Clinical studies cover any form of research involving people. This includes studies, to understand more about neuromuscular disorders and how they progress, and clinical trials, to assess the safety and effectiveness of specific interventions. These interventions could be medical products, for example, drugs or devices; medical procedures, such as a type of surgery; or changes to a person’s lifestyle, such as an exercise regime.

As with any experiment, researchers design clinical trials to answer specific research questions. After reviewing what is already known about the drug or intervention, from preclinical studies or previous trials, researchers will develop a specific plan or protocol, outlining:

  • who can take part (eligibility criteria)
  • how many people are needed at each stage to ensure robust data collection
  • how long the study will last
  • whether or not there will be a control group (to limit bias)
  • how the treatment will be administered and at what dosage
  • what will be measured (outcome measures) and when
  • how the data will be reviewed and analysed.