Tumor Rebiopsy in Children and Adolescents With Recurrent or Progressive Solid Malignancies

NCT ID: NCT03276702

Last Updated: 2023-06-06

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 8 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2017-12-13
• Study Completion Date: 2019-07-31

Brief Summary

It has been shown that the genomic landscape of tumors can dramatically change at the time of disease progression. This suggests that therapies aimed at treating the tumor at diagnosis may not be relevant at the time of relapse. Obtaining fresh tissue at the time of relapse could facilitate the identification of potential targets that have developed through mutational evolution of the primary tumor and would provide an unprecedented opportunity to expand the library of patient derived xenografts (PDX) that are available for comprehensive preclinical testing in the setting of relapsed disease.

The purpose of this observational study is to prospectively collect fresh tumor tissue from pediatric patients with solid tumors who have recurrent or progressive disease and submit this tissue for comprehensive genomic analysis using clinically available CLIA-certified platforms that include WGS, WES and RNAseq, and to assess the reasons for a patient and/or a parent for making a decision to proceed with or refuse a re-biopsy procedure in the presence of a recurrent or progressive solid tumor. The data obtained will be used to develop new novel therapies that incorporate new pharmacological agents in pediatric solid tumors.

PRIMARY OBJECTIVES:

• To prospectively collect fresh tumor tissue from pediatric patients with solid tumors who have recurrent or progressive disease and submit this tissue for comprehensive genomic analysis.
• To assess the reasons a patient and/or a parent make the decision to proceed with or refuse a re-biopsy procedure in the presence of a recurrent or progressive solid tumor.

SECONDARY OBJECTIVES:

• To estimate refusal and acceptance rates for re-biopsy and the parental/patient attitudes towards a re-biopsy.
• To estimate the incidence of complications associated with biopsy.
• To estimate the percentage of procedures that fail to obtain adequate tissue for genomic analysis.

Detailed Description

This is a prospective, non-therapeutic study with two components: biopsy of tumor for genomic studies and prospective assessment of parental factors related to participation. All patients at St. Jude Children's Research Hospital (SJCRH) who meet the eligibility criteria will be approached for study participation. The re-biopsy is not mandatory and will only be performed after the patient and family have consented. A questionnaire to assess the impact and perception of an optional/research only biopsy will be administered following the decision regarding re-biopsy. For those consenting to the re-biopsy, approximately 4-8 weeks after biopsy, the family and patient will be asked to complete a second questionnaire to assess decisional regret.

Participants may repeat participation in this trial with subsequent relapses, provided informed consent is obtained prior to each re-biopsy procedure. For those participants who consent to repeat participation, all protocol procedures will be repeated.

Conditions

Tumor, Solid

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Participants

St. Jude Children's Research Hospital patients with relapsed or progressive solid tumor will be asked to complete a questionnaire on two occasions and optional re-biopsy of tumor tissue.

Re-biopsy

Intervention Type: PROCEDURE

For those participants who agree, a biopsy of their tumor to obtain tissue for genetic analysis will be done at the time of relapse or disease progression.

Questionnaire

Intervention Type: OTHER

Parents or patients will be asked to complete a questionnaire to assess the impact and perception of an optional/research only biopsy. A second questionnaire will be administered approximately 4-8 weeks after biopsy to assess decisional regret.

Interventions

Re-biopsy

For those participants who agree, a biopsy of their tumor to obtain tissue for genetic analysis will be done at the time of relapse or disease progression.

Intervention Type: PROCEDURE

Questionnaire

Parents or patients will be asked to complete a questionnaire to assess the impact and perception of an optional/research only biopsy. A second questionnaire will be administered approximately 4-8 weeks after biopsy to assess decisional regret.

Intervention Type: OTHER

Other Intervention Names

Tumor tissue biopsy; Assessment

Eligibility Criteria

Inclusion Criteria

• Age 2-21 years
• Patient has been diagnosed with a solid tumor malignancy that is recurrent or progressive and for whom there is no fresh tumor specimen available.
• Availability of gross disease amenable to biopsy.
• Karnofsky ≥ 50% for patients >16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age.

Exclusion Criteria

• Diagnosis of a non-solid tumor malignancy.
• Patient with known coagulopathy that requires replacement therapy.
• Patient with anesthesia risk that would place the patient at a higher than expected risk for complications (ASA PS4 and ASA PS5).
• Karnofsky or Lansky performance score of < 50.
• BMI for age > 95th percentile.
• Refusal or inability to provide written informed consent according to institutional guidelines.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

St. Jude Children's Research Hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Alberto Pappo, MD

Role: PRINCIPAL_INVESTIGATOR

St. Jude Children's Research Hospital

Locations

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Countries

United States

Related Links

http://www.stjude.org

St. Jude Children's Research Hospital

http://www.stjude.org/protocols

Clinical Trials Open at St. Jude

Other Identifiers

REBIOP

Identifier Type: -

Identifier Source: org_study_id