Pediatric Prospective Personalized Immune and Target Identification Trial

NCT04859543 | Completed DMC

• 1 other identifier: KDO-2019-001
• Study Type: interventional
• Target: 80
• Locations: 2 countries
• Sites: 3

Brief Summary

PPROSPERITIT is a prospective clinical study assessing the use of comprehensive molecular profiling to define the best matching targeted and immune treatment for relapsed, refractory or very high risk pediatric CNS tumors.

Conditions

Central Nervous System Neoplasms; Pediatric Cancer; GEN1 Gene Mutation; Tumor, Brain

Keywords

Pediatric oncology, CNS tumors, Personalized medicine

Outcome Measures

Primary Outcomes (1)

• The proportion of patients in which F1Heme molecular testing identified at least 1 clinically relevant alteration at the time of MTB decision.

  Evaluation of the feasibility of FoundationOneHeme

  Diagnostic assessment is done within 28 days from enrolment patient in the study.

Study Arms (1)

Diagnostic group

OTHER

Tumor and blood samples will be collected from each patient and broad molecular profiling will be performed. The results of the evaluation of the tumor specimens will determine if the patient's tumor has an actionable mutation for which treatment is available.

Device: FoundationOneHeme

Interventions

FoundationOneHeme DEVICE

Tumor tissue obtained during standard surgery will be subsequently examined histopathologically and the content of cancer cells will be determined. Broad molecular profiling of the tumor; with potential results finding such molecular changes, for which the specific targeted anti-tumor treatment will be performed.

Diagnostic group

Eligibility Criteria

• Age: 1 Year - 19 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patient Informed consent form must be appropriately obtained under the applicable local and regulatory requirements. Each patient must sign a consent form prior the enrollment to document their willingness to participate.
• The subject is male or female, aged 1 - 19 years
• The subject must have a histologically proven recurrent/ refractory or very high-risk CNS tumors
• Patients must be in good overall physical condition, which allows tumor biopsy
• Patients must have a life expectancy of at least 3 months.
• Patients must have a tumor amenable to image-guided or direct vision biopsy and be willing and able to undergo a tumor biopsy and/or blood taking for molecular profiling.
• Patients must be accessible for follow-up.

You may not qualify if:

• Patients with known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
• Pregnant and/or breastfeeding women, if applicable
• No intention to treat the patient.

Sponsors & Collaborators

• Masaryk University: lead
• Brno University Hospital: collaborator
• Roche s.r.o.: collaborator

Study Sites (3)

Medical University of Vienna

Vienna, Spitalgasse 23,, 1090, Austria

Location

Motol University Hospital

Prague, V Úvalu 84,, 150 06, Czechia

Location

University Hospital Brno

Brno, Černopolní 9,, 613 00, Czechia

Location

MeSH Terms

Conditions

Central Nervous System Neoplasms; Neoplasms; Brain Neoplasms

Condition Hierarchy (Ancestors)

Nervous System Neoplasms; Neoplasms by Site; Nervous System Diseases; Brain Diseases; Central Nervous System Diseases

Study Officials

• Jaroslav Sterba, Prof, MD

  Brno University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 22, 2021
• First Posted: April 26, 2021
• Study Start: February 2, 2021
• Primary Completion: December 10, 2024
• Study Completion: December 10, 2024
• Last Updated: May 1, 2026

Record last verified: 2021-04

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (2); AU (1)