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Why Clinical Trials for PSC?

Your guide to taking part

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Am I eligible (suitable) to take part in a clinical trial?

  • Will my doctor contact me about clinical trials?
  • My hospital is not running the clinical trial I want to take part in. What should I do?
  • Why do I need to sign a consent form?
  • What questions should I ask?
  • What is a study visit?
  • Can I choose the days I attend?

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What is a clinical trial?

Clinical trials are a form of research, which test how well a new treatment works in people who have a particular disease. They are absolutely necessary to see whether medicines are safe and effective, and before new treatments can be made widely available.

Why are clinical trials important for PSC?

Why consider taking part in a clinical trial?

Why isn't there an effective treatment for PSC yet?

How are medicines developed?

Clinical trial 'phases'

Why are clinical trials important for PSC?

At the moment, PSC is incurable; there are no medicines that slow or stop the disease getting worse, or which reduce the need for liver transplantation. There is also limited evidence for treatments that are used to help with symptoms. As doctors and scientists learn more about PSC, they develop ideas about how to alter the way it progresses in individual patients. This can be with new medicines, or testing whether a particular medicine used for another disease can work in PSC. However, doctors cannot start giving out such medicines without evidence that they work in PSC.

As a result, each new treatment must go through the clinical trial process to prove that it does indeed help people with PSC and does not pose unnecessary risks. Only after the process is complete can the new treatments be approved by health regulators and medicine licensing agencies.


The ONLY way to find a treatment, cure, or achieve better control of symptoms for PSC is through well-designed clinical trials.

Why consider taking part in a clinical trial?

It’s a big decision, and there are many things to consider. Whilst no trial can guarantee that the treatment will work, by taking part you are doing something more for your PSC than simply ‘watching and waiting.’ As there is no treatment currently available for PSC, by taking part you get the chance to receive a new medicine that may potentially help, and also have the possibility of ongoing access to treatment once the clinical trial finishes. Secondly, you might help discover a treatment for the broader group of people living with PSC if the clinical trial is successful.

As with any medical test, treatment, or procedure, all clinical trials have their own risks, which the study contact or investigator will discuss with you in depth. Importantly, clinical trials are very closely scrutinised, so you will receive extra care, more regular monitoring and surveillance from the research team during the course of (and for a period of time after completion of) the study.

Your participation allows researchers to understand more about PSC, and that may lead to new ways to help patients, even if the treatments being tested do not prove to be effective. The information and knowledge generated in clinical trials is priceless.

"PSC already takes so much away from us. Usually we are having to react to changing symptoms or worsening of the condition, but by signing up for a clinical trial, we are taking back some of that control." Gareth, clinical trial participant.

Claire has taken part in a number of different clinical trials. Find out what she says about taking part.

Why isn’t there an effective treatment for PSC yet?

Getting a successful result from a clinical trial depends on several factors:


Disease progression in PSC

The rates at which PSC progresses (i.e. the time from diagnosis until someone may need a liver transplant or develop other complications of the disease) varies immensely from one person to the next. This can be as short as a few months in some people, to several decades in others.


This makes it difficult for researchers to see if a treatment is having any real benefit on disease progression in a clinical trial, and so doctors and researchers rely on a combination of biomarkers to monitor how the disease is evolving (and therefore how a medicine is affecting this). A biomarker is simply something that can be measured (such as a blood test or scan) to help predict how a person’s disease is likely to evolve.


One of the ways PSC Support drives research is by funding research to help discover important biomarkers in PSC, and to learn which combination of scans, blood tests, symptoms and biopsies are the most accurate for understanding how the disease is evolving in a person.



Clinical trials cost millions of pounds, and a pharmaceutical company must be interested in PSC and have confidence that their treatment is safe and will work before going ahead with a trial.


Enough patients willing to take part

To prove a drug is safe and works, it must be tested on many patients. Testing it on only a few would not build up enough evidence of its safety and effectiveness for it to be approved for use for everyone. Because PSC is a very rare disease, there is a real risk that there won’t be enough participants, and therefore, a concern that clinical trials cannot get off the ground. If clinical trials cannot happen for this reason, then there will never be a treatment for PSC.


Rules and regulations

Each country (or region) has a regulatory body or authority responsible for the safety, quality and effectiveness of medicines and how they are developed. The regulatory authority in the UK is called the Medicines and Healthcare products Regulatory Agency (MHRA). Pharmaceutical companies developing new drugs must meet strict regulatory safety and efficacy standards set by the MHRA before they are allowed to start the trial in the UK.

How are medicines developed?

As PSC and its underlying processes become better understood, researchers and scientists identify targets for potential new treatments and start to look for molecules or compounds that act on these targets and interfere with an aspect of the underlying disease or help with the symptoms.


Sometimes, researchers take existing drugs already approved for human use and identify new conditions that they might be able to treat. This is called drug repurposing. In order to understand their effectiveness in people with the new condition, they are tested in clinical trials.


Pre-clinical trials

The researchers then look at whether these compounds really do have an effect and if they are safe using cells, animal models and computer modelling. Pre-clinical testing sometimes includes extensive safety evaluation of the new drug in animals called toxicology. These studies are often required by the regulatory authorities and can take years to complete before a trial drug can be tested in humans.


Clinical trials (humans)

If the compound still looks promising, it is now a potential treatment and needs to be tested in human clinical trials. Before any such testing is allowed, the researchers must get permission from the regulatory authorities (e.g. the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK) who look very closely at the evidence before agreeing that human clinical trials can go ahead. The safety of all volunteers who take part is paramount.


The development of new treatments to the point they are available for doctors to prescribe for the condition usually requires clinical trials over three or four phases, and a single clinical trial may span more than one phase, especially in rare diseases like PSC. Because there are so few patients, researchers must develop study designs that maximise everyone’s participation.

Trial Phases

Phase 1 - Is the treatment safe and how does it work?

Phase 1 trials are exploratory studies, focusing on safety, that look at various dosages of a new treatment, test treatment plans (e.g. every day, twice a day, once a week, and so on) and whether the treatment interacts with other medicines, and/or food.

Phase 2 - Does the treatment show any signs that it may be working?

Phase 2 trials are studies that, for the first time, look at whether a drug works well enough in volunteer patients. They usually test the drug at fewer dose levels and/or regimens compared to Phase 1 trials, and in order to determine the optimal dose for later test evaluation. Researchers continue to closely monitor how the body responds to the treatment and side-effects, and how the treatment is best administered.

Often Phase 2 trials involve small numbers of patients and look for very early signs that the new medicine may be helping – for instance, a positive impact on liver blood tests.

Phase 3 - Is the treatment better than what’s already available?

Phase 3 trials are studies that gather more information to confirm a drug's safety and effectiveness by studying different or larger patient populations. Usually a selected dosage from previous Phase 2 studies is used and sometimes in combination with other drugs. These studies typically involve more participants and are used to confirm the Phase 2 trial results.

In PSC, ‘effectiveness’ in phase 3 trials often looks at multiple areas of the disease, including a reduction in markers of liver scarring (fibrosis), reduction in liver inflammation, improvement in liver biopsy and scan findings, and the effects on quality of life.

Licensing the treatment

The evidence gathered about risks and benefits of the new treatment is critical. Again, regulatory authorities (e.g. MHRA) carefully check the data before deciding whether or not the treatment can be licensed for use.

Even if the treatment passes this hurdle, it’s not the end of the story. The newly approved medicine needs to be evaluated for its cost-effectiveness so it can be made available through the NHS in the UK. To make that decision, government authorities look at all the evidence and talk to people who will be affected by the decisions: patients and patient organisations, manufacturers of the treatment, and the manufacturers of any treatment that might be affected by the proposed new one, and to clinicians and other professional groups affected. This is where patient organisations need to be strong and prepared, and able to advocate for patients. When we get an effective treatment for PSC (which we will!), we must ensure PSC patients have access to it.

Phase 4 - The longer term and wider view

Phase 4 trials are studies that gather even more information about the treatment’s safety, efficacy, or optimal use and are conducted after it has been shown to work and has been licensed for use in the real world. These studies look at the treatment’s longer-term risks and benefits, and how the drug works in the wider population (for example, in PSC patients who were not suitable for previous trials).


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