Tumor Rebiopsy in Children and Adolescents With Recurrent or Progressive Solid Malignancies
| Status: | Recruiting |
|---|---|
| Healthy: | No |
| Age Range: | 2 - 21 |
| Updated: | 1/5/2019 |
| Start Date: | December 13, 2017 |
| End Date: | May 2023 |
| Contact: | Alberto Pappo, MD |
| Email: | referralinfo@stjude.org |
| Phone: | 866-278-5833 |
A Protocol for Prospectively Collecting Fresh Tumor Tissue in Children and Adolescents With Recurrent or Progressive Solid Malignancies
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.
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.
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.
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.
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.
We found this trial at
1
site
262 Danny Thomas Pl
Memphis, Tennessee 38105
Memphis, Tennessee 38105
(901) 495-3300
Principal Investigator: Alberto Pappo, MD
Phone: 866-278-5833
St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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