About clinical trials and Usher
Promising treatments all have to go through human clinical trials before they are available on the market. Human clinical trials generally take several years to complete and consist of a number of phases. These trials are to ensure the safety and effectiveness of the treatment and consist of four phases.
These are normally small trials involving around 30 patients. The purpose is to assess the effectiveness of the technique or treatment as well as work out whether the treatment is safe, what dose should be recommended for further development and to observe any possible side effects.
Phase 2 trials often involve a larger number of patients and focus on whether the treatment has the desired effect. Safety and side effects continue to be monitored as the previous trial will have only involved small numbers of patients and different people can react differently to the same treatment.
Phase 3 will only go ahead if the results of Phase 2 indicate that the treatment may be as good, or better, than any existing treatment. Phase 3 involves hundreds or thousands of participants. At this stage trials are usually randomised, meaning participants are divided into two or more groups at random. One group will receive the treatment that is being trialled while the other receives any treatment which already exists or a placebo. A placebo is a treatment that has no actual therapeutic benefits and is used as a control. Placebos are used to help overcome bias because of the natural tendency of people to think that any treatment may be better than nothing.
For rare diseases, such as RP, it may not be possible or appropriate to conduct large phase 3 studies. If, as for many rare diseases there is no current treatment, then a licence could be considered on the basis of earlier phase data.
Phase 4 trials may be undertaken once the treatment is proven to work and has been granted a licence. The side effects and safety of the drug continue to be monitored but so too are any longer term risks and benefits for a wider group of patients than in the previous trials.
Taking part in clinical trials
Sense is regularly asked how people can take part in clinical trials and would first recommend raising the issue with your specialist. However there are also a number of registers that you can check to find out if there are any trials recruiting people similar to yourself.
One register is the UK Clinical Trials Gateway. There you can search for relevant trials by conditions near to where you live. All trials state whether they are recruiting, who they are looking to recruit, and how to make contact. It should be noted however that at the time of writing there were no trials related to Usher listed on the Gateway.
Additionally, the Usher Syndrome Coalition in the USA is encouraging people with Usher syndrome from all around the world to join a registry. Those signing up will be kept informed of any clinical trials they might be able to participate in as well as being kept informed of any advances in possible treatments.
If Sense is approached by research scientists looking to recruit for trials involving treatments for Usher syndrome in the future we will notify those people with Usher with whom we are in contact.
Important: It is important to remember that the outcome of a clinical trial you are involved with may not be what you desire and it is possible you could experience unexpected side effects. The team involved in any study will explain it to you fully, and you will be given written information (Patient Information Sheet). Only after you have been given enough time (at least 24 hours) to consider your involvement in a study, will the team ask for your consent. You should not agree to a study that you have any worries about.
You should also understand that you may be under follow-up from a clinical trial for a long time. This may mean that you may not be able to take part in another clinical trial whilst under follow-up.
Created: February 2016
Review due: February 2018
First published: Wednesday 2 December 2015
Updated: Monday 16 January 2017