Blood biomarkers for treatment-related brain injury in children with brain tumors

Biomarkörer för hjärnskada Hos Barn Med hjärntumör

This project will test whether blood plasma biomarkers can show treatment-related brain injury in children with primary brain tumors by comparing those who receive radiotherapy, those who do not, and healthy children of the same age and sex.

Quick facts

• Study type: Observational
• Enrollment: 560 (estimated)
• Ages: N/A to 17 Years
• Sex: All
• Sponsor: Uppsala University (Academic / other)
• Drugs / interventions: radiation, chemotherapy
• Locations: 1 site (Uppsala)
• Trial ID: NCT07381959 on ClinicalTrials.gov

What this trial studies

This is an observational, longitudinal biomarker study enrolling children aged 0–17 with primary brain tumors and age- and sex-matched healthy controls. Researchers will collect plasma samples before surgery, shortly after surgery, at multiple follow-up points up to 36 months, and more frequently during any radiotherapy, to profile proteins and extracellular vesicles. Results from children treated with surgery, chemotherapy, and radiotherapy will be compared with radiation-naive patients and healthy controls to identify treatment-specific signals. The study uses proteomics and extracellular vesicle analyses to look for sensitive, minimally invasive markers of treatment-induced brain injury.

Who should consider this trial

Good fit: Children aged 0–17 with a primary brain tumor treated or diagnosed at Uppsala University Hospital or another Swedish university hospital (including groups referred for radiotherapy and those not referred), and healthy children aged 0–17 for the control group.

Not a fit: Adults, children without primary brain tumors, or patients unable to provide the planned plasma samples (including those treated outside participating centers or unable to attend follow-up) are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, the findings could enable earlier, less invasive detection of treatment-related brain injury and guide safer, more personalized treatment plans for children.

How similar studies have performed: Related adult and limited pediatric studies using plasma proteomics and extracellular vesicles have shown promising signals for brain injury and radiation effects, but sensitive, validated biomarkers in children remain largely unestablished.

Eligibility criteria

Inclusion Criteria, Group A (treated with radiation):

* Age 0-17 years old, AND
* Diagnosed with primary brain tumor through surgery, biopsy, or other diagnostic method (e.g., for germinoma) at Uppsala University Hospital or at another university hospital in Sweden, AND
* Referred for radiotherapy at Skandion Clinic in Uppsala and/or at their local hospital (in some cases), with/without the addition of other cancer treatments.

Inclusion Criteria, Group B (treated without radiation/radiation naive):

* Age 0-17 years old, AND
* Diagnosed with primary brain tumor through surgery, biopsy, or other diagnostic method (e.g., for optic pathway glioma in neurofibromatosis type 1 and germinoma) at Uppsala University Hospital or at another university hospital in Sweden, AND
* Not referred to radiotherapy and treated with/without the addition of other cancer treatments.

Inclusion Criteria, Group C (healthy control group):

* Age 0-17 years old at time of recruitment

Exclusion Criteria, Groups A + B:

* Diagnosed with a tumor only in the spinal cord (solitary spinal tumor).
* Diagnosed with a tumor considered palliative already at diagnosis (e.g., diffuse intrinsic pontine glioma)

Exclusion Criteria, Group C:

* Have diagnosis of chronic disease that requires continuous medication.

Exclusion Criteria, all groups:

* Unable to provide informed consent due to language difficulties.

Where this trial is running

Uppsala

• Uppsala University — Uppsala, Sweden (Recruiting)

Study contacts

• Principal investigator: Arja Harila, MD, PhD — Uppsala University
• Study coordinator: Christoffer Ehrstedt, MD, PhD

Conditions Brain Tumor; Childhood Cancer; Childhood Brain Tumor; Radiotherapy; Cranial radiation therapy; Plasma biomarkers; Extracellular vesicles; Proteomics