The Use of 5-ALA in Paediatric Patients With High Grade Brain Tumours

A Phase II Multicentre Trial for the Use of 5-ALA Paediatric Patients With High Grade Brain Tumours

The aim of the study is to examine the safety and feasibility of using 5-ALA in children who have MRI scans showing an aggressive looking brain tumour. It will also study if 5-ALA can help the surgeon achieve maximal tumour removal and discriminate between normal brain tissue and tumour.

Patients will be between 3-18 years (inclusive) and all patients will receive 5-ALA 3-6 hours prior to resection surgery.

Inclusion Criteria:

• Magnetic Resonance Imaging confirmation of diagnosis consistent with either: primary presentation of high grade brain tumour OR patient relapsing with previously confirmed ependymoma or high-grade glial tumour

• Tumours will be either intrinsic supratentorial or infratentorial / posterior fossa, non-brainstem, tumours

• Patient is appropriate for maximal safe resection of tumour, based on Investigator and Multidisciplinary Team judgement

• 3 - 18 years inclusive

• Adequate liver and kidney function

  • creatinine - less than one and a half times the upper limit of normal.
  • haemoglobin above the lower limit of normal
  • Alanine transferase or Aspartate transferase less than twice the upper limit of normal (if both performed, then both results must be lower than twice the upper limit of normal
  • bilirubin lower than one and a half times the upper limit of normal

• Normal Coagulation profile

  o Prothombin Time, Activated Partial Prothombin Time and Thrombin Time all within normal limits

• Urine porphyrin - Test to be completed and sent for analysis

• Blood pressure lower than or equal to the upper limit of normal

• Negative pregnancy test in women of childbearing potential*

• Informed Consent *A positive pregnancy test that is suspected to be due to the location of the tumour is to be queried with the TMG prior to registration of the patient

Exclusion Criteria:

• Patients with low grade tumours (either radiological or histological diagnosis)
• Patient is appropriate for biopsy only, based on Investigator and MDT judgement
• Known history (including family history) of porphyria
• Hypersensitivity to the active substance or to porphyrins
• History of light sensitivity reactions
• Pregnant or breastfeeding women