Centre for Clinical Trials in Rare Neurodevelopmental Disorders (CCTRND)

History

The Centre for Clinical Trials in Rare Neurodevelopmental Disorders (CCTRND) was established in 2016 with a small team of just two. Over three years, it has grown to include a multidisciplinary team from diverse backgrounds, who together support research into treatments for those living with neurodevelopmental disorders.

CCTRND Team

Within the Centre for Children’s Health Research (CCHR), the CCTRND team works to support and conduct a portfolio of clinical trials providing access to new treatment options for children with rare neurodevelopmental disorders including; Autism Spectrum Disorder, Fragile X Syndrome, Angelman’s Syndrome, Rett Syndrome and 22q11.2 Deletion Syndrome. Our team is made up of a variety of Medical, Nursing, Psychology and Administrative staff trained in Good Clinical Practice.

Emily Milburn: Clinical Trial Coordinator
Ellen Taylor: Project Support Officer
Suzie Harte: Senior Research Officer
Mary Daly: Clinical Research Nurse
Clare Larkin: Clinical Research Nurse
Michael Duhig: Educational and Developmental Psychologist, Team Leader
Selina Dunn: Educational & Developmental Psychology Registrar
Amy Lynch: Clinical Psychology Registrar
Dr Mary-Clare Sherlock: Medical Registrar
Dr Yeeshu Singh: Paediatric Neurology and Epilepsy Fellow
Dr Geoff Wallace
Dr Wallace is currently appointed as a Senior Medical Officer within Children’s Health Queensland and holds a Senior Lecturing role at the University of Queensland. He has worked in multiple children’s hospitals across Queensland and in private practice in as a specialist in paediatric neurology. Geoff has supported the compassionate access scheme for children in Queensland to access medicinal cannabis to treat severe, drug-resistant epilepsy.
Dr Helen ‘Honey’ Heussler
Dr Heussler is the current Medical Director of the Child Development Program at Children’s Health Queensland. She is appointed as Senior Medical Officer in Sleep Medicine and as an Associate Professor at The University of Queensland’s School of Medicine. Honey is on multiple advisory committees for family groups and research groups throughout Australia and Internationally, providing expert opinion and experience in research and training. She is dedicated to initiatives and research supporting children with developmental disabilities and in children with sleep difficulties.

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Current research

1. Medicinal Cannabis Compassionate Access Scheme as treatment for children with severe treatment-resistant epilepsy

2. Study of Cannabinoid gel in children and adolescents with 22q11.2 Deletion Syndrome

  • We are currently trialling a topical cannabinoid gel (ZYN002) on children 22q11.2 Deletion Syndrome (22Qds). This study is looking to see if treatment with ZYN002 is safe, tolerated and effective in the treatment of symptoms of 22qDS. Male and female children between the ages of 6 to 18 years old with a diagnosis of 22qDS may be eligible for this trial.
  • STATUS: RECRUITING
  • Further information from ANZCTR: https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=377493&isReview=true
  • Contact information Email: CHQ_CCTRND@health.qld.gov.au
    Phone: 07 3069 7532

3. Study of Ganaxolone treatment in children and young adults with cyclin-dependent kinase-like (CDKL5) deficiency disorder (CDD)

4. Study of OV101 in paediatric individuals with Angelman Syndrome

  • We are currently trialling a new drug called OV101 (gaboxadl) in children with Angelman Syndrome (AS). This study is looking to see if treatment with OV101 is safe, tolaterated and effective with the symptoms of patients with AS. Male and female children between the ages of 2 and 12 years of age with molecular confirmation of Angelman Syndrome may be eligible for this trial.
  • STATUS: RECRUITING
  • Further information from: ANZCTR
  • Contact information Email: CHQ_CCTRND@health.qld.gov.au
    Phone: 07 3069 7532

5. Parent/guardian interview study of patients accessing the Medicinal Cannabis Compassionate Access Scheme

  • We are currently interviewing parents and guardians of children who have or are currently accessing the Medicinal Cannabis Compassionate Access Scheme (CAS) through Children’s Health Queensland. These interviews will explore the experiences of families of children who have participated in the CAS and compare these experiences with objective seizure evidence. If your child has been involved in the CAS program, please contact Centre for Clinical Trials in Rare Neurodevelopmental Disorders to discuss your possible involvement in the interview study.
  • STATUS: RECRUITING
  • Contact information Email: CHQ_CCTRND@health.qld.gov.au
    Phone: 07 3069 7532

6. Eye gaze tracking as a novel objective diagnostic tool for anxiety in children

  • We are currently exploring the repeatability and usefulness of eye gaze tracking as a tool for diagnosing anxiety in infants and children. Study One involves the validation of the tool with infants and children recruited from our general community, and subsequent studies will include children diagnosed with a rare neurodevelopmental disorder and children diagnosed with anxiety. If you have a child aged 6 months to 12 years with no specific diagnosed neurodevelopmental disorder, and you are interested in validating the test environment, please contact the CCTRND to discuss your involvement in Study One.
  • STATUS: RECRUITING
  • Contact information Email: CHQ_CCTRND@health.qld.gov.au
    Phone: 07 3069 7532

Clinical trials explained

What we offer

The Centre for Clinical Trials in Rare Neurodevelopmental Disorders offers new treatment opportunities for your child that may not currently be widely available as a typical treatment avenue.

What is a clinical trial?

A clinical trial develops a safe and controlled environment to test new treatments and provide evidence as to how new treatments or interventions might work to improve health conditions and services. Research involving children, from babies to adolescents, is essential to improve our understanding of childhood diseases and conditions and provide care for children and young people based on the best possible evidence. At present, many medicines given to children have not been tested specifically with children. As a result, there is only limited evidence available to prescribers about the most appropriate dosage, and about how children may respond¹.

¹https://globalhealthtrainingcentre.tghn.org/elearning/education/elearning-courses/children-clinical-research/2272/

New drug treatments are tested to ensure their safety by going through phased trials.

  • Phase 1: clinical safety trials to identify dosage range and side effects;
  • Phase 2: efficacy and safety trials in a larger group (several hundred);
  • Phase 3: clinical trial in a large group to monitor efficacy and side effects;
  • Phase 4: studies to monitor drug therapy effectiveness in the population.

The findings of clinical trials are collated and made available through a variety of channels including scientific publications. The ‘levels of evidence pyramid’ demonstrates how clinical trials contribute to evidence-based decisions about clinical treatment options for our patients.

Evidence-based medicine pyramid

Figure 1: Evidence-based medicine pyramid. Sackett, D. et al, 2000

How can a clinical trial benefit you?

A clinical trial may allow your child to access new treatments that are not available on the general market. Taking part in a clinical trial may or may not benefit you or your child directly. In some circumstances it may be helpful to determine if a particular medication might be suitable for your child such as in cross-over trials. However, participation in trials will further advance information as to the best solutions to support children with a variety of neurodevelopmental challenges, helping other families and children across the globe.

What do I do to get involved in a clinical trial?

There are certain criteria that a clinical trial need to meet to proceed safely and within a research code of ethics. You can express your interest in a trial by providing basic demographic information for your child like age and gender and any specific information such as current medications or medical diagnosis. You may be accepted to join the trial and be ‘screened’ to ensure your child will be suitable for the trial and that the trial may benefit them.

Conditions

What is a rare neurodevelopmental disorder?

The term neurodevelopmental disorder is used in two ways, firstly to describe genetic conditions which affect a child’s neurological development such as Fragile X Syndrome. Secondly, to highlight conditions of multi-faceted origin which impair neurodevelopment such as autism spectrum disorders2. A rare neurodevelopmental disorder refers to one which is not commonly seen and affects a small number of people. The Centre for Clinical Trials in Rare Neurodevelopmental Disorders supports research that could provide new treatment and interventions to improve the health conditions and services available to this population.

2Bishop DV. Which neurodevelopmental disorders get researched and why?. PLoS One. 2010;5(11):e15112. Published 2010 Nov 30. doi:10.1371/journal.pone.0015112

Publications and Conference Presentations

Publications

  1. Heussler H, Cohen J, Silove N, Tich N, Bonn-Miller MO, Du W, O’Neill C, Sebree T. 2019. A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome, Journal of Neurodevelopmental Disorders. https://www.ncbi.nlm.nih.gov/pubmed/31370779
  2. Kwan Cheung K A, Peiris H, Wallace G, Holland O J, Mitchell M D. 2019. The Interplay between the Endocannabinoid System, Epilepsy and Cannabinoids, International Journal of Molecular Sciences. https://www.mdpi.com/1422-0067/20/23/6079
  3. Morawska A, Mitchell A E, Etel E, Kirby G, McGill J, Coman D, Inwood A. 2019. Psychosocial functioning in children with phenylketonuria: Relationships between quality of life and parenting indicators, Child: care, health and development. https://onlinelibrary.wiley.com/doi/abs/10.1111/cch.12727

Presentations

  1. National Fragile X Association, United States of America: Helen Heussler, Jonathan Cohen, Natalie Silove, Nancy Tich, Carol O’Neill, Marcel O. Bonn-Miller. 2019. Transdermal Cannabidiol (CBD) Gel for the Treatment of Fragile X Syndrome (FXS).
  2. Children’s Health Queensland Health Services Research Workshop, Brisbane. 2019. Real Life Stories: Parental and Carer Perceptions and Observations of the Benefits of Cannabidiol Treatment for Children Diagnosed with Refractory Epilepsy.

Symposium

  1. 22Q11.2DS Research Symposium, 7-8 December 2019, Brisbane, QLD.
  2. Inaugural Scientific Symposium, Centre for Clinical Trials in Rare Neurodevelopmental Disorders, 6-7 February 2020, Queensland Children’s Hospital, Brisbane, QLD.

Posters

  1. Society for the Study of Behavioural Phenotypes National Symposium, Melbourne. Helen Heussler, Jonathan Cohen, Natalie Silove, Nancy Tich, Carol O’Neill, Marcel O. Bonn-Miller. 2019. Transdermal Cannabidiol (CBD) Gel for the Treatment of Fragile X Syndrome (FXS)
  2. Society for the Study of Behavioural Phenotypes International Research Symposium, Birmingham: Elizabeth Merikle, Vanessa Patel, Terri Sebree, Carolyn Kreusser and Helen H. Heussler. 2019. Content Validity of the ABC-CFXS and Subscales in Fragile X Syndrome.
  3. International Congress on Clinical Trials in Cannabis. Suzanne Harte, Stephen Malone, Yeeshu Singh, Geoffrey Wallace. 2019. Parental and Carer Perceptions and Observations of the Benefits of Cannabidiol Treatment for Children Diagnosed with Refractory Epilepsy.
  4. Inaugural Scientific Symposium, Centre for Clinical Trials in Rare Neurodevelopmental Disorders, Brisbane. Suzanne Harte, Geoffrey Wallace, Honey Heussler. 2020. Eye gaze tracking as a biometric assessment tool for anxiety: A pilot study of cortisol related anxiety in children.

Video presentations

Location

The Centre for Clinical Trials in Rare Neurodevelopmental Disorders is located in the Centre for Children’s Health Research (CCHR), South Brisbane, providing full access to the academic environment within the research building and close integration with clinical staff and families in the nearby Queensland Children’s Hospital.

Contact information

e: CHQ_CCTRND@health.qld.gov.au
p: 07 3069 7532
Monday-Fridays 8am-4pm

Useful links

Australian New Zealand Clinical Trials Registry
Australian Clinical Trials
Autism Awareness Australia
Rare Voices Australia
Genetic Alliance Australia
Centre for Genetics Education
Orphanet
Positive Exposure