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Pediatric Prospective Personalized Immune and Target Identification Trial (PPROSPERITIT)

Primary Purpose

Central Nervous System Neoplasms, Pediatric Cancer, GEN1 Gene Mutation

Status

Recruiting

Phase

Not Applicable

Locations

Czechia

Study Type

Interventional

Intervention

FoundationOneHeme

About this trial

This is an interventional diagnostic trial for Central Nervous System Neoplasms focused on measuring Pediatric oncology, CNS tumors, Personalized medicine

Eligibility Criteria

1 Year - 19 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Sites / Locations

University Hospital BrnoRecruiting
Motol University HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Diagnostic group

Arm Description

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.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Full Information

NCT ID

NCT04859543

First Posted

April 22, 2021

Last Updated

April 23, 2021

Sponsor

Masaryk University

Collaborators

Brno University Hospital, Roche s.r.o.

1. Study Identification

Unique Protocol Identification Number

NCT04859543

Brief Title

Pediatric Prospective Personalized Immune and Target Identification Trial

Acronym

PPROSPERITIT

Official Title

Prospective, Interventional Diagnostic, Multicenter, Non-treatment Clinical Study Identifying Specific Molecular Changes by Using Genomic Sequencing Technologies in Refractory/Recurrent or Very High-risk Pediatric CNS Tumors.

Study Type

Interventional

2. Study Status

Record Verification Date

April 2021

Overall Recruitment Status

Recruiting

Study Start Date

February 1, 2021 (Actual)

Primary Completion Date

December 1, 2024 (Anticipated)

Study Completion Date

December 31, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Masaryk University

Collaborators

Brno University Hospital, Roche s.r.o.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

Keywords

Pediatric oncology, CNS tumors, Personalized medicine

7. Study Design

Primary Purpose

Diagnostic

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

80 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Diagnostic group

Arm Type

Other

Arm Description

Intervention Type

Device

Intervention Name(s)

FoundationOneHeme

Intervention Description

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.

Primary Outcome Measure Information:

Title

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

Description

Evaluation of the feasibility of FoundationOneHeme

Time Frame

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

19 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Kristyna Noskova, PhD

Phone

00 420 549 496 147

noskova@med.muni.cz

First Name & Middle Initial & Last Name or Official Title & Degree

Jana Vinklerova, PhD

Phone

00 420 549 497 782

jvinkler@med.muni.cz

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jaroslav Sterba, Prof, MD

Organizational Affiliation

University Hospital Brno

Official's Role

Principal Investigator

Facility Information:

Facility Name

University Hospital Brno

City

Brno

ZIP/Postal Code

62500

Country

Czechia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Pavel Tinka, MD

Phone

00420 532 234 511

tinka.pavel@fnbrno.cz

First Name & Middle Initial & Last Name & Degree

Klara Vejmelkova, MD

Phone

00420 532 234 511

vejmelkova.klara@fnbrno.cz

First Name & Middle Initial & Last Name & Degree

Jaroslav Sterba, Prof., MD

First Name & Middle Initial & Last Name & Degree

Pavel Tinka, MD

First Name & Middle Initial & Last Name & Degree

Zdenek Pavelka, MD

First Name & Middle Initial & Last Name & Degree

Michal Kyr, MD, PhD

Facility Name

Motol University Hospital

City

Prague

Country

Czechia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Michal Zapotocky, MD, PhD

Phone

00 420 224 436 401

michal.zapotocky@fnmotol.cz

First Name & Middle Initial & Last Name & Degree

David Sumerauer, MD, PhD

Phone

00 420 224 436 401

david.sumerauer@fnmotol.cz

First Name & Middle Initial & Last Name & Degree

Michal Zapotocky, MD, PhD

First Name & Middle Initial & Last Name & Degree

David Sumerauer, MD, PhD

12. IPD Sharing Statement

Plan to Share IPD

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Pediatric Prospective Personalized Immune and Target Identification Trial

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