Pediatric Prospective Personalized Immune and Target Identification Trial

Pediatric Cancer Clinical Trial

— 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.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04859543
• Other study ID #: KDO-2019-001
• Status: Recruiting
• Phase: N/A
• Start date: February 1, 2021
• Est. completion date: December 31, 2024

Study information

• Verified date: April 2021
• Source: Masaryk University
• Contact: Kristyna Noskova, PhD
• Phone: 00 420 549 496 147
• Email: noskova[@]med.muni.cz
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 80
• Est. completion date: December 31, 2024
• Est. primary completion date: December 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 19 Years

Eligibility

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.

Related Conditions & MeSH terms

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

Intervention

Device:
• 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.

Locations

Country	Name	City	State
Czechia	University Hospital Brno	Brno
Czechia	Motol University Hospital	Prague

Sponsors (3)

Lead Sponsor	Collaborator
Masaryk University	Brno University Hospital, Roche s.r.o.

Country where clinical trial is conducted

Czechia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.