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 

    Register

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

    Parkinson’s Disease is a progressive, degenerative neurological condition affecting a dopamine system of the brain, resulting in movement disorders.  It is Australia’s second commonest neurological disease and is growing in numbers at a rate of 17% per year over the last six years. Approximately two thirds of patients with Parkinson’s disease experience one or more sleep-related non-motor symptoms, with insomnia being the most common reported in 37% of patients. The neurodegenerative process in the brainstem, disturbances of circadian rhythm, the effect of symptoms of Parkinson's Disease on sleep, and concomitant sleep disorders contribute to sleep disturbances.

    Melatonin is a hormone released by the pineal gland which regulates the sleep-wake cycle, temperature regulation, reproductive rhythm, and immune function. Melatonin regulates the circadian rhythm by promoting the desire to sleep at night. Studies in other populations indicate that melatonin can increase sleep efficiency, decrease night-time activity, and shorten sleep latency, and may be particularly effective to help promote sleep onset.

    Sleep disorders often cause major discomfort in Parkinson’s Disease and impact on the health and quality of life of sufferers and their families. Despite the recognised impact of sleep disturbance in PD, there have been few studies in this area.

    Melatonin is used to help sleep, but this scientific study will determine whether there is a true effect in individual patients with Parkinson’s disease. By recording individual symptoms in a sleep diary and using a watch to record sleep patterns, we will be able to identify individual responders and non-responders to melatonin.

    This will have positive impacts on health and quality of life for both patients and their family members (especially carers).

    Participating in the study

    To be eligible for this study people must be:

    • Aged over 30 years
    • Diagnosed with Parkinson’s Disease by a neurologist
    • Experiencing sleep difficulties, particularly getting to sleep.

    If already on melatonin you can still join the study.

    Please click on the links below to download more detailed information about our study.

    Three good reasons for taking part in this study

    1. You will find out if melatonin helps you by improving your ability to get to sleep, and your sleep duration and quality.
    2. At the end of your time on the study, a detailed and individual report about whether the melatonin worked for you will be given to your doctor to discuss with you.
    3. You will help others by assisting us to determine if melatonin should be included in treatment plans for people with Parkinson’s Disease who are having trouble sleeping

    Parkinsons project contact: insompd@uq.edu.au

    Other sleep health projects include:

    • Randomised double blind placebo controlled phase III trial of oral melatoning for the prevention of delirium in hospitalised advanced cancer patients

    • How much improvement in sleep time is enough? A survey of parent, clinicians and consumers

    • Actigraph validation study

    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 MyNAP trial currently underway. 

    9 News: New medical study to trial natural medicine for sleepless children with ADHD

    UQ News: National study seeks sweet relief for childrenw ith ADHD

    For more information see our Facebook page and Twitter page.

    Core research team

    My Nap team

    • Honey Heussler
    • Manisha Witmans
    • Sunita Vohra
    • Salima Punja
    • Chris Schmidt
    • Marcin Sowa

    InSom-PD investigative team

    • A/Prof John O’Sullivan, Senior Visiting Neurologist, Royal Brisbane & Women’s Hospital (RBWH) and Associate Professor of Medicine, Neurology Department, School of Medicine, St Andrew’s, Wesley and RBWH Hospitals, The University of Queensland
    • Dr Susanna Mantovani, Neurodegenerative Diseases Research Fellow, The Wesley Medical Research and UQ School of Biomedical Sciences, The Wesley Medical Research and The University of Queensland
    • Dr Alex Ritchie, Thoracic and Sleep Physician, St Andrew's War Memorial Hospital, Royal Brisbane and Women’s Hospital
    • Dr Simon Smith, Senior Research Fellow, Recover Injury Research Centre, The University of Queensland and Adjunct A/Prof, Centre for Children’s Health Research (Institute for Health and Biomedical Innovation)  Queensland University of Technology.

    PhD and Masters students

    • Chale Alemayehu
    • Guy Bashford