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Chemotherapy

Multimodal Therapy for Rhabdomyosarcoma

Phase 2

Waitlist Available

Led By Matthew J. Krasin, MD

Research Sponsored by St. Jude Children's Research Hospital

Eligibility Criteria Checklist

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

Must have

Initiation of chemotherapy is planned within 6 weeks of the definitive biopsy or surgical resection

Adequate bone marrow function

Must not have

Undergoing upfront surgical resection of the primary tumor

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years after last low or intermediate risk arm enrollment

Awards & highlights

No Placebo-Only Group

Summary

This trial will treat people with newly diagnosed rhabdomyosarcoma with different intensities of chemotherapy, radiation and surgery based on how high-risk the cancer is. For the intermediate and high risk patients, they will receive an additional 12 weeks of maintenance therapy with anti-angiogenic chemotherapy.

Who is the study for?

This trial is for children and young adults under 22 with newly diagnosed rhabdomyosarcoma (RMS), a type of muscle cancer. They must have low, intermediate, or high-risk disease levels, be able to start chemotherapy within 6 weeks after diagnosis, and have good organ function. Pregnant or breastfeeding individuals can't join.

What is being tested?

The study tests risk-adapted therapy using standard/intensified chemo, radiation, surgery for RMS patients. It aims to improve survival rates in intermediate/high-risk groups by adding maintenance anti-angiogenic therapy after initial treatment.

What are the potential side effects?

Possible side effects include those from chemotherapy like nausea, hair loss, blood disorders; surgical complications; radiation-related skin issues; and specific toxicities from proton beam therapy such as damage to nearby tissues.

Eligibility Criteria

Inclusion Criteria

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

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I am starting chemotherapy within 6 weeks after my biopsy or surgery.

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My bone marrow is working well.

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I need emergency radiation therapy.

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I do not have any ongoing, untreated infections.

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I have been recently diagnosed with localized rhabdomyosarcoma.

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I can take care of myself and perform daily activities.

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I am younger than 22 years old.

Exclusion Criteria

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

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I am scheduled for initial surgery to remove my cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years after last enrollment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years after last enrollment

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

Treatment Details

Study Objectives

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

Primary study objectives

Event-free survival (intermediate risk arm)

Secondary study objectives

Event-free survival (high risk arm)

Incidence of CTC grade 3 and higher toxicities related to proton bream therapy (low and intermediate and high risk arms)

Local failure rate (high risk arm)

+7 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

4Treatment groups

Experimental Treatment

Group I: Low-Risk, Subset 2Experimental Treatment7 Interventions

Lymph node sampling will take place pretreatment and pre-surgery. Participants receive 12 weeks of chemotherapy (vincristine, dactinomycin, cyclophosphamide). The tumor is evaluated to determine how it responded to treatment. Radiation therapy and/or surgical resection is performed to destroy or remove the remaining tumor. Twelve additional weeks of chemotherapy (vincristine, dactinomycin, cyclophosphamide) is given, followed by evaluation for tumor response. If delayed for medical reasons, radiation therapy and/or surgical resection is done at this time. Participants then receive 16 weeks of additional chemotherapy (vincristine, dactinomycin and cyclophosphamide). No further treatment is given, and participants are observed closely. Myeloid growth factor will be given if needed. Participants also receive \^1\^1C-methionine as described in the intervention section.

Group II: Low-Risk, Subset 1Experimental Treatment8 Interventions

Lymph node sampling will take place pretreatment and pre-surgery. Participants receive 12 weeks of chemotherapy (vincristine, dactinomycin and cyclophosphamide). They are then evaluated to determine how the tumor responded to treatment. Twelve additional weeks of chemotherapy (vincristine and dactinomycin) is given, followed by evaluation for tumor response. No further treatment is given, and participants are observed closely. Myeloid growth factor is given if needed. Participants also receive \^1\^1C-methionine as described in the intervention section.

Group III: Intermediate-RiskExperimental Treatment10 Interventions

Lymph node sampling takes place pretreatment and pre-surgery. Participants receive 12 weeks of chemotherapy (vincristine, dactinomycin, cyclophosphamide). The tumor is evaluated for treatment response. Radiation therapy and/or surgical resection is done. Twelve weeks of chemotherapy (vincristine, dactinomycin, cyclophosphamide) is followed by evaluation for tumor response. If delayed for medical reasons, radiation therapy and/or surgical resection is done at this time. Participants receive 16 weeks of chemotherapy (vincristine, dactinomycin, cyclophosphamide) followed by 12 weeks of maintenance treatment (bevacizumab, sorafenib, oral cyclophosphamide). No further treatment is given, and participants are observed closely. Myeloid growth factor is given if needed. Participants also receive \^1\^1C-methionine as described in the intervention section.

Group IV: High-RiskExperimental Treatment16 Interventions

Lymph node sampling takes place pretreatment and pre-surgery. Participants receive 6 weeks (2 cycles) chemotherapy (vincristine and irinotecan). The tumor is evaluated for treatment response. 3 cycles of chemotherapy \[vincristine, doxorubicin, cyclophosphamide/ifosfamide, etoposide (or etoposide phosphate) (VDC/IE)\] are given. Dexrazoxane is given prior to each dose of doxorubicin. Radiation therapy begins at week 4 or 20 (depending on tumor location) while receiving vincristine and irinotecan. 2 cycles of VDC/IE, 4 cycles of modified vincristine, dactinomycin, cyclophosphamide (VAC), then 2 cycles of modified vincristine/irinotecan (total of 54 weeks). High risk participants also receive additional maintenance therapy beginning week 55 with anti-angiogenic chemotherapy (bevacizumab, sorafenib, cyclophosphamide). Myeloid growth factor is given as needed. Participants also receive \^1\^1C-methionine as described in the intervention section.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vincristine

2003

Completed Phase 4

~2970

Dactinomycin

2010

Completed Phase 3

~1310