Liquid Biopsy in Ewing Sarcoma and Osteosarcoma as a Prognostic And Response Diagnostic: LEOPARD

NCT ID: NCT06068075

Last Updated: 2026-01-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

340 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-05-08

Study Completion Date

2027-01-01

Brief Summary

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This is a prospective multicenter biomarker study evaluating the prognostic impact of ctDNA detection at diagnosis in patients with Ewing sarcoma or osteosarcoma.

Detailed Description

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The purpose of this study is to evaluate whether ctDNA in the blood can provide information about the chances of Ewing sarcoma or osteosarcoma coming back after treatment. This research study is evaluating a new advanced laboratory test to detect small pieces of tumor genes in the peripheral blood known as circulating tumor DNA (ctDNA).

Part A : During this part of the research study (Part A) participants will be asked to provide blood samples at pre-defined times. These blood samples may help find specific genetic alterations commonly seen in Ewing sarcoma or osteosarcoma that may allow investigators to learn more about the uses of ctDNA. The results of the ctDNA analysis will not be returned to participants.

Approximately 90 patients will take part in this study across multiple centers.

Part B: This research study is evaluating new advanced laboratory tests to detect small pieces of tumor genes in the peripheral blood known as circulating tumor DNA (ctDNA). Part B of the research study, which focuses on ctDNA tests that can be returned to providers and patients with Ewing sarcoma. This part of the study will allow comparison of commercial ctDNA testing from Foundation Medicine to our research testing. It is expected that about 60 people with Ewing sarcoma will take part in Part B of this research study.

The sponsor of this protocol is Dana-Farber Cancer Institute and is providing funding for the study. Additional funding for this study is provided by the Conquer Cancer Foundation of the American Society of Clinical Oncology, Alex's Lemonade Stand Foundation, Boston Children's Hospital Office of Faculty Development, the Friends of Dana-Farber Cancer Institute, The Harvard Catalyst Program, and the Spada Pediatric Sarcoma Foundation

Conditions

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Ewing Sarcoma Ewing Sarcoma of Bone Ewing Sarcoma of Soft Tissue Peripheral Primitive Neuroectodermal Tumor Peripheral Primitive Neuroectodermal Tumor of Bone Peripheral Primitive Neuroectodermal Tumor of Soft Tissue High-grade Osteosarcoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

DIAGNOSTIC

Blinding Strategy

NONE

Study Groups

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REG EWING or OSTEO: ctDNA EVALUATION

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) per standard ctDNA workflow

Group Type NO_INTERVENTION

No interventions assigned to this group

EWING ctDNA RETURN OF RESULTS

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 Type EXPERIMENTAL

FoundationOne Liquid CDx

Intervention Type OTHER

a FoundationOne liquid biopsy test kit will be sent to the laboratory of the subject's treating center, in addition to the paired cell stabilizing tube from the primary center

Interventions

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FoundationOne Liquid CDx

a FoundationOne liquid biopsy test kit will be sent to the laboratory of the subject's treating center, in addition to the paired cell stabilizing tube from the primary center

Intervention Type OTHER

Other Intervention Names

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Liquid Biopsy

Eligibility Criteria

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Inclusion Criteria

* For Part A, subjects must meet all of the following eligibility criteria.
* 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.
* Prior Therapy:

* Patients should have only previously had a biopsy, and not had prior attempt at tumor resection.
* Not yet started chemotherapy or radiation therapy OR patient has started chemotherapy or radiation therapy, but an appropriate pre-treatment baseline sample was collected and processed for ctDNA under a local banking study in DFCI Pediatrics and is available to use for this study.
* Planned to receive chemotherapy as follows:

\-- VDC/IE as per COG protocols AEWS0031, AEWS1031 or AEWS1221 (for patients with Ewing sarcoma or PNET); or MAP as per COG protocol AOST0331 (for patients with osteosarcoma).
* For Part B subjects must meet all of the following eligibility criteria.
* Age: ≥ 12 months of age at time of study enrollment
* Diagnosis: Patients with histologic diagnosis (by institutional pathologist) of newly diagnosed Ewing sarcoma or peripheral primitive neuroectodermal tumor (PNET) of bone or soft tissue
* Prior Therapy:

* Patients should have had only frontline therapy as per institutional standard, and maintenance therapy if given (no relapse therapy).
* If frontline systemic therapy already completed (not including maintenance or metastatic site radiation), therapy completed within 6 months of enrollment to Part B.
* Subjects must have a willing physician provider supporting their participation in Part B.
* For Part B, providers are eligible to receive the provider survey if they are listed as the primary provider for the patient at the study site.

