Dialog Box

Children's Tumour Foundation of Australia

TiNT MEK Inhibitor Clinical Trial

TiNT MEK Inhibitor Clinical Trial

TiNT: A phase II study of Trametinib in paediatric, adolescent and young adult patients with Neurofibromatosis Type 1 and associated plexiform neurofibromas or progressive optic pathway gliomas.

In 2019, ANZCHOG approached the Children's Tumour Foundation (CTF) to support its efforts to run a phase II MEK Inhibitor clinical trial across multiple paediatric and adolescent/young adult cancer centres in Australia and New Zealand. 

This is the first Australian-led clinical trial of the MEK Inhibitor drug, Trametinib and the first NF research study to receive funding from the Australian Federal Government. The seven-year study will investigate the effectiveness of Trametinib for the treatment of plexiform neurofibromas and optic pathway gliomas. In addition to monitoring the expected reduction in tumour size, it will also monitor the improvement in pain, function, and quality of life, as well as brain function and social development.

For the past 18 months, the Children's Tumour Foundation has been advocating and raising funds to support the trial, which is expected to start enrolling patients as early as January 2021.

Once the trial is complete, the results will contribute to a body of evidence that supports making this drug affordable and available to anyone living with these specific tumour types as a front-line treatment, and not a last resort. 

Webinar: Everything you need to know about the TiNT MEK Inhibitor Clinical Trial

Presented: Wednesday 2 December 2020


Enrolments are expected to begin as early as January 2021 for up to 60 patients from hospitals around the country and will occur directly through existing patient lists. To be eligible for the trial, a patient will need:

  • to be aged between 3 months and 25 years when they start the trial
  • to have NF1 
  • to have an optic pathway glioma where the tumour has continued to grow or the vision tests have deteriorated after treatment such as chemotherapy; or
  • to have a plexiform neurofibroma which is causing significant problems and where surgery is not an option. This could include symptoms like nerve pain, cosmetic issues, nerve or spinal compression and other problems. 

You may be eligible for the control arm of the sub-study if you are between 3 months and 25 years old with NF1.

The team are also looking for volunteers with NF1 who do not have a plexiform NF or progressive OPG and therefore do not need treatment with Trametinib. These children / young people will be asked to complete the same cognitive (learning) assessments as children receiving the medication to provide a comparison. 




  • Sydney Children's Hospital, Randwick
  • Sydney Children's Hospital, Westmead
  • The Royal Children's Hospital, Melbourne
  • Monash Children's Hospital, Melbourne
  • Queensland Children's Hospital, Brisbane
  • Women's and Children's Hospital, Adelaide
  • Perth Children's Hospital
  • John Hunter Children's Hospital, Newcastle
  • Royal Hobart Hospital
  • Starship Children's Hospital, Auckland
  • Christchurch Children's Hospital

expected Timings :  starting JANUARY 2021

  • Recruitment Duration: 2 Years
  • Treatment Duration: 2 Years
  • Follow-up Duration: 3 Years
  • Total trial Duration: 7 Years


Click the below link to be redirected to the official Australian and New Zealand Clinical Trial Registry (ANZCTR) website: