Diagnostic Testing for Pediatric Leukemia

Recruiting at 196 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: PedAL BCU, 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to improve the diagnosis and treatment of leukemia in young patients. Researchers will collect blood and bone marrow samples to study leukemia that is hard to treat or has returned after treatment. The goal is to enhance screening for other treatment trials and discover better methods to help children and young adults with leukemia. Participants under 22 years old with leukemia that is difficult to treat or has recurred may be suitable for this study. As a Phase 1/Phase 2 trial, the research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

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 prior data suggests that this diagnostic testing is safe for pediatric leukemia patients?

Research has shown that taking blood and bone marrow samples is generally safe and well-tolerated. This process, integral to many studies, gathers important information about diseases like leukemia. No major safety concerns have been reported with collecting these samples. They are crucial for helping doctors learn more about treating and diagnosing leukemia, especially when it recurs or is difficult to treat.

Pediatric biobanks, which store these samples, emphasize addressing ethical concerns like consent and data protection, but they report no major safety issues from the collection process itself. Overall, collecting these samples is a routine and safe procedure in research settings. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about this trial because it focuses on a new diagnostic approach for pediatric leukemia through biospecimen collection. Unlike standard testing methods that might rely heavily on imaging or symptom observation, this approach involves collecting blood and bone marrow samples at multiple stages: baseline, end of treatment cycles, and at relapse. This method allows for a more precise and dynamic understanding of the disease's progression and response to treatment. By closely monitoring these samples, researchers hope to improve diagnosis accuracy and tailor treatments more effectively for young patients.

What evidence suggests that this biospecimen collection is effective for diagnosing and treating pediatric leukemia?

Research shows that taking blood and bone marrow samples, as done in this trial, can help identify specific features of leukemia in children. By examining individual cells, researchers can observe the detailed environment of leukemia in young patients. Studies have also found that genetic changes can be spotted early, aiding in the development of more targeted and effective treatments. Real-time genomic analysis, which examines genes in their current state, aids in accurately classifying leukemia, crucial for selecting the right treatment. Overall, using these samples can lead to better diagnosis and treatment strategies for childhood leukemia. ^{4 6 7 8 9}

Who Is on the Research Team?

Michele S Redell

Principal Investigator

Children's Oncology Group

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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?

PedAL BCU, LLC

Lead Sponsor

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+

Published Research Related to This Trial

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]

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]

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]

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]

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]

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 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]

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]

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 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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12048633/

Identification of leukemia-enriched signature through the ... Single-cell transcriptome profiling enables unparalleled characterization of the heterogeneous microenvironment of pediatric leukemias.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5161115/

Childhood Leukemia and Primary Prevention - PMC Perhaps the most straightforward way to obtain pre-diagnostic biospecimens would be to establish a prospective birth cohort and follow leukemia incidence in the ...

3. molecular-cancer.biomedcentral.com molecular-cancer.biomedcentral.com/articles/10.1186/s12943-024-02118-4

Epigenome-wide analysis across the development span of ... This study provides proof-of-concept to detect at birth epigenetic precursors of pediatric pre-B ALL. These alterations were reproducible with different ...

4. nature.com nature.com/articles/s41375-025-02565-y

Real-time genomic characterization of pediatric acute ... Effective treatment of pediatric acute leukemia is dependent on accurate genomic classification, typically derived from a combination of ...

5. nature.com nature.com/articles/s41375-025-02774-5

Clinical experience of using integrated whole genome and ... Our successful implementation of iWGS-WTS in pediatric AML diagnosis demonstrates its potential to streamline resource use, accelerate disease ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9444944/

Rates of Laboratory Adverse Events by Course in Pediatric ... Prevalence of grades 3–4 laboratory adverse events by chemotherapy course ascertained by ExtractEHR for ALL courses

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04726241

NCT04726241 | The Pediatric Acute Leukemia (PedAL) ... This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials.

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9871420/

Pediatric biobanks to enhance clinical and translational ... Pediatric biobanks are helpful for providing biological material for research purposes, addressing ethical and legal issues (ie data protection, consent, etc.)

9. nctnbanks.cancer.gov nctnbanks.cancer.gov/catalog/

NCTN Biospecimen Catalog The information presented here is estimated and for informational purposes only. Please consult the specific biospecimen resource for more detailed ...