Clinical trials: common concerns answered

A scleroderma medicine will never be given to people in a trial without being tested first³. These tests usually happen in laboratories and animals. Tests continue until the researchers and health authorities have evidence and confidence that it is safe to start testing in humans.There may still be risks – and researchers will explain them clearly⁴ – but designing trials to reduce risk and protect you is always the priority⁵.

• A medicine has usually been tested in laboratories and earlier research stages before it is given to anyone in a trial⁶
• Often the researchers start with very low doses, evaluate the body’s responses and safety, and then step by step slowly increase the dose.
• Clinical trials must be reviewed by strict ethics committees and regulators before they begin⁷
• Participants are closely monitored throughout the study⁸. This will be by the study team and often an independent data monitoring committee.
• In the unlikely case of an emergency, the trial will be stopped⁹ and the teams will be on hand to help you straight away¹⁰

You can leave a clinical trial at any time. Saying yes today does not take away your right to say no later, even if you’ve signed a consent form.

• Someone taking part in a clinical trial can leave without giving a reason at any time¹¹
• You have a legal right to stop taking part in research or change your level of involvement at any time¹². You do not need to explain why. There will be no negative impact on your care.
• Leaving a clinical trial will not affect your ongoing scleroderma treatment¹³

Some people living with scleroderma do decide to join a clinical trial to try a new treatment when the standard treatment doesn’t work¹⁴.

But every clinical trial is unique¹⁵. Each research team is looking for different types of volunteers¹⁶ – for example, people who are newly diagnosed with scleroderma¹⁷, or people in a specific age range¹⁸. There may even be research looking specifically at female or male bodies and how scleroderma affects them differently.¹⁹

Some clinical trials are designed to test new treatments²⁰. Others look at everyday habits, such as diet or lifestyle, and their effect on preventing scleroderma²¹.

Each clinical trial has a clear list of details about who can and can’t join the trial²². For example, the researchers might be looking for people of a certain age, gender or type of scleroderma²³.

These requirements (known as eligibility criteria) can be found in the informed consent form you will get before agreeing to join a trial²⁴. They are in place to make sure the researchers get the most accurate results and that the trial is as safe as possible²⁵.

Children with scleroderma can take part in some clinical trials²⁶.

In fact, clinical trials in children are urgently needed to help researchers understand how the condition affects each child over time, and to develop better treatments and approaches to care²⁷.

• Trials involving children with scleroderma are carefully regulated with extra precautions, and only approved when potential benefits outweigh risks²⁸
• A parent or legal guardian must give consent for a child to take part in a trial²⁹, and get the child involved in the decision whenever possible³⁰

You can find more information for parents of a child with scleroderma here

If you join a clinical trial, you will be treated as a volunteer partner in research, not treated as an experiment.

• Research is carried out “with” participants, not “to” them – and you will be told what’s happening and encouraged to ask questions at every stage of the clinical trial³¹
• You will have a dedicated research team, clear information and ongoing support³²
• Most participants in clinical trials say they feel well cared for and supported³³
• In one survey, 95% of participants either “agreed” or “strongly agreed” that the researchers treated them with respect³⁴

A placebo is an inactive or “dummy” treatment that does not contain an active medical ingredient³⁵.

Although it has no direct effect on your body, a placebo can sometimes lead to improvements in symptoms because of expectations, beliefs or the context in which the treatment is given³⁶.

Not all trials for scleroderma treatments use placebos³⁷. The consent form will explain whether your trial has a placebo group or not.

• If there is a chance you may get a placebo and you don’t want it, you don’t have to join that trial³⁸
• Many trials compare new treatments with standard care, not just a placebo³⁹
• If the trial you’re joining does use a placebo, your research team will clearly explain what’s involved during the consent process⁴⁰
• You don’t get to choose if you get the trial treatment or the placebo, and you may not know if you get the placebo until after the trial⁴¹. The medical and research teams may not know, either.
• If your trial includes a placebo, the placebo will be given alongside your standard medical treatment⁴² Sometimes, in trials with a placebo group, people on placebo are switched to the treatment group at a certain point. The type of study where this happens is often called a cross-over trial, or open-label extension trial.

Clinical trials happen at all stages of treatment⁴³. Researchers are often looking for different types of volunteers, including⁴⁴:

• Early-stage patients
• People who experience specific complications
• People who’ve been living with scleroderma for years
• Healthy volunteers
• Women
• People from a diverse background

Not all research involves drugs⁴⁵. Clinical trials may involve:

• New medicines⁴⁶
• Scans, x-rays or monitoring tools⁴⁷
• Lifestyle or symptom tracking⁴⁸
• Questionnaires⁴⁹

Some studies will involve additional tests, such as blood tests, x-rays or scans to monitor your health and see how the treatment works⁵⁰.

This can mean many studies do need participants to make more hospital visits than they would if they were having their usual treatment⁵¹.

However, depending on the type of research, tests can take place in both clinical and non-clinical settings, such as⁵²:

• Hospitals
• Doctor’s surgeries
• Schools
• Care homes
• Your home

Some clinical trials may tell patients to carry on taking their current treatment.

This is so the researchers can understand if the new treatment gets the best results when someone takes them alongside current treatments⁵³.

Joining a clinical trial might involve expenses such as travel, parking, hotel and/or childcare. Each scleroderma trial is different, but researchers may have a budget to cover these costs⁵⁴.This is heavily regulated by country governments.

Speak with your research team – and your insurance company if you have one – about which costs are covered before you sign up for the trial.

New studies into scleroderma are happening all the time. Researchers can’t carry out this vital work without volunteers⁵⁵.

Scleroderma is uncommon, affecting between 7 and 40 people in 100,000⁵⁶. This means it can be challenging to find people to take part in a trial.

Getting involved in research means your experience can make a meaningful contribution to progress for other people who live with scleroderma – both now and in the future⁵⁷.