Diagnostic Testing for Pediatric Leukemia

Recruiting at 178 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: LLS PedAL Initiative, LLC

Disqualifiers: Over 22 years, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

960 Participants Needed

Trial Summary

What is the purpose of this trial?

This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and blood from patients with leukemia that has come back after treatment or is difficult to treat may provide information about the patient's leukemia that is important when deciding how to best treat it, and may help doctors find better ways to diagnose and treat leukemia in children, adolescents, and young adults.

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications.

What data supports the effectiveness of the treatment Biospecimen Collection for pediatric leukemia?

Research shows that using blood-derived liquid biopsies can help identify specific genetic changes in children's tumors, which can guide treatment decisions. This approach has been successful in identifying targetable mutations in pediatric patients with high-risk solid tumors, suggesting it could be useful for leukemia as well. ^{1 2 3 4 5}

Is blood-derived liquid biopsy safe for children with cancer?

The study on blood-derived liquid biopsy (LB) for children with high-risk cancers did not report any safety issues, suggesting it is generally safe for use in pediatric patients. ^{1 6 7 8 9}

How does the Biospecimen Collection treatment for pediatric leukemia differ from other treatments?

Biospecimen Collection is unique because it involves collecting samples like blood or tissue to better understand the genetic makeup of leukemia, rather than directly treating the disease. This approach can help identify specific genetic changes in leukemia, which may guide more personalized treatment strategies in the future. ^{4 9 10 11 12}

Research Team

Michele S Redell

Principal Investigator

Children's Oncology Group

Eligibility Criteria

This trial is for children, adolescents, and young adults under 22 with leukemia that's come back or is hard to treat. They must have consent from parents/guardians if needed, meet FDA and NCI human study requirements, and have specific types of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), mixed phenotype acute leukemia (MPAL), juvenile myelomonocytic leukemia (JMML), or relapsed ALL.

Inclusion Criteria

This criterion means that the patient needs to meet certain conditions, but the specific conditions are not provided.

I am under 22 years old.

My leukemia linked to Down syndrome has returned or is not responding to treatment.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Biospecimen Collection

Patients undergo collection of blood and/or bone marrow samples at baseline, end of treatment cycle(s), and at relapse/refractory disease status (if applicable)

Varies

Follow-up

Participants are monitored every 3 months for 2 years, and then every 6 months for 3 years after completion of the study

5 years

Treatment Details

Interventions

• Biospecimen Collection

Trial Overview The trial involves collecting bone marrow and blood samples from patients to understand the clinical and biological characteristics of their acute leukemias. This information may help determine eligibility for other sub-trials aimed at finding better ways to diagnose and treat pediatric leukemia.

Participant Groups

Group I: Screening (biospecimen collection) Experimental Treatment 1 Intervention

Patients undergo collection of blood and/or bone marrow samples at baseline, end of treatment cycle(s), and at relapse/refractory disease status (if applicable).

Who Is Running the Clinical Trial?

LLS PedAL Initiative, LLC

Lead Sponsor

Trials

4

Recruited

1,100+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Children's Oncology Group

Collaborator

Trials

467

Recruited

241,000+

Findings from Research

A study involving 45 pediatric patients with relapsing or refractory solid tumors demonstrated that liquid biopsy using Foundation One® Liquid CDx is a feasible method for molecular profiling, successfully identifying targetable mutations in 25 samples.

Out of the patients analyzed, 6 received treatment based on liquid biopsy results, leading to one patient achieving stable disease for eight months, highlighting the potential of liquid biopsies in guiding treatment decisions in pediatric oncology.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Blood-Derived Liquid Biopsies Using Foundation One&#174; Liquid CDx for Children and Adolescents with High-Risk Malignancies: A Monocentric Experience. Cahn, F., Revon-Riviere, G., Min, V., et al.[2022]

Circulating tumor DNA can be detected in the blood of pediatric cancer patients using advanced technologies, which may enhance cancer diagnosis and treatment monitoring.

