These trials involve giving new treatments (that have already been tested in the lab) to people to see if they work as expected. They are the only way to find out if a new drug is safe, if it works, and if it works better than any other drugs that may be available.
These trials have several different stages, and at each one, doctors are looking for different information.
Phase 1 trials
These trials are usually very small, involving only a few people. They are sometimes called ‘first-in-man’ studies, because they are the earliest tests of a new treatment in people. They aim to find out the safe dose to give and if there are any side effects.
Phase 2 trials
Once a phase 1 trial has shown that a new treatment is safe, a phase 2 trial can start to investigate whether it is effective. Phase 2 trials involve a few more people than phase 1 trials, but still usually less than 100. They also tell doctors more about the best dose to give and if there are possible side effects.
Phase 3 trials
Phase 3 trials involve large numbers of people. They are designed to test a new treatment against the current standard treatment, if one exists. If a phase 3 trial shows that the new treatment gives better results, it may become the new standard treatment.
Phase 4 trials
Some people also talk about phase 4 trials. These are for treatments that are already licensed. These focus on investigating the long-term implications of using the treatment.
Before they can be sold in Europe medical devices, such as insulin pumps and glucose meters, must carry a ‘CE’ mark to say they comply with safety regulations. In the UK, devices can only carry this mark if they get approval from the Medicines and Healthcare Regulatory Authority (MHRA), which makes its decision based on information from the manufacturer, including the results of clinical trials.
This means clinical trials are an essential part of getting new medical technology approved. The trials need to show that the device is safe to use, how best to use it, and if it works as expected. For example, the artificial pancreas is being tested in children and adults, overnight and during the day, and with varying amounts of supervision.
Once that information is collected, it can be submitted to the MHRA as part of the approval process.
Other kinds of trial
Clinical trials aren’t always focused on new treatments. Scientists doing lab research often need blood samples or information from people with type 1 to help them understand the condition better. So researchers are always looking for people who are willing to help them.
These studies can be quite varied, so each one may want you to do something slightly different. Some may ask you to fill in a single questionnaire or give a blood sample, while other studies might want a similar commitment over a number of years, to track how things change.
Many studies also need to involve people who don’t have type 1 themselves: they might need volunteers who have a brother or sister with type 1, or people who don’t have any link to type 1 at all. In this way friends and family can play a vital role in the search for the cure for type 1 diabetes.
We are funding many clinical trials, but one of the biggest challenges can be finding volunteers to take part in the studies. So if you are interested in helping with vital research, please visit our list of clinical trials or find out more about what it is like to take part in a trial. You can also look for trials through the NIHR’s Be Part of Research website.
Whatever kind of trial it is, deciding to participate is a very personal decision. Before you decide to get involved, the research team will talk you through exactly what you will be asked to do and what the possible effects might be. If, after talking to them, you decide you don’t want to participate, don’t worry. You are under no pressure to take part and the research team will not think any less of you or hassle you to change your mind.
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