Exclusion Criteria

* Patients with distant metastatic disease.
* Patients with known Ewing-like sarcoma (e.g., BCOR-CCNB3 or CIC-DUX4 translocated small round cell sarcomas) are not eligible.
* Patients who are enrolled with an initial diagnosis of Ewing sarcoma and subsequently found to have Ewing-like sarcoma will be replaced. Samples obtained prior to removal from study will be analyzed and reported descriptively. Patients with Ewing-like tumors may continue to provide samples and clinical data until they meet off-study criteria per protocol.
* Patients weighing \< 5 kg at time of diagnosis
* Patients with a second malignant neoplasm
* Patients without detectable tumor at the time of study enrollment (ie, complete tumor resection prior to study enrollment)
* Patients already receiving tumor-directed therapy at the time of study enrollment except when a pre-treatment baseline sample has already been obtained under a local banking study in DFCI Pediatrics that would be eligible for analysis under this study.
* Patients with osteosarcoma with a pelvic primary tumor site Pregnancy
* Patients with known Ewing-like sarcoma (e.g., BCOR-CCNB3 or CIC-DUX4 translocated small round cell sarcomas) are not eligible.
* Samples obtained prior to removal from study will be analyzed and reported descriptively. Patients with Ewing-like tumors may continue to provide samples and clinical data until they meet off-study criteria per protocol
* Patients weighing \< 5 kg at time of enrollment
* Patients diagnosed with relapsed disease and/or having started therapy directed at disease relapse
* Pregnancy
* Resides outside of the United States
* For Part B, providers at non-study centers will not be eligible to receive the provider survey.
Minimum Eligible Age

12 Months

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alex's Lemonade Stand Foundation

INDUSTRY

Sponsor Role collaborator

Conquer Cancer Foundation

OTHER

Sponsor Role collaborator

Harvard University

OTHER

Sponsor Role collaborator

Sam Day Foundation

UNKNOWN

Sponsor Role collaborator

Dana-Farber Cancer Institute

OTHER

Sponsor Role lead

Responsible Party

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David S Shulman, MD

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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David S Shulman, MD

Role: PRINCIPAL_INVESTIGATOR

Dana-Farber Cancer Institute

Locations

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Childrens Hospital Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status RECRUITING

Massachusetts General Hospital Cancer Center

Boston, Massachusetts, United States

Site Status RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status RECRUITING

Children's Hospital's and Clinics of Minnesota

Minneapolis, Minnesota, United States

Site Status RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Lifespan / Rhode Island Hospital

Providence, Rhode Island, United States

Site Status RECRUITING

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status RECRUITING

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

University of Utah Childrens Medical Center

Salt Lake City, Utah, United States

Site Status RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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David S. Shulman, MD

Role: CONTACT

617-632-6670

Facility Contacts

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Edwin Choy, MD

Role: primary

David Shulman, MD

Role: primary

617-632-6670

Pricilla Merriam, MD

Role: primary

617-632-5204

David S Shulman, MD

Role: primary

617-632-6670

Kris Ann Schultz

Role: primary

Brian Turpin, MD

Role: primary

Bhuvana Setty, MD

Role: primary

Rochelle Bagatell, MD

Role: primary

Bradley DeNardo, MD

Role: primary

Michael Bishop, MD

Role: primary

Avanthi Shah, MD

Role: primary

Luke Maese, MD

Role: primary

Other Identifiers

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18-138

Identifier Type: -

Identifier Source: org_study_id

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