Clinical trials – also called clinical studies – are carefully planned research studies that involve people with lived experience. They play a vital role in finding out whether new treatments, tests and approaches to managing scleroderma are safe, effective and helpful.
If you have – or support someone - with you can get involved to help develop better treatments.
Different kinds of trials – and why they matter for scleroderma
Researchers need clinical trials to understand different things about medicines, including:
- Safetywhat are the side effects?
- Clinical activitywhat happens in the body and to the body?
- Dosewhat is the best amount?
- Efficacydoes the treatment or approach work well?
But not all clinical research for scleroderma is about testing new medicines. Different trials focus on things like:
- physiotherapy
- symptom management
- quality of life
- earlier organ damage diagnosis.
A wide range of research approaches is needed to meet the needs of:
- people with different types of scleroderma.
- people at different stages such as diagnosis or remission.
- people with different symptoms and risk of progression.
Types of clinical research
There are many different types of clinical trials, including:
Treatment trials (including Randomised Controlled Trials – RCTs) test how safe and effective medicines, therapies or procedures are to see which one works best. This is the most common type of clinical trial.
Prevention trials look at ways to stop a condition getting worse. They may compare different medicines, therapies or everyday factors such as sleep, nutrition and exercise.
Diagnostic trials test new or improved tools or tests to help make diagnosis more accurate, quicker or easier.
Screening trials focus on finding disease or complications earlier, particularly in people at increased risk – for example, family members of someone living with scleroderma. Earlier detection can help guide monitoring and treatment choices.
Genetic and biomarker studies explore links between genes and biological markers and disease risk type or progression. This research can:
- help predict who may develop scleroderma
- understand why it affects people differently
- biomarkers can also be used in treatment trials
Quality‑of‑life research looks at ways to improve comfort, emotional wellbeing and day-to-day life. They may focus on factors like anxiety, depression, coping strategies or side effects of treatment.
Observational studies and patient registries collect information about how scleroderma affects people over time. This helps researchers understand disease patterns, outcomes and real‑life experiences.
Who funds clinical trials for scleroderma?
In Europe, clinical trials for scleroderma may be funded and organised by a range of organisations, including:
- Pharmaceutical or biotechnology companies
- Universities or academic research centres
- Hospitals or healthcare organisations
- Charities and patient organisations
- Government or public bodies and publicly funded research organisations
Who oversees clinical trials for scleroderma?
In European Union (EU) and European Economic Area (EEA) countries, all clinical trials involving people must go through strict checks by the European Medicines Agency (EMA) before they can begin. This is to protect the rights, safety and interests of the people taking part.
Each application for a clinical trial must follow EU Clinical Trials Regulation. And as part of the approval process, each application is reviewed both by:
- check patient safety
- look at study progress
- recommend whether to continue, change, or stop a trial.
Who runs clinical trials for scleroderma?
Each trial is run by a team of researchers who all have different skills and expertise. The size of the team depends on how complex the study is and how many hospitals or centres are involved.
Teams vary, but often include the following people:
- Principal Investigator (PI)the lead researcher who has overall responsibility
- Study doctorswho assess, treat and monitor participants
- Research nurseswho support and monitor participants
- Study or clinical research coordinatorswho manage the day-to-day running of the trial
- Pharmacistswho prepare, store and manage any medicines for the study
- Lived-experience participantswhose involvement and insights are a vital part of the research
Why are clinical trials organised in phases?
Clinical trials happen in stages, also known as phases. Each stage is designed to answer different questions. This means researchers can build up evidence step by step.
The priority at each stage is to make sure that the people taking part are safe.
At the end of each stage, the researchers review the results and decide whether to move forward to the next stage.
The main phases of a clinical trial
These phases below are a common pattern – but some trials do things differently, depending on what is being studied.
Pre-clinical studies
There are often four phases (I-IV) of a clinical study for a new medicine. Before the phases begin, there is a preclinical testing period, where samples are tested in a laboratory and in animals. These are known as pre-clinical studies.
No people take part yet
Phase I – Safety
Small groups of people (20-100) take part to find out whether a treatment is safe, what happens in the body, and what side effects may occur.
20–100 people. Each figure is about 30 people; solid figures show the smallest group size, outlined figures the largest.
Phase II – Safety and dosing
Larger groups (100-300) help researchers understand the best dose and continue to measure side effects.
100–300 people. Each figure is about 30 people; solid figures show the smallest group size, outlined figures the largest.
Phase III – Safety and effectiveness
Even larger studies (300-3,000) compare the new treatment with existing care or control groups to confirm whether it works and whether the benefits outweigh the risks.
300–3,000 people. Each figure is about 30 people; solid figures show the smallest group size, outlined figures the largest.
Phase IV – Real‑world evidence
Once a treatment is approved and in use, ongoing studies monitor long‑term effects and safety in everyday care settings.
Monitoring continues after approval
During a clinical trial, moving on to the next phase only happens if:
- Earlier results show the treatment is safe enough and shows promise.
- The benefits are considered to outweigh the possible risks or side effects.
Researchers always put safety first
Protecting people who take part in clinical trials is central to how all research is designed and carried out. The trials are carefully reviewed before they begin and are monitored all the way through to make sure the rights, safety and wellbeing of participants come first.
Before a study can go ahead, researchers must clearly show that:
- The potential benefits outweigh the possible risks
- Side effects are anticipated, closely monitored and accurately recorded
- Participants get clear information about what taking part involves
- The people involved are volunteers and can leave the study at any time without giving a reason and without any negative effects on their care
Sources (33)
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“Clinical research and clinical trials are fundamental to developing new medical interventions, ensuring treatments are safe and effective before reaching the market.”
- Clinical trials: National Institutes of Health (NIH) https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics
“When you volunteer to take part in clinical research, you help doctors and researchers learn more about disease and improve health care for people in the future.”
- Clinical Trials and Clinical Research: A Comprehensive Review https://pmc.ncbi.nlm.nih.gov/articles/PMC10023071/
“Clinical trials evaluate the safety and efficacy of a therapeutic drug”
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“Clinical trials assess side effects and how the body reacts to treatment”
- Clinical Trial Phase: Definition, Objectives and Phases https://www.pharmaacademias.com/clinical-trial-phase-definition-objectives-and-phases/
“Clinical trials help determine ‘the optimal dosage’”
- Clinical Trial Phase: Definition, Objectives and Phases https://www.pharmaacademias.com/clinical-trial-phase-definition-objectives-and-phases/
“They provide data on how a drug is absorbed, distributed, metabolised and excreted (what happens in the body)”
- Clinical trials: National Institutes of Health (NIH) https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics
“There are different types of clinical trials: Prevention trials… Screening trials… Diagnostic trials… Treatment trials… Behavioral trials… Quality of life trials…”
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“Experience suggests that African Americans may express autoimmune disease differently than other racial groups.”
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“There are different types of clinical trials: Prevention trials… Screening trials… Diagnostic trials… Treatment trials… Behavioral trials… Quality of life trials…”
- Clinical trials: National Institutes of Health (NIH) https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics
“Treatment trials test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.”
- Clinical trials: National Institutes of Health (NIH) https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics
“Prevention trials look for better ways to prevent a disease in people who have never had the disease or to prevent the disease from returning. Approaches may include medicines, vaccines, or lifestyle changes.”
- Clinical trials: National Institutes of Health (NIH) https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics
“Diagnostic trials study or compare tests or procedures for diagnosing a particular disease or condition.”
- Clinical trials: National Institutes of Health (NIH) https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics
“Screening trials test new ways for detecting diseases or health conditions.”
- Biomarkers: Promising and valuable tools towards diagnosis, prognosis and treatment of Covid‑19 and other diseases https://pmc.ncbi.nlm.nih.gov/articles/PMC9884646/
“Biomarkers… are used… for screening and risk assessment… to monitor the development of a disease… and to identify patients who are likely to respond to therapy.”
- Clinical trials: National Institutes of Health (NIH) https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics
“Quality of life trials (or supportive care trials) explore and measure ways to improve the comfort and quality of life of people with conditions or illnesses.”
- Participate in Observational Studies: National Cancer Institute (NCI) https://www.cancer.gov/research/participate/how/observational-studies
“In observational studies, researchers follow groups of people over a period of time… [and] can help researchers learn more about… patterns and trends… [and] the experiences of people who have had [a disease].”
- Funding – Health Research Authority (HRA) https://www.hra.nhs.uk/planning-and-improving-research/research-planning/funding/
“Funding for research can come a variety of sources, including: commercial funders, including industry and private companies and non-commercial funders such as government departments, research councils, charities, National Institute of Health in USA (NIH) and the European Commission.”
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“In a clinical trial the rights, safety, dignity and well-being of subjects should be protected… A clinical trial shall be subject to scientific and ethical review and shall be authorized.”
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“European Union (EU) pharmaceutical legislation known as the Clinical Trials Regulation entered into application on 31 January 2022. It aims to ensure the EU offers an attractive and favourable environment for carrying out clinical research on a large scale, with high standards of public transparency and safety for clinical trial participants.”
- Clinical Trials Regulation: European Medicines Agency (EMA) https://www.ema.europa.eu/en/human-regulatory-overview/research-development/clinical-trials-human-medicines/clinical-trials-regulation
“Each clinical trial application is reviewed by national competent authorities in every country taking part, alongside ethics committees, as part of the regulatory approval process.”
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“In accordance with the Clinical Trials Regulation (EU) 536/2014 (CTR) Article 4, ‘A clinical trial shall be subject to scientific and ethical review and shall be authorised in accordance with this Regulation’ and that ‘The ethical review shall be performed by an ethics committee in accordance with the law of the Member State concerned.’”
- Clinical Research Team: Johns Hopkins Medicine https://www.hopkinsmedicine.org/research/understanding-clinical-trials/clinical-research-team
“Conducting clinical research requires a team of people with a variety of skills and experience… research team members vary by study site, type, design, and purpose.”
- Clinical Research Team: Johns Hopkins Medicine https://www.hopkinsmedicine.org/research/understanding-clinical-trials/clinical-research-team
“Research team members vary by study site, type, design, and purpose, but may include: Principal investigator (PI), Study physicians, Research nurse, Study coordinator, Research pharmacists, Participants.”
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“All investigational medicinal products (IMPs) go through a series of trials before they can be licensed for use. There are usually 4 phases of trials in humans (clinical trials).”
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““Before testing a drug in people… preclinical laboratory studies” are carried out to assess safety and other effects.”
- Clinical trials: World Health Organization (WHO) https://www.who.int/health-topics/clinical-trials/#tab=tab_1
“Phase I studies usually test new drugs for the first time in a small group of people to evaluate a safe dosage range and identify side effects.”
- Clinical trials: World Health Organization (WHO) https://www.who.int/health-topics/clinical-trials/#tab=tab_1
“Phase II studies test treatments that have been found to be safe in phase I but now need a larger group of human subjects to monitor for any adverse effects.”
- Clinical trials: World Health Organization (WHO) https://www.who.int/health-topics/clinical-trials/#tab=tab_1
“Phase III studies are conducted on larger populations and in different regions and countries, and are often the step right before a new treatment is approved.”
- Clinical trials: World Health Organization (WHO) https://www.who.int/health-topics/clinical-trials/#tab=tab_1
“Phase IV studies take place after country approval and there is a need for further testing in a wide population over a longer timeframe.”
- National Cancer Institute https://www.training.seer.cancer.gov/treatment/clinical-trials/
“Testing and evaluation is accomplished via sequential steps called phases. Each phase has a purpose, and one phase must be completed before advancing to the next.”
- ICH Guideline for Good Clinical Practice – Monitoring: ICH GCP https://ichgcp.net/monitoring
“The purposes of trial monitoring are to verify that: (a) The rights and well-being of human subjects are protected.”