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

NCT06678867 | Not Yet Recruiting Phase 2 DMC

• 2 other identifiers: UCL/154416CCLGA 2022 27 Dandapani
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

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.

Conditions

Brain Tumor

Keywords

Ependymoma; Glioma; Paediatric; 5-ALA; Brain tumour

Outcome Measures

Primary Outcomes (1)

• Cohort 1: Complete resection rate and fluorescence rate of supratentorial instrinsic high grade brain tumours. Cohort 2: Fluorescence rate of infratentorial tumours.

  Timeframe between informed consent and surgery

  Two weeks

Study Arms (1)

Patients with high grade brain tumours

OTHER

This is a single-arm study with two cohorts

Drug: 5-ALA (Gliolan)

Interventions

5-ALA (Gliolan) DRUG

Gliolan 30 ml/mg powder for oral solution, Single dose 3-6 hours prior to surgery

Patients with high grade brain tumours

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University College, London: lead
• University of Nottingham: collaborator

Study Sites (1)

Nottingham Children's Hospital

Nottingham, United Kingdom

Location

MeSH Terms

Conditions

Brain Neoplasms; Ependymoma; Glioma

Condition Hierarchy (Ancestors)

Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Central Study Contacts

Paed 5-ALA Trial Manager

CONTACT

020 7679 9860; ctc.paed5-ala@ucl.ac.uk

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is a phase II, multicentre, non-randomised open-label trial with two cohorts.

Study Record Dates

• First Submitted: October 28, 2024
• First Posted: November 7, 2024
• Study Start: March 25, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 30, 2026
• Last Updated: March 27, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)