Safety of 5-Aminolevulinic Acid in Children with Brain Tumors

Clinical Safety Study on 5-Aminolevulinic Acid (5-ALA) in Children and Adolescents With Brain Tumors

Phase 2 Interventional Universität Münster · NCT04738162

This study is testing if a special drug called 5-aminolevulinic acid can safely help doctors see and remove brain tumors in children and teenagers.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment80 (estimated)
Ages3 Years to 17 Years
SexAll
SponsorUniversität Münster Academic / other
Locations7 sites (Augsburg and 6 other locations)
Trial IDNCT04738162 on ClinicalTrials.gov

What this trial studies

This phase II clinical trial investigates the safety of 5-aminolevulinic acid (5-ALA) for fluorescence-guided resections in children and adolescents with intra-axial brain tumors. The study will enroll 80 patients and aims to assess the positive predictive value of fluorescent tissue in identifying tumors, the extent of tumor resection through early post-operative MRI, and the pharmacokinetics of 5-ALA in this population. The trial is conducted across multiple centers to gather comprehensive data on the use of 5-ALA in pediatric patients.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents aged 3 to under 18 years with a first diagnosis of intra-axial brain tumors requiring surgical resection.

Not a fit: Patients with non-contrast enhancing tumors or those not requiring surgical intervention may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could improve surgical outcomes and safety for children undergoing tumor resections.

How similar studies have performed: Previous studies have shown success with 5-ALA in adults, but this application in pediatric populations is novel.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Age 3 - \<18 years
* First radiological diagnosis of intra-axial, contrast-enhancing tumor on MRI or recurrent supratentorial intra-axial brain tumor (malignant glioma, astrocytoma, malignant ependymoma, atypical teratoid rhabdoid tumors (AT/RT), Oligodendroglioma, etc.)
* Resection is part of therapeutic strategy with an emphasis on neurological safety
* Informed consent by the parents or guardians and if possible assent of the patient after education of purpose and risks of study. Patients that are able to understand should provide assent to participate in the trial
* Female adolescents: not pregnant (pregnancy test required for adolescents of child-bearing age) and not breast-feeding (for at least 24 hours after Gliolan intake). Female patients of childbearing potential and male patients who are sexually active must be practising a highly effective method of birth control up to 6 weeks after the tumor operation consistent with local regulations regarding the use of birth control methods for subjects participating in clinical trials.

Exclusion Criteria:

* Extra-axial tumors such as craniopharyngioma
* Entities precluding surgical resection
* Acute or chronic porphyria
* Hypersensitivity to 5-ALA or porphyrins
* Renal insufficiency: serum creatinine \> 2x upper limit of normal (ULN)
* Hepatic insufficiency: serum bilirubin \> 2x ULN, serum γ-glutamyl transferase \> 2,5 x ULN, alanine transaminase (ALT) and aspartate transaminase (AST)\> 2,5 ULN
* Blood clotting: INR (international normalized ratio) out of acceptable limits
* Other malignant disease
* Patients with pre-existing cardiovascular diseases
* Co-administration with other potentially phototoxic substances (e.g. tetracyclines, sulfonamides, fluoroquinolones, hypericin extracts)
* Planned administration of potentially hepatotoxic substances within 24 hours after 5-ALA administration

Where this trial is running

Augsburg and 6 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Brain Tumor, Pediatric5-ALAintra-axialFluorescence-guided resectionpediatric
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.