Clinical trial NCT03276702: Tumor Rebiopsy in Children and Adolescents With Recurrent or Progressive Solid Malignancies
Official title: A Protocol for Prospectively Collecting Fresh Tumor Tissue in Children and Adolescents With Recurrent or Progressive Solid Malignancies
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Tumor, Solid
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
First posted: 2017-09-08
Enrollment: 8
Sex: ALL
Minimum age (years): 2
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: St. Jude Children's Research Hospital patient with a solid tumor malignancy that is progressive or recurrent.

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.

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.

Primary outcomes:
measure: Percent of good quality tumor samples obtained
timeFrame: Once at study enrollment
Secondary outcomes:
Other outcomes:
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Alberto Pappo, MD
role: PRINCIPAL_INVESTIGATOR
References: