Translation of Paediatric Precision Medicine Clinical Trials
The Clinical Trials program expanded the capabilities and resources for conducting innovative drug and gene therapy trials for children with rare genetic diseases and cancer, further enhancing investments in clinical trial infrastructure by the New South Wales Government.
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This project supported the delivery of clinical trials of novel gene and cell therapies to children with rare genetic diseases and cancer. We put children at the centre of trial design and development in order to increase the number of paediatric clinical trials in NSW.
We grew the capacity of clinicians and researchers to identify targets and opportunities for trials; enhanced understanding and awareness of trial methodology and protocol design; and developed an education, implementation and service model to deliver early phase and novel trials in the NSW health system.
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Lead Investigators
- Ms Lani Attwood
Advanced Therapeutics Program Manager, Kids Research, Sydney Children’s Hospitals Network - Adjunct Associate Professor Paula Bray
Director of Research, Sydney Children’s Hospitals Network - Professor Chris Cowell
[Past] Director of Research, Sydney Children’s Hospitals Network
Research Team
- Professor Craig Munns, [Past] Clinical Trials Lead, Sydney Children’s Hospital Westmead
- Dr Laura Fawcett, Clinical Trials Lead, Sydney Children’s Hospital Randwick
- Amy Boland, [Past] Clinical Trials Program Manager, Sydney Children’s Hospitals Network
- Ms Lani Attwood
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- Children’s Cancer Institute (Australia)
- Children’s Medical Research Institute (Australia)
- University of Sydney
- UNSW Sydney
- Pharmaceutical/Biotechnology
- Sydney Children’s Hospitals Network, Clinical Research Centre
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Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L, McMillan HJ, Finkel RS, Swoboda KJ, Kwon JM, Zaidman CM. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nature medicine. 2022 Jul;28(7):1381-9.
Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L, McMillan HJ, Finkel RS, Swoboda KJ, Kwon JM, Zaidman CM. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial. Nature medicine. 2022 Jul;28(7):1390-7.
D’Silva AM, Holland S, Kariyawasam D, Herbert K, Barclay P, Cairns A, MacLennan SC, Ryan MM, Sampaio H, Smith N, Woodcock IR. Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy. Annals of Clinical and Translational Neurology. 2022 Mar;9(3):339-50.
Dedrick RM, Smith BE, Cristinziano M, Freeman KG, Jacobs-Sera D, Belessis Y, Whitney Brown A, Cohen KA, Davidson RM, van Duin D, Gainey A. Phage Therapy of Mycobacterium Infections: Compassionate-use of Phages in 20 Patients with Drug-Resistant Mycobacterial Disease. Clinical Infectious Diseases. 2022 Jun 9.
Shih ST, Farrar MA, Wiley V, Chambers G. Newborn screening for spinal muscular atrophy with disease- modifying therapies: a cost-effectiveness analysis. Journal of Neurology, Neurosurgery & Psychiatry. 2021 Dec 1;92(12):1296-304.