The review highlights the need to adapt liquid biopsy techniques to the specific genomic characteristics of pediatric tumors and addresses challenges in implementing these tests in clinical practice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of circulating tumor DNA in pediatric solid tumors: The promise of liquid biopsies. Abbou, SD., Shulman, DS., DuBois, SG., et al.[2020]

A centralized biobank for pediatric cancer in Norway has been successfully established, collecting approximately 12,000 samples from 510 patients since its inception in 2017, with a remarkable 96% consent rate among newly diagnosed patients.

The biobank employs standardized procedures for sample collection, processing, and storage, ensuring high-quality biological material is available for research on rare pediatric malignancies, which is crucial due to their unique genomic characteristics.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Norwegian childhood cancer biobank. Hermansen, JU., Wojcik, DM., Robinson, N., et al.[2022]

The OncoKids panel is a next-generation sequencing assay that effectively detects various diagnostic, prognostic, and therapeutic markers in pediatric cancers, using only 20 ng of DNA and RNA from diverse sample types.

Validated on 192 clinical samples, the OncoKids panel demonstrated strong analytical sensitivity and reproducibility, supporting its use for routine clinical testing in a wide range of pediatric malignancies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

OncoKids: A Comprehensive Next-Generation Sequencing Panel for Pediatric Malignancies. Hiemenz, MC., Ostrow, DG., Busse, TM., et al.[2019]

In a study involving 75 pediatric patients with recurrent or refractory solid tumors, genomic analysis revealed actionable alterations in 60.9% of cases, indicating a significant potential for targeted therapies.

The study demonstrated that research biopsies are feasible and safe, with a low complication rate, allowing for effective molecular analysis to guide treatment decisions in advanced pediatric cancers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Molecular Screening for Cancer Treatment Optimization (MOSCATO-01) in Pediatric Patients: A Single-Institutional Prospective Molecular Stratification Trial. Harttrampf, AC., Lacroix, L., Deloger, M., et al.[2018]

The ExtractEHR automated package effectively identified and graded laboratory adverse events in a cohort of 1,077 children with acute myeloid leukaemia and acute lymphoblastic leukaemia, demonstrating its scalability and accuracy in capturing adverse event rates.

The study found that ExtractEHR reported significantly higher rates of laboratory adverse events compared to those manually reported in clinical trials, suggesting that automated systems can enhance the understanding of chemotherapy risks in pediatric patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rates of laboratory adverse events by course in paediatric leukaemia ascertained with automated electronic health record extraction: a retrospective cohort study from the Children's Oncology Group. Miller, TP., Getz, KD., Li, Y., et al.[2023]

A pediatric biobank has been successfully implemented, enrolling 4900 patients and linking biological specimens to electronic health record data, which enhances research capabilities in diverse clinical settings.

The biobank addresses specific challenges in pediatric research, such as the need for re-consent when patients turn 18 and the potential to include family members, which can help in studying rare diseases more effectively.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Development of the Precision Link Biobank at Boston Children's Hospital: Challenges and Opportunities. Bourgeois, FT., Avillach, P., Kong, SW., et al.[2020]

The University of Chicago has developed a biobanking and sequencing program aimed at understanding the genetic factors contributing to cancer in children, which will help tailor anticancer therapies based on individual genetic profiles.

This program will also analyze how genomic alterations in tumors change over time and between different tumor sites, potentially improving treatment strategies and outcomes for pediatric cancer patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Establishing a translational genomics infrastructure in pediatric cancer: the GREAT KIDS experience. Pinto, N., Volchenboum, SL., Skol, AD., et al.[2019]

The Swedish Childhood Tumor Biobank (BTB) has collected over 1100 fresh-frozen tumor samples from pediatric patients, providing high-quality biospecimens and genomic data that enhance the understanding of childhood tumors.

Bioinformatics analyses of next-generation sequencing data from 82 brain tumors revealed that 79 tumors had their diagnoses confirmed or clarified, and 68 patients had known or likely driver mutations detected, highlighting the potential of genomic data to improve patient management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Swedish childhood tumor biobank: systematic collection and molecular characterization of all pediatric CNS and other solid tumors in Sweden. Díaz de Ståhl, T., Shamikh, A., Mayrhofer, M., et al.[2023]

In a study of 24 children with B-lineage acute lymphoblastic leukemia (ALL), the absence of detectable residual leukemia in bone marrow at the end of chemotherapy did not guarantee a cure, as some patients still experienced relapse later.

Among the patients who relapsed, those with detectable residual leukemia had a higher risk of early relapse, indicating the need for ongoing monitoring even after treatment completion to predict potential relapses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Molecular residual disease status at the end of chemotherapy fails to predict subsequent relapse in children with B-lineage acute lymphoblastic leukemia. Ito, Y., Wasserman, R., Galili, N., et al.[2017]

The ALLseq next-generation sequencing panel effectively detects a wide range of genetic alterations in acute lymphoblastic leukemia (ALL), demonstrating 100% sensitivity and specificity, making it a reliable tool for clinical use.

This panel can provide clinically relevant information for over 83% of pediatric patients with ALL, aiding in diagnosis, risk assessment, and treatment decisions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Design and Validation of a Custom Next-Generation Sequencing Panel in Pediatric Acute Lymphoblastic Leukemia. Gil, JV., Such, E., Sargas, C., et al.[2023]

Liquid biopsy is an innovative method that uses blood samples to detect cancer by analyzing shed biomolecules, offering a less invasive way to monitor the disease over time.

This review emphasizes the importance of adapting liquid biopsy techniques specifically for pediatric cancers, which can enhance research quality and efficiency in understanding and treating these conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Roadmap to Liquid Biopsy Biobanking from Pediatric Cancers-Challenges and Opportunities. Kahana-Edwin, S., Cain, LE., Karpelowsky, J.[2021]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Blood-Derived Liquid Biopsies Using Foundation One&#174; Liquid CDx for Children and Adolescents with High-Risk Malignancies: A Monocentric Experience. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Assessment of circulating tumor DNA in pediatric solid tumors: The promise of liquid biopsies. [2020]

3. United States pubmed.ncbi.nlm.nih.gov

The Norwegian childhood cancer biobank. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

OncoKids: A Comprehensive Next-Generation Sequencing Panel for Pediatric Malignancies. [2019]

5. United States pubmed.ncbi.nlm.nih.gov

Molecular Screening for Cancer Treatment Optimization (MOSCATO-01) in Pediatric Patients: A Single-Institutional Prospective Molecular Stratification Trial. [2018]

6. England pubmed.ncbi.nlm.nih.gov

Rates of laboratory adverse events by course in paediatric leukaemia ascertained with automated electronic health record extraction: a retrospective cohort study from the Children's Oncology Group. [2023]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Development of the Precision Link Biobank at Boston Children's Hospital: Challenges and Opportunities. [2020]

8. England pubmed.ncbi.nlm.nih.gov

Establishing a translational genomics infrastructure in pediatric cancer: the GREAT KIDS experience. [2019]

9. England pubmed.ncbi.nlm.nih.gov

The Swedish childhood tumor biobank: systematic collection and molecular characterization of all pediatric CNS and other solid tumors in Sweden. [2023]

10. United States pubmed.ncbi.nlm.nih.gov

Molecular residual disease status at the end of chemotherapy fails to predict subsequent relapse in children with B-lineage acute lymphoblastic leukemia. [2017]

11. Switzerland pubmed.ncbi.nlm.nih.gov

Design and Validation of a Custom Next-Generation Sequencing Panel in Pediatric Acute Lymphoblastic Leukemia. [2023]

12. United States pubmed.ncbi.nlm.nih.gov

Roadmap to Liquid Biopsy Biobanking from Pediatric Cancers-Challenges and Opportunities. [2021]