Clinical trial to evaluate the effects of chemotherapy with anthracycline, after radiotherapy, in a group of pediatric and young adult patients with glioblastoma

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Azienda Ospedaliera Universitaria Meyer IRCCS

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

21 Jun 2022 → ongoing

Decision date (initial)

2025-01-13

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-519135-41-00

EudraCT number

2020-005131-74

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Efficacy

To evaluate the safety and tolerability of prolonged administration of doxorubicin in combination with radiotherapy and temozolomide in pediatric and young adult patients with gliobastoma

Secondary objectives 1

1. Evaluate the effectiveness of treatment by determining event-free survival (EFS), disease progression (PFS), and overall survival (OS)

Conditions and MedDRA coding

Glioblastoma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Patients with histological-molecular diagnosis according to WHO 2016 classification: Glioblastoma IDH-wildtype (9440/3), giant cell glioblastoma (9441/3), gliosarcoma (9442/3), epithelioid glioblastoma (9440/3), IDH glioblastoma mutant (9445/3), NOS glioblastoma (9440/3), diffuse astrocytoma (9400/3), diffuse midline glioma H3 K27M mutated, including multifocal, metastatic, or previously untreated gliomatosis cerebri contextes (with chemo and radiotherapy) or treated only surgically (total, almost partial, partial, biopsy).
2. Males and females between the ages of 3 and 30
3. Life expectancy > or = 12 months
4. Karnofsky/Lansky > o = 80%
5. Adequate haematological function: Absolute white blood cell count >= 2.0 x 10^9 /l; Hemoglobin >= 10 g/dl; Platelet count >= 50 x 109/l
6. Adequate liver function: Total bilirubin <= 2.5 x ULN; ALT / AST <= 5.0 x ULN
7. Adequate renal function: Serum creatinine <= 1.5 x ULN
8. Written informed consent from the patient, parents or legal guardians
9. Patient availability during treatment and ability to comply with the protocol

Exclusion criteria 5

1. Evidence of any other serious illness or condition that is a contraindication to study therapy (e.g. severe mental retardation, severe cerebral palsy, severe congenital syndromes, heart disease)
2. Perform a 1st line chemotherapy cycle at the same time as the start of the study
3. Simultaneous participation in other research projects
4. State of pregnancy or breastfeeding
5. Use of inadequate contraceptive methods

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary safety endpoint is defined as: - Time to early discontinuation of the experimental treatment with Doxorubicin - Percentage of subjects with Serious Adverse Events (SAE) that lead to withdrawal from the study - Percentage of SAE - Mortality due to adverse events - Proportion of early discontinuation of the experimental treatment with Doxorubicin

Secondary endpoints 4

1. Event-free survival (EFS) that is the time between the date of enrollment and the date of occurrence of one of the following events: disease progression, any new lesions, clinical deterioration due to the tumor; failure to return for evaluation following death or deterioration of conditions.
2. Overall survival (OS) defined as the time between the date of enrollment and the date of death from any cause
3. Progression-free survival (PFS) defined as the time between the date of enrollment and the date of progression according to modified RANO criteria
4. Proportion of treatment responder (CR, complete responder; PR, partial responder; SD, stable disease; PD, disease progression) according to modified RANO criteria

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Azienda Ospedaliera Universitaria Meyer IRCCS

Sponsor organisation

Azienda Ospedaliera Universitaria Meyer IRCCS

Address

Viale Gaetano Pieraccini 24

City

Florence

Postcode

50139

Country

Italy

Scientific contact point

Organisation

Azienda Ospedaliera Universitaria Meyer IRCCS

Contact name

Clinical Trial Office

Public contact point

Organisation

Azienda Ospedaliera Universitaria Meyer IRCCS

Contact name

Clinical Trial Office

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 10 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications