N-of 1 trials
Medicine is developing towards an era where treatments can be tailored to the individual. This is usually done by genetic testing to determine the likelihood that a treatment will work. However, there is a simpler way - single patient or N-of-1 trials.
Single Patient trials (also called N-of-1 trials) are multiple-cycle, double blind, placebo-controlled crossover trials using standardised measures of effect. The randomisation order is independently generated for each patient. At the end of the trial the order is revealed, and the patient response is compared against the presence or absence of the test treatment.
N-of-1 trials are usually used for testing the effectiveness of medicines in individual patients. However, it is possible to combine the results of many N-of-1 trials to determine the effect of a therapy for a population, thus allowing rapid accumulation of strong evidence on treatment effects. Advantages of N-of-1 trials are, firstly, the individual patient and their primary clinician receive direct evidence about the effect of the treatment versus the comparator on their own symptoms; therefore treatment can be individually tailored; secondly, a series of individual trials of a given treatment can yield an estimate of the population effect.
Our group are leading the development and conduct of N-of-1 trials internationally, and have successfully completed over 500 of these trials in the areas of palliative care and paediatric medicine. Our current work includes an international collaboration with researchers in the University of Alberta, in Canada.
- Melatonin For Initial Insomnia In Stimulant-Treated Paediatric ADHD. (Funded by the NHMRC, Project Contacts: Dr Jane Nikles, Professor Geoff Mitchell)
- Using n-of-1 trials to determine the effectiveness of paracetamol in advanced cancer patients on opioids. (Funded by the NHMRC, Project Contacts: Professor Geoff Mitchell, Dr Jane Nikles)
- Using single patient trials to determine the effectiveness of psycho-stimulants in end stage fatigue in cancer patients. (Funded by the NHMRC, Project Contacts: Dr Jane Nikles, Professor Geoff Mitchell)
- N-of-1 trials of pilocarpine vs placebo for dry mouth in palliative care patients. (Funded by The Cancer Council, Project Contacts: Dr Jane Nikles, Professor Geoff Mitchell)
- N-of-1 trials of stimulants compared to placebo for traumatic brain injury in children. (Funded by Motor Accident Insurance Corporation, Project Contacts: Professor Geoff Mitchell, Dr Jane Nikles)
For more information regarding our research, please contact Professor Geoff Mitchell