Phase I Trial of Deflexifol for Refractory or Recurrent Paediatric Central Nervous System (CNS) Tumours

Abstract

This is a multicentre Phase I clinical trial to establish if Deflexifol is safe and effective in children, adolescents and young adults with recurrent or refractory brain tumours.

Who is it for? Participants may be eligible for this study if they are older than 12 months and up to 21 years old and have a recurrent or refractory brain tumour; or newly diagnosed diffuse intrinsic pontine glioma (DIPG) or diffuse midline glioma (DMG) participants after completion of radiotherapy.

Study details Participants will receive Deflexifol every 2 weeks for up to approximately one year, if there is ongoing clinical benefit. Deflexifol will be administered via an injection over 3-5 minutes (bolus), followed by a continuous intravenous infusion over 46 hours. Pharmacokinetics will be measured during the first 4 weeks and safety will be assessed throughout the course of treatment and during follow up visits. Participants will have physical examinations, blood tests, urine tests, echocardiogram, electrocardiogram (ECG) and MRI scans.

This study will test the safety and effectiveness of this new drug in children and adolescents in cases where treatment options are limited.