The MINIMISE-Pilot study: seeking to minimise disease progression in limited scleroderma

Background

Limited systemic sclerosis or limited scleroderma (also known as limited cutaneous systemic sclerosis) usually affects the skin of the face, neck, lower legs, or lower arms causing thickening and hardening in these areas and can lead to complications involving internal organs. It is estimated that around 70% of people with systemic sclerosis have limited systemic sclerosis, with the remainder having the diffuse systemic sclerosis (dcSSc). Despite the higher prevalence of lcSSc there has and continues to be less clinical trial activity in this area. 

At present there are no drugs which can cure or modify the course of scleroderma. The immunosuppressive drug mycophenolate mofetil (MMF) is routinely used to manage diffuse systemic sclerosis and scleroderma-associated interstitial lung disease, but emerging evidence suggests it could also be beneficial in managing the limited form of systemic sclerosis.

The MINIMISE-Pilot Trial

Clinicians wish to establish whether a 2g daily dose of MMF, taken orally, is of benefit to people with early limited systemic sclerosis (less than 7 years post-diagnosis) by slowing down the progression of their condition. Before a clinical trial in a large group of patients can be carried out, clinicians must first establish the benefits and potential risks of MMF to patients with lcSSc, the feasibility of conducting a large trial, and determine how it can be best delivered. The MINIMISE-Pilot (Mycophenolate in limited cutaneous systemic sclerosis) study will address these key issues and provide information to assist in the planning of a larger trial of this treatment.

The MINIMISE-Pilot trial is funded by Versus Arthritis. SRUK as a patient organisation is supporting the trial and is proud to be involved in this important first step which could change the way in which patients with lcSSc are managed.

I am interested in participating and want to know more about the trial

The MINIMISE-Pilot trial is a randomised prospective open label trial aiming to recruit 120 participants. This means those who are recruited to the study will be randomly assigned to one of two groups: participants in the CONTROL group will continue to take their current treatments used to manage their condition; participants in the MMF group will receive MMF in addition to the treatments they currently take to manage their condition. Both clinicians and participants will know which treatment group they belong to, making it an open label trial.

Those interested in participating will be invited to attend a screening visit to assess their eligibility for the study. Following this, eligible participants will be invited to attend a ‘baseline’ visit where they will be allocated at random to either the control or MMF group. Participants will be followed for 48-96 weeks. There will be four further clinic visits where participants will be expected to attend the clinical trial site or be seen remotely (via video link or over the telephone), these will take place at Week 24, 48, 72 and 96 – these may be timed to coincide with the participant’s usual hospital appointments with their scleroderma specialist. Participants in the MMF group will also require additional blood samples to be taken as part of safety monitoring. These can be done at the hospital or at the participant’s GP.

Those in the MMF group will be expected to take MMF orally for up to 96 weeks. Those in the control group will not receive any further medication beyond what they are prescribed through their routine clinical care. However, participants in both groups will benefit from the same clinical assessments as part of their trial clinic visits and will receive four routine telephone calls in between their clinic visits as part of remote patient monitoring.   

I am interested - am I eligible to join?

Those meeting the following criteria may be eligible for the study:

• Adults i.e. those 18 years of age or over
• Confirmed diagnosis of limited cutaneous systemic sclerosis with less than 7 years from the first non-Raynaud’s symptom of SSc.
• Must be UK based/ registered with a GP in the UK
• Must be willing to comply with the trial protocol and attend clinical visits
• Must not be currently enrolled in another clinical study investigating a treatment for SSc
• Must not be pregnant
• Female participants of childbearing age (or male participants with partners of childbearing age) must be willing to use a suitable form of contraception for the duration of the study.

More in depth information on the eligibility criteria for this trial can be found on University College London’s Comprehensive Clinical Trials Unit web page.

It sounds like I’m eligible - how can I join this clinical trial?

The Royal Free Hospital London is the lead clinical site for the MINIMSE-Pilot Trial. The trial is also being conducted at a further 13 hospitals in the UK as indicated below. More information on the lead clinicians at each can be found on University College London’s Comprehensive Clinical Trials Unit web page.

• Royal United Hospital, Bath
• Ninewells Hospital, Dundee 
• Aintree University Hospital, Liverpool 
• Royal Free Hospital, London 
• Salford Royal Hospital, Manchester 
• Freeman Hospital, Newcastle 
• Royal Hallamshire Hospital, Sheffield 
• New Cross Hospital, Wolverhampton 
• Royal Infirmary Manchester 
• Darlington Memorial Hospital 
• Queen Elizabeth Hospital, Birmingham 
• Southmead Hospital, Bristol 
• Addenbrookes Hospital, Cambridge 
• Chapel Allerton Hospital, Leeds

If you are interested in participating, you should contact the clinical leads at the relevant site. If you do not currently receive treatment at one of these hospitals it may be possible for you to be referred to another hospital to allow you to participate in the trial. Please speak to your GP or consultant about referral to the trial.