Chemotherapy + Targeted Therapy for Ewing Sarcoma

Recruiting in Palo Alto (17 mi)

Overseen by Sara M Federico, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: St. Jude Children's Research Hospital

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

This protocol will study treatment for Ewing sarcoma family of tumors (ESFT) and desmoplastic small round cell tumor (DSRCT). Participants with ESFT will be divided into two treatment groups, A or B, based on tumor characteristics. Group A (standard risk) participants have tumor that is not in the pelvis, has not spread to other parts of the body, and are less than 14 years of age. Because previous clinical trials have shown that standard treatment is very effective for children whose tumors have these characteristics, these participants will receive standard treatment. Group B (high risk) participants are 14 years of age or older or have tumor in the pelvis, or the tumor has spread to other parts of the body. Participants with DSRCT in the abdomen and/or pelvis or with tumor that cannot be removed by surgery alone or has spread to other parts of the body will be included in Group B. Participants in this group are considered high risk because there is a greater chance of tumor recurring following standard treatments currently in use. All participants will be followed and evaluated for 10 years following completion of therapy.

Research Team

Sara M Federico, MD

Principal Investigator

St. Jude Children's Research Hospital

Eligibility Criteria

This trial is for children under 14 with non-pelvic ESFT and young adults up to 25 years old with high-risk ESFT or DSRCT, including those with metastatic disease, pelvic tumors, or intra-abdominal DSRCT. Participants must have proper organ function and no prior cancer treatments (except emergency radiation). They should not be pregnant/breastfeeding and must agree to use birth control.

Inclusion Criteria

I am newly diagnosed with a specific type of abdominal cancer that cannot be surgically removed or has spread.

I am newly diagnosed with Ewing sarcoma, and it has spread or is in my pelvis, or I was 14 or older when diagnosed.

My liver is functioning well, with bilirubin levels at or below 3.0 mg/dL.

Exclusion Criteria

Participant is pregnant or breastfeeding.

I had cancer before, but it wasn't skin cancer or it's been 5 years since my skin cancer was treated and I'm in remission without having had chemo, immunotherapy, or radiation.

Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Treatment Details

Interventions

• Bevacizumab (Monoclonal Antibodies)
• Cyclophosphamide (Alkylating agents)
• Doxorubicin (Anti-tumor antibiotic)
• Etoposide (Topoisomerase Inhibitor)
• Ifosfamide (Alkylating agents)
• Radiation (Radiation)
• Sorafenib (Protein Kinase Inhibitor)
• Surgery (Procedure)
• Temozolomide (Alkylating agents)
• Temsirolimus (Protein Kinase Inhibitor)
• Vincristine (Vinca alkaloids)

Trial Overview The study tests standard treatment in Group A (standard risk) children under 14 without pelvic tumors or spread of the disease. Group B (high risk), older than 14, with pelvic tumors or metastasis receive more aggressive therapy. Treatments include chemotherapy drugs like vincristine and doxorubicin, targeted therapies such as temsirolimus and sorafenib, surgery, radiation, plus long-term follow-up.

Participant Groups

Group I: Group A (Standard Risk) Active Control 7 Interventions

Participants will receive vincristine, doxorubicin, cyclophosphamide, ifosfamide and etoposide. Doxorubicin will be omitted following a total cumulative dose of 375 mg/m\^2. Depending on the size and location of the participant's tumor, they will have surgery alone, radiation alone, or surgery followed by radiation. Local control measures (surgery and/or radiation therapy) will be instituted after 6 courses of chemotherapy. Total duration of treatment is approximately 29 weeks.

Group II: Group B (High Risk) Active Control 10 Interventions

Participants will receive vincristine, doxorubicin, cyclophosphamide, ifosfamide, etoposide, irinotecan, temozolomide, temsirolimus, bevacizumab, and sorafenib. Depending on the size and location of the participant's tumor, they will have surgery alone, radiation alone or surgery followed by radiation.

Cyclophosphamide is already approved in Canada, Japan for the following indications:

Approved in Canada as Neosar for:

• Breast cancer
• Ovarian cancer
• Multiple myeloma
• Leukemia
• Lymphoma
• Rheumatoid arthritis

Approved in Japan as Endoxan for:

• Breast cancer
• Ovarian cancer
• Multiple myeloma
• Leukemia
• Lymphoma

Who Is Running the Clinical Trial?

St. Jude Children's Research Hospital

Lead Sponsor

Trials

451

Recruited

5,326,000+

Dr. James R. Downing

St. Jude Children's Research Hospital

Chief Executive Officer since 2014

MD from University of Michigan Medical School

Dr. Ellis J. Neufeld

St. Jude Children's Research Hospital

Chief Medical Officer since 2017

MD, PhD from Harvard Medical School

University of Tennessee Health Science Center

Collaborator

Trials

49

Recruited

24,600+

University of Tennessee

Collaborator

Trials

202

Recruited

146,000+

Larry Dillaha

University of Tennessee

Chief Executive Officer since 2021

MD from the University of Tennessee, Memphis

Robert Elfont

University of Tennessee

Chief Medical Officer since 2021

MD, PhD

Nemours Children's Clinic

Collaborator

Trials

128

Recruited

18,000+

R. Lawrence Moss

Nemours Children's Clinic

Chief Executive Officer since 2018

MD, FACS, FAAP

Matthew Davis

Nemours Children's Clinic

Chief Medical Officer since 2023

MD, MAPP

University of Florida

Collaborator

Trials

1,428

Recruited

987,000+

Dr. Stephen J. Motew

University of Florida

Chief Executive Officer since 2024

MD cum laude from the University of Illinois at Chicago School of Medicine, Master's in Healthcare Administration from the University of North Carolina at Chapel Hill

Dr. Timothy E. Morey

University of Florida

Chief Medical Officer since 2023

MD and Bachelor's from the University of Florida