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CompletedNCT03276702Updated Jun 6, 2023

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

An observational study in Tumor, Solid, sponsored by St. Jude Children's Research Hospital. Completed at 1 site in United States. Open to participants aged 2 Years to 21 Years. Per ClinicalTrials.gov, last updated 2023-06-06.

Sponsored by St. Jude Children's Research Hospital · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
8
Ages
2 Years to 21 Years
Sex
All
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Study 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.
Read the 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.

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Conditions studied

  • Tumor, Solid

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03

Who can participate

Ages eligible
2 Years to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

St. Jude Children's Research Hospital patient with a solid tumor malignancy that is progressive or recurrent.

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

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.
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Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
8 participants (actual)
Patient registry
No
Biospecimen retention
Samples with dna

Groups and cohorts

  • 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.

    Procedure: Re-biopsy · Other: Questionnaire

Interventions

  • ProcedureRe-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.

    Also known as: Tumor tissue biopsy

  • OtherQuestionnaire

    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.

    Also known as: Assessment

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What researchers measure

Primary outcomes

  1. Percent of good quality tumor samples obtained

    Good quality of the sampled tumor is defined as greater than 50% viable tumor from which at least 2 µg DNA and 1 µg RNA can be extracted.

    Time frame: Once at study enrollment

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Study locations

1 site
  • St. Jude Children's Research Hospital
    Memphis, Tennessee 38105, United States
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References and documents

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Registry details

Key details

Study ID
NCT03276702
Lead sponsor
St. Jude Children's Research Hospital
Responsible party
Sponsor
First posted
Sep 8, 2017
Start date
Dec 13, 2017
Primary completion
Jul 31, 2019
Completion
Jul 31, 2019
Last update
Jun 6, 2023

Study contacts

Alberto Pappo, MD
principal investigator · St. Jude Children's Research Hospital

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Jun 2023. You cannot join it, but the record below documents what was studied.

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