailed Participant Withdrawal Logs
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
Time frame: From Baseline through to study completion for a minimum of 48 weeks or a maximum of 96 weeks
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 .
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.
Time frame: From Baseline, every 6 months, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
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.
The number of participant loss to follow- up as assessed by the Participant Withdrawal Logs
Time frame: From Baseline, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
The number of participant loss to follow -up in each group (MMF or control) will be recorded using detailed Participant Withdrawal Logs
7 secondary outcomes
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
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
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
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.
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.
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
Time frame: From Baseline, every 6 months, through study completion up until a minimum of 48 weeks or a maximum of 96 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2023.
- Sponsor website link to trialLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. No plan to share IPD has been made at this time
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christopher Denton principal investigator
University College, London
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mycophenolate Mofetil 500mg in people with Systemic Sclerosis and Limited Cutaneous Systemic Sclerosis, run by University College, London at 12 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 27 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The total number of lcSSc patients screened during the 12 month recruitment period, measured from information provided on the site Screening Logs (time frame: From first site activation up to a period of 12 months). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2023.