NCT04738162

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 78 (actual)
• Sponsor: Universität Münster · Academic / Other
• Sex: All
• Age: 3 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

In this prospective, open, single-armed, multicenter, phase II study for application of 5-ALA in children and adolescents with brain tumors 80 patients will be investigated. Primary objective of the study is to determine the safety of 5-ALA for fluorescence-guided resections in children and adolescents with intra-axial brain tumors. Secondary objectives are * to determine whether fluorescent tissue truly signifies tumor (positive predictive value) in various pediatric brain tumors * to determine the degree of tumor resection on early post-operative MRI * and to determine the pharmacokinetics of 5-ALA in this population.

Detailed description

In 2007, 5-aminolevulinic acid (5-ALA) was approved in Europe by the European Medicines Agency (EMA) (brand name: Gliolan®) for "the visualization of malignant tissue during surgery for malignant glioma (WHO III and IV) in adults." Similarly, approval for 5-ALA was granted by the FDA in 2017 as an "optical imaging agent indicated in patients with gliomas (suspected World Health Organization Grades III or IV on preoperative imaging) as an adjunct for the visualization of malignant tissue during surgery" (brand name: Gliolan®). Goal of the study is to investigate if the use of 5-ALA is safe in children and get preliminary information on the type of paediatric brain tumors which are suitable for fluorescence-guided resection with 5-ALA.

Conditions

• Brain Tumor, Pediatric

Interventions

Type	Name	Description
DRUG	5-Aminolevulinic Acid Hydrochloride, Oral	Application of 5-ALA oral solution (20mg/kg bw) 4 hours (range 3.5-4.5 hours) prior to anesthesia followed by fluorescence-guided tumor resection Tumor resection is performed conventionally using a surgical microscope. A change from white light to blue light is possible at anytime to make the fluorescence visible

Timeline

• Start date: 2020-09-25
• Primary completion: 2025-11-24
• Completion: 2025-11-24
• First posted: 2021-02-04
• Last updated: 2025-12-10

Locations

7 sites across 2 countries: Germany, Netherlands