In the pursuit of targeted therapies for myasthenia, pharmaceutical companies are carrying out extensive clinical trials to ensure the effectiveness and safety of these medicines.

What are clinical trials?

Clinical trials are research studies that are carried out when assessing the effectiveness and safety of a new medicine. They are divided into stages of progress that differ based on aim, size of cohort (the group of participants), and whether the phase is randomised. What randomisation means in this context is that the cohort will be split into two groups – one will receive the new treatment and the other will receive the standard therapy. Neither group is aware of what treatment they have received. This helps researchers assess the true effectiveness of the drug.

What do the stages of clinical trials mean?

There are generally four stages to a clinical trial. There is also technically a stage ‘0’ for clinical trials where the new therapy will be tested on a very small number of people or assessed using computers. From stage 1 onwards, the safety and dosage of the drug is examined. A trial cannot pass on to the next phase until safety has been assured. As a trial progresses, more participants are recruited. At stage 4, the medicine will have gained regulatory approval and be tested for its long-term effectiveness and safety.

Look for UK-based clinical trials in myasthenia

Read more about current medicines for myasthenia