N-of-1 trials are usually used for testing the effectiveness of medicines in individual patients.

Medicine is developing towards an era where treatments can be tailored to the individual.  This can be 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.

The advantage of N-of-1 trials is that the individual patient and their clinician receive direct evidence about the effect of the treatment versus the comparator on their own symptoms, therefore treatment can be individually tailored.

It is also 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.

Our group have successfully completed over 600 of these trials in various areas eg palliative care and paediatric medicine. Our current work includes an international collaboration with researchers at the University of Alberta, in Canada.


    Sleep Health

    Several projects with the aim to improve sleep in both adults and children are currently underway:

    Melatonin for initial insomnia in stimulant-treated paediatric ADHD (Funded by NHMRC)

    MyNap project contact: mynap@uq.edu.au 


    Melatonin for insomnia in Parkinson's Disease (Funded by Wesley Medical Research)

    Parkinsons project contact: insompd@uq.edu.au


    Other projects

    • Single Patient Multiple Cross-Over Trials To Determine The Efficacy Of Pilocarpine 5mg Orally Dissolving Tablets In Relieving Dry Mouth In Patients Experiencing Xerostomia
    • A series of N-of-1 trials to assess therapeutic interchangeability of two Enalapril formulations in the treatment of hypertension, Addis Ababa, Ethiopia
    • A pilot series of n-of-1 randomised controlled trials to compare Pregabalin and Gabapentin for persistent neuropathic pain

    Completed projects

    • Using n-of-1 trials to determine the effectiveness of paracetamol in advanced cancer patients on opioids. (Funded by the NHMRC)
    • Using single patient trials to determine the effectiveness of psycho-stimulants in end stage fatigue in cancer patients. (Funded by the NHMRC)
    • N-of-1 trials of stimulants compared to placebo for traumatic brain injury in children. (Funded by Motor Accident Insurance Commission)

    The following projects have positions available for postgraduate students.

    Pediatric sleep research

    Does melatonin assist children with ADHD and insomnia to sleep better?

    Children’s sleep survey –how much improvement in sleep is enough? – from parent, child and clinician perspectives

    Parkinson’s Disease sleep research

    Does melatonin assist patients with Parkinson’s Disease and insomnia to sleep better?

    Quality use of medicines

    A series of papers about the impact of using Dose Administration Aids in Australian veterans

    N-of-1 trial research

    A series of systematic reviews of different applications of N-of-1 trials focusing on adverse events, dose comparisons and other applications of N-of-1 trials

    The n-of-1 team collaborates with top international researchers in the field and with clinicians who wish to use n-of-1 trials in their practice including sleep physicians, sleep psychologists, various clinicians and other researchers with an interest in sleep health in Australia and internationally.

    The n-of-1 research team regularly engage with support and consumer groups, giving talks and updates on research.

    They have recently been in the media discussing the melatonin for insomnia in ADHD trial currently underway. 

    For more information see our Facebook page and Twitter page.

    The melatonin for insomnia in Parkinson's Disease trial is supported by Parkinson's Australia. For more information see our Facebook page. 

    Core research team

    • Associate Professor Jane Nikles

      Principal Research Fellow
      UQ Centre for Clinical Research
      Principal Research Fellow
      RECOVER Injury Research Centre
      Casual Snr Researh Assist/Tech
      School of Nursing, Midwifery and Social Work
    • Dr Thomas Harris

      Dr Thomas Harris

      Adjunct Fellow
      UQ Centre for Clinical Research
    • Megdelawit Melaku

      Ms Megdelawit Melaku

      Casual Snr Research Technician & Casual Research Assistant
      UQ Centre for Clinical Research
      Casual Research Assistant
      Primary Care Clinical Unit