Evaluating anthracycline treatment after radiotherapy for glioblastoma in young patients

Interventional, Single-arm, Open-label Open-label, Phase II Trial to Evaluate the Role of Anthracycline Infusion After Radio Therapy (RT) in Pediatric and Young Adults With Glioblastoma (pGBM).

This study tests if adding a heart-safe version of doxorubicin to radiotherapy and temozolomide can help young patients with glioblastoma do better than with standard treatments.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 3 Years to 30 Years
• Sex: All
• Sponsor: Meyer Children's Hospital IRCCS (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Florence)
• Trial ID: NCT06297512 on ClinicalTrials.gov

What this trial studies

This study investigates the safety and effectiveness of combining doxorubicin with whole body radiotherapy and temozolomide in pediatric and young adult patients diagnosed with glioblastoma. The research aims to determine if this combination can improve treatment outcomes compared to standard therapies. The study focuses on patients who have not received prior chemotherapy or radiotherapy, ensuring a clear assessment of the new treatment's impact. By utilizing continuous infusion of doxorubicin, the study also aims to minimize potential cardiotoxicity associated with traditional bolus administration.

Who should consider this trial

Good fit: Ideal candidates include males and females aged 3 to 30 years with specific types of glioblastoma who have not received prior treatment.

Not a fit: Patients with glioblastoma who have previously undergone chemotherapy or radiotherapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment approach could lead to improved survival rates and quality of life for young patients with glioblastoma.

How similar studies have performed: While the use of anthracyclines in glioblastoma treatment is supported by preliminary studies, this specific combination approach is novel and has not been extensively tested.

Eligibility criteria

Inclusion Criteria:

* Patients with histological-molecular diagnosis according to WHO 2016 classification: IDH-wildtype glioblastoma (9440/3), giant cell glioblastoma (9441/3), gliosarcoma (9442/3), epithelioid glioblastoma (9440/3), IDH-mutated glioblastoma (9445/3), glioblastoma NOS (9440/3), diffuse astrocytoma (9400/3), diffuse midline glioma H3 K27M mutated, including multifocal, metastatic or gliomatosis cerebri pictures of first diagnosis Not previously treated (with chemo and radiotherapy) or treated only surgically (total, near partial, partial, biopsy).
* Males and females between the ages of 3 and 30 years old
* Life expectancy ≥ 12 months
* karnofsky/Lansky ≥ 80 %
* Adequate hematologic function: Absolute leukocyte count ≥ 2.0 x 109/l, Hemoglobin ≥ 10 g/dl, Platelet count ≥ 50 x 109/l
* Adequate liver function: Total bilirubin ≤ 2.5 x ULN, ALT/AST ≤ 5.0 x ULN
* Adequate renal function:Serum creatinine ≤ 1.5 x ULN
* Written informed consent from the patient, parents or legal guardians
* Patient's willingness during treatment and ability to comply with the protocol

Exclusion Criteria:

* Evidence of any other serious disease or condition that is a contraindication to study therapy (e.g. severe mental retardation, severe cerebral palsy, severe syndromes congenital syndromes, heart disease)
* Performance of a course of 1st-line chemotherapy at the same time as study initiation
* Concurrent participation in other research projects
* Pregnancy or lactation status
* Use of inappropriate contraceptive methods

Where this trial is running

Florence

• Meyer Children's Hospital IRCCS — Florence, Italy (Recruiting)

Study contacts

• Principal investigator: Iacopo Sardi — Meyer Children's Hospital IRCCS
• Study coordinator: Iacopo Sardi

Conditions Glioblastoma