What is the mechanism of action of this treatment?

Peripheral Primitive Neuroectodermal Tumor (PPNET) treatments commonly include chemotherapy, radiation therapy, and surgery. Chemotherapy agents like vincristine, ifosfamide, and doxorubicin damage cancer cell DNA, preventing replication and causing cell death. Radiation therapy uses high-energy particles to destroy cancer cells, and surgery aims to remove the tumor. The study of ctDNA as a prognostic biomarker is crucial as it aids in early relapse detection and treatment response monitoring, allowing for more personalized and timely interventions.

What side effects are associated with this type of intervention?

Peripheral Primitive Neuroectodermal Tumor (PPNET) treatments, similar to those in the ctDNA Detection trial, often involve multi-agent chemotherapy regimens such as vincristine, doxorubicin, cyclophosphamide (VDC), and ifosfamide with etoposide (IE). Common side effects include peripheral neuropathy, cardiotoxicity, myelosuppression, hemorrhagic cystitis, nephrotoxicity, encephalopathy, and secondary malignancies. These side effects require careful monitoring and supportive care.

What prior FDA approvals exist?

Peripheral Primitive Neuroectodermal Tumor (PPNET) is closely related to Ewing sarcoma, and treatments often overlap. FDA-approved treatments for Ewing sarcoma, which may also apply to PPNET, include chemotherapy agents such as cyclophosphamide, ifosfamide, etoposide, and vincristine. Additionally, newer targeted therapies and immunotherapies, such as monoclonal antibodies against the insulin-like growth factor 1 receptor (e.g., figitumumab), are under investigation. These treatments align with the approaches being studied in the ctDNA Detection trial for their prognostic and therapeutic potential.

What other types of research exist?

Promising novel alternatives to ctDNA detection for prognostic biomarker analysis in PPNET patients include: 1) Liquid biopsy using circulating tumor cells (CTCs) which can provide real-time insights into tumor dynamics and treatment response. 2) Advanced imaging techniques like PET/MRI with novel tracers that can offer detailed tumor characterization and monitor treatment efficacy. 3) Multi-omics approaches integrating genomics, transcriptomics, and proteomics to provide a comprehensive molecular profile of the tumor. 4) Use of exosomal RNA and proteins as non-invasive biomarkers for tumor progression and response to therapy.

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Liquid Biopsy for Bone Cancer

N/A

Recruiting

Led By David S Shulman, MD

Research Sponsored by Dana-Farber Cancer Institute

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age: ≥ 12 months of age at time of study enrollment to 50 years of age

Diagnosis: Patients with histologic diagnosis (by institutional pathologist) of newly diagnosed, localized or regionally disseminated Ewing sarcoma or peripheral primitive neuroectodermal tumor (PNET) of bone or soft tissue or; Patients with histologic diagnosis (by institutional pathologist) of newly diagnosed, non-pelvic, localized or regionally disseminated high-grade osteosarcoma. NOTE: Staging will be assessed according to standard of care at the treating center.

Must not have

Patients with a second malignant neoplasm

Patients without detectable tumor at the time of study enrollment (ie, complete tumor resection prior to study enrollment)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a blood test that looks for small pieces of tumor DNA in patients with Ewing sarcoma or osteosarcoma. The goal is to see if this test can help predict if the cancer will return after treatment. A group of patients will participate to help researchers understand how useful this test can be.

Who is the study for?

The LEOPARD trial is for individuals aged 12 months to 50 years with a new diagnosis of Ewing sarcoma or high-grade osteosarcoma, who have not started or just begun treatment. It's not for those with distant metastatic disease, other types of sarcomas, complete tumor resection before joining the study, pelvic primary tumors in osteosarcoma patients, or if pregnant.

What is being tested?

This trial tests the use of FoundationOne Liquid CDx liquid biopsy to see how well it predicts outcomes and monitors response to therapy in patients with Ewing sarcoma or osteosarcoma. Participants will be monitored over time to assess changes in ctDNA levels.

What are the potential side effects?

As this is a diagnostic study focusing on liquid biopsies rather than drug treatments, there are no direct side effects from the intervention being tested.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 1 year and 50 years old.

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I have been diagnosed with Ewing sarcoma, PNET, or high-grade osteosarcoma that is not in my pelvis.

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I have only had a biopsy, no surgery to remove my tumor.

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I am scheduled to receive chemotherapy.

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I have been diagnosed with Ewing sarcoma or PNET of bone or soft tissue.

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I am receiving specific chemotherapy protocols for Ewing sarcoma or osteosarcoma.

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I am at least 1 year old.

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I have only received the first round of treatment for my condition, and maintenance therapy if it was given.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been diagnosed with a second type of cancer.

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My tumor was completely removed before joining the study.

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My cancer is not Ewing-like sarcoma.

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I weigh less than 5 kilograms.

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My cancer originates in the bones of my pelvis.

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My cancer has spread to distant parts of my body.

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I weighed less than 5 kg when my condition was diagnosed.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Event-free survival rate

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: EWING ctDNA RETURN OF RESULTSExperimental Treatment1 Intervention

This study involves collection of blood samples at pre-specified time points as well as collection of information about this disease. For each timepoint, submit two tubes of blood (17 mL total or a little more than 3 teaspoons) to a commercial testing laboratory called Foundation Medicine and one tube of blood (10 mL or 2 teaspoons) to standard ctDNA workflow

Group II: REG EWING or OSTEO: ctDNA EVALUATIONActive Control1 Intervention

0 / 15Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Peripheral Primitive Neuroectodermal Tumor (PPNET) treatments commonly include chemotherapy, radiation therapy, and surgery. Chemotherapy agents like vincristine, ifosfamide, and doxorubicin damage cancer cell DNA, preventing replication and causing cell death. Radiation therapy uses high-energy particles to destroy cancer cells, and surgery aims to remove the tumor. The study of ctDNA as a prognostic biomarker is crucial as it aids in early relapse detection and treatment response monitoring, allowing for more personalized and timely interventions.

Role of Molecular Profiling in Soft Tissue Sarcoma.