There is a worldwide research effort underway to develop new and effective treatments for MND. The only drug licenced in the UK is riluzole, which slows down the progression of MND. A great deal has already been learnt about how clinical trials should and should not be carried out, paving the way to future trials.
It is important to remember that clinical trials are not treatments but scientific experiments. Due to the design of clinical trials, participants may not receive the new drug under investigation and may experience some side effects.
How do clinical trials proceed?
Traditionally, clinical trials are divided into four phases as described below. However, with increasingly complex trial designs the distinction between Phase II and Phase III trials often becomes blurred.
Phase 1 examines the safety of the potential new treatment, often in just a few (5 – 20) people. In many cases, this phase involves healthy volunteers rather than patients. Participants are monitored for adverse reactions or side effects; if any appear that are judged too dangerous, the drug will not advance to further clinical trials phases.
Phase 2 determines the optimal dose size, timing of doses and drug delivery route (e.g., by mouth, or injection) for the next phase of testing. Although Phase 2 may provide some indication of the drug’s ability to treat the disease, the numbers of patients involved in this phase is too small for such findings to be relied upon.
Phase 3 aims to show whether the drug has a beneficial effect on patients. This stage of testing will usually involve hundreds of patients, which is enough to allow a reliable assessment of the drug’s efficacy. Phase 3 results will determine whether a drug is approved for use to treat a disease.
Phase 4 occurs after the drug has been approved for sale. With the drug in general use, further data can be gathered on its effects in an extremely large number of people over an extended period of time.
Over the years, new designs of clinical trials have been developed to help improve the speed at which new potential treatments can be found. This has led to the development of platform trials.
Traditional clinical trials are designed to test a single potential treatment against a placebo (dummy drug). This type of trial design can help to show whether a treatment is effective against the disease or not. However, setting up trials for each individual potential treatment can be costly, time-consuming and present logistical challenges.
Platform trials have been implemented in recent years to combat the expensive and time-consuming set up process. These types of trials are designed to last for years to come and have a multi-arm adaptive design. This means that:
- Multiple treatments can be tested at the same time
- Treatments can be removed quickly if found to have no benefit
- New treatments can be added throughout the trial
- A larger number of people are randomised onto a treatment than a placebo
- An increased number of people are eligible for the trial as strict criteria for participation is reduced
What makes a good clinical trial?
Anybody could claim to have tested a treatment in a clinical trial but how would you know if their results were reliable? What makes for a really rigorous test of a potential new treatment?
A placebo controlled, double-blind, randomised clinical trial on a large number of participants, which is published in a reputable journal, is deemed a good clinical trial. Genuine and trustworthy trials will never expect participants to make any payment.
If you are told that a treatment has been tested in clinical trials, ask for more details and find out if the trial design included:
In a well-designed Phase 2 or 3 clinical trial, half of the participants will be given placebo (‘dummy drug’) instead of the trial drug. This is to ensure that the beneficial effects observed in the trial are entirely down to the trial treatment itself and not due to the power of positive thinking, the extra attention from medical staff that comes with participating in a trial, or any other factor.
Rigorous trials are 'double blinded'. This means that neither the doctors and nurses involved in the trial nor the trial participants know who is taking the real drug and who is taking the placebo. This prevents unintentional bias creeping in when participants are reporting how they feel or when the researchers are making assessments and looking at data.
Participants in trials should be assigned to the placebo group or treatment group at random. This is usually carried out by computer and prevents bias in choosing which patients get the trial drug.
Large number of participants
The later stages of clinical testing, where the goal is to establish with certainty whether or not a treatment is of benefit, will generally involve hundreds of participants. Only by including this many people can researchers be sufficiently sure that the effects they are seeing are not occurring by chance.
The results of successful clinical trials will usually be shared with the research community through publication in reputable medical journals. Before the results can be published in these journals, the methods used in the trial will be scrutinised by independent scientists to ensure that they are reliable.
If you have any questions about MND clinical trials and treatments you can contact our Research Development team.