Clinical trial NCT06297512: Evaluate the Role of Anthracycline After Radio Therapy in Patients With Glioblastoma (pGBM).
Official title: 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).
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Glioblastoma
Lead sponsor: Meyer Children's Hospital IRCCS
Sponsor class: OTHER
First posted: 2024-03-07
Enrollment: 20
Sex: ALL
Minimum age (years): 3
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Glioblastoma (GBM) and diffuse intrinsic bridge gliomas (DIPG) only the most aggressive forms of cancer, and their prognosis remains bleak. Currently, the standard of treatment is TMZ concomitant with radiotherapy, and, at the end of combined treatment, as adjuvant therapy. In vitro and in vivo experimental studies have suggested that anthracyclines are effective antineoplastics for the treatment of gliomas. In patients with solid tumors treated with anthracyclines, continuous infusion administration compared with bolus administration has been shown to provide a better safety profile especially with regard to cardiotoxicity. Based on this evidence, this study aims to evaluate the safety and antitumor activity of combined treatment with Dox, WBRT (whole body radiotherapy), and TMZ in pediatric and young adult patients affected by GMB


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

Primary outcomes:
measure: Evaluation prolonged Dox
timeFrame: through study completion, an average of 1 year
measure: Percentage of Withdrawal from the study rate
timeFrame: through study completion, an average of 1 year
measure: Percentage of SAEs
timeFrame: through study completion, an average of 1 year
measure: Mortality rate
timeFrame: through study completion, an average of 1 year
measure: Early discontinuation of dox treatment rate
timeFrame: through study completion, an average of 1 year
Secondary outcomes:
measure: Event-free survival (EFS), disease progression (PFS), and overall survival (OS)
timeFrame: through study completion, an average of 1 year
Other outcomes:
Locations:
city: Florence
country: Italy
facility: Meyer Children's Hospital IRCCS
Study officials:
affiliation: Meyer Children's Hospital IRCCS
name: Iacopo Sardi
role: PRINCIPAL_INVESTIGATOR
References: