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

UKM2013_0034

In this prospective, open, single-armed, multicenter, phase II study for application of 5-ALA in children and adolescents with supratentorial 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 supratentorial, 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.

2020-09-25

2024-10-01

2024-10-01

Recruiting

Early phase I

80

Inclusion Criteria: * Age 3 - \<18 years * First radiological diagnosis of intra-axial, supratentorial 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: * Posterior fossa tumors * Extra-axial tumors such as craniopharyngioma * Germ cell tumor or 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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'DIAGNOSTIC', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 80, 'type': 'ESTIMATED'}}

2023-12-11