Pediatric Prospective Personalized Immune and Target Identification Trial

NCT ID: NCT04859543

Last Updated: 2021-04-26

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: NA
• Total Enrollment: 80 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-02-01
• Study Completion Date: 2024-12-31

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.

Detailed Description

PPROSPERITIT will identify specific molecular changes by using genomic sequencing technologies in refractory/recurrent or very high-risk pediatric CNS tumors. The study will employ an analytically validated comparison of a selection of targeted agents/immune therapies on the basis of commercially available comprehensive genomic profiling FoundationOneHeme panel (F1Heme, comprising DNA and RNA analysis) vs selection of agents based on more complex DNA/RNA/Protein based analyses. This will be coupled to a computer algorithm that uses preexisting definitions and prioritization of target-agent pairs to assign patients by actionable mutation results to a targeted treatment. The selection of targeted agents will be performed by a multidisciplinary molecular tumor board, but the recommended treatment will not be a part of the PPROSPERTIT study.

Conditions

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: DIAGNOSTIC
• Blinding Strategy: NONE

Study Groups

Diagnostic group

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.

Group Type: OTHER

FoundationOneHeme

Intervention Type: 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.

Interventions

FoundationOneHeme

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.

Intervention Type: DEVICE

Eligibility Criteria

Inclusion Criteria

1. 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.

2. The subject is male or female, aged 1 - 19 years

3. The subject must have a histologically proven recurrent/ refractory or very high-risk CNS tumors

4. Patients must be in good overall physical condition, which allows tumor biopsy

5. Patients must have a life expectancy of at least 3 months.

6. 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.

7. Patients must be accessible for follow-up.

Exclusion Criteria

1. Patients with known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.

2. Pregnant and/or breastfeeding women, if applicable

3. No intention to treat the patient.

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 19 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Brno University Hospital

OTHER

Sponsor Role: collaborator

Roche s.r.o.

UNKNOWN

Sponsor Role: collaborator

Masaryk University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Jaroslav Sterba, Prof, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital Brno

Locations

University Hospital Brno

Brno, , Czechia

Site Status: RECRUITING

Motol University Hospital

Prague, , Czechia

Site Status: RECRUITING

Countries

Czechia

Central Contacts

Kristyna Noskova, PhD

Role: CONTACT

noskova@med.muni.cz

00 420 549 496 147

Jana Vinklerova, PhD

Role: CONTACT

jvinkler@med.muni.cz

00 420 549 497 782

Facility Contacts

Pavel Tinka, MD

Role: primary

tinka.pavel@fnbrno.cz

00420 532 234 511

Klara Vejmelkova, MD

Role: backup

vejmelkova.klara@fnbrno.cz

00420 532 234 511

Michal Zapotocky, MD, PhD

Role: primary

michal.zapotocky@fnmotol.cz

00 420 224 436 401

David Sumerauer, MD, PhD

Role: backup

david.sumerauer@fnmotol.cz

00 420 224 436 401

Other Identifiers

KDO-2019-001

Identifier Type: -

Identifier Source: org_study_id