RECRUITING Phase 3 ClinicalTrials.gov

Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma

Sponsor: Children's Oncology Group • ClinicalTrials.gov ID: NCT05304585

Registry updated: 2026-08-25

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Description

Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.

Interventions

Biopsy Procedure (Procedure)
Undergo tumor biopsy
Bone Scan (Procedure)
Undergo bone scan
Computed Tomography (Procedure)
Undergo CT scan
Cyclophosphamide (Drug)
Given IV
Dactinomycin (Biological)
Given IV
Magnetic Resonance Elastography (Procedure)
Undergo MRI
Positron Emission Tomography (Procedure)
Undergo PET scan
Radiation Therapy (Radiation)
Undergo radiation
Vincristine (Drug)
Given IV

Eligibility criteria

Inclusion Criteria: * All patients must be enrolled on APEC14B1 (NCT02402244) and consented to the Molecular Characterization Initiative (Part A) prior to enrollment and treatment on ARST2032 (this trial). * Patients must be =\< 21 years at the time of enrollment. * Patients must have newly diagnosed embryonal rhabdomyosarcoma (ERMS), spindle cell/sclerosing RMS, or FOXO1 fusion negative alveolar rhabdomyosarcoma (ARMS) (institutional FOXO1 fusion results are acceptable). RMS types included under ERMS include those classified in the 1995 International Classification of Rhabdomyosarcoma (ICR) as ERMS (classic, spindle cell, and botryoid variants), which are reclassified in the 2020 World Health Organization (WHO) classification as ERMS (classic, dense and botryoid variants) and spindle cell/sclerosing RMS (encompassing the historical spindle cell ERMS variant and the newly recognized sclerosing RMS variant). Enrollment in APEC14B1 is required for all patients. * All patients will be evaluated for stage and clinical group. Note that clinical group designation assigned at the time of enrollment on study remains unchanged regardless of any second-look operation that may be performed. * Patients will be eligible for the very low-risk stratum (Regimen VA) if they have Stage 1, CG I disease. * Patients will be eligible for the low-risk stratum (Regimen VAC/VA) if they have Stage 1, CG II disease, Stage 2, CG I or II disease, or Stage 1, CG III (orbit only) disease. * Paratesticular Tumors: Staging ipsilateral retroperitoneal lymph node sampling (SIRLNS) is required for all patients \>= 10 years of age with paratesticular tumors who do not have gross nodal involvement on imaging. * Extremity Tumors: Regional lymph node sampling is required for histologic evaluation in patients with extremity tumors. * Clinically or radiographically enlarged nodes must be sampled for histologic evaluation. * Patients must have a Lansky (for patients =\< 16 years of age) or Karnofsky (for patients \> 16 years of age) performance status score of \>= 50. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing performance score. * Peripheral absolute neutrophil count (ANC) \>= 750/uL (within 7 days prior to enrollment). * Platelet count \>= 75,000/uL (transfusion independent) (within 7 days prior to enrollment). * Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a serum creatinine (within 7 days prior to enrollment) based on age/gender as follows: * Age: 1 month to \< 6 months; Maximum serum creatinine (mg/dL): 0.4 (male) : 0.4 (female) * Age: 6 months to \< 1 year; Maximum serum creatinine (mg/dL): 0.5 (male) : 0.5 (female) * Age: 1 to \< 2 years; Maximum serum creatinine (mg/dL): 0.6 (male) : 0.6 (female) * Age: 2 to \< 6 years; Maximum serum creatinine (mg/dL): 0.8 (male) : 0.8 (female) * Age: 6 to \< 10 years; Maximum serum creatinine (mg/dL): 1 (male) : 1 (female) * Age: 10 to \< 13 years; Maximum serum creatinine (mg/dL): 1.2 (male) : 1.2 (female) * Age: 13 to \< 16 years; Maximum serum creatinine (mg/dL): 1.5 (male) : 1.4 (female) * Age \>= 16 years; Maximum serum creatinine (mg/dL): 1.7 (male) : 1.4 (female) * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment), and * If there is evidence of biliary obstruction by the tumor, then the total bilirubin must be \< 3 x ULN for age. * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L. * Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L * If there is evidence of biliary obstruction by the tumor, then the total bilirubin must be \< 3 x ULN for age * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L * All patients and/or their parents or legal guardians must sign a written informed consent. * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met. Exclusion Criteria: * Patients who have received prior chemotherapy and/or radiation therapy for cancer prior to enrollment. Surgical resection alone of previous cancer(s) is permitted. * Patients who have received chemotherapy or radiation for non-malignant conditions (e.g., autoimmune diseases) are eligible. Patients must discontinue chemotherapy for non-malignant conditions prior to starting protocol therapy. * Vincristine is sensitive substrate of the CYP450 3A4 isozyme. Patients must not have received drugs that are moderate to strong CYP3A4 inhibitors and inducers within 7 days prior to study enrollment. * Patients unable to undergo radiation therapy, if necessary, as specified in the protocol. * Evidence of uncontrolled infection. * Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential. * Lactating females who plan to breastfeed their infants. * Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation.

Conditions

Embryonal Rhabdomyosarcoma Fusion-Negative Alveolar Rhabdomyosarcoma Spindle Cell/Sclerosing Rhabdomyosarcoma

Locations

Children's Hospital of Alabama — Birmingham, Alabama, United States
USA Health Strada Patient Care Center — Mobile, Alabama, United States
Phoenix Childrens Hospital — Phoenix, Arizona, United States
Arkansas Children's Hospital — Little Rock, Arkansas, United States
Kaiser Permanente Downey Medical Center — Downey, California, United States
Loma Linda University Medical Center — Loma Linda, California, United States
Miller Children's and Women's Hospital Long Beach — Long Beach, California, United States
Children's Hospital Los Angeles — Los Angeles, California, United States
Cedars-Sinai Medical Center — Los Angeles, California, United States
Mattel Children's Hospital UCLA — Los Angeles, California, United States
Valley Children's Hospital — Madera, California, United States
UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
Kaiser Permanente-Oakland — Oakland, California, United States
Children's Hospital of Orange County — Orange, California, United States
Lucile Packard Children's Hospital Stanