Clinical trial NCT06678867: The Use of 5-ALA in Paediatric Patients With High Grade Brain Tumours
Official title: A Phase II Multicentre Trial for the Use of 5-ALA Paediatric Patients With High Grade Brain Tumours
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Brain Tumor
Keywords: Ependymoma
Glioma
Paediatric
5-ALA
Brain tumour
Lead sponsor: University College, London
Sponsor class: OTHER
Collaborators: name: University of Nottingham
First posted: 2024-11-07
Enrollment: 80
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This is a phase II, multicentre, non-randomised open-label trial with two cohorts.
Primary purpose: OTHER

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Cohort 1: Complete resection rate and fluorescence rate of supratentorial instrinsic high grade brain tumours. Cohort 2: Fluorescence rate of infratentorial tumours.
timeFrame: Two weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Nottingham
country: United Kingdom
facility: Nottingham Children's Hospital
Study officials:
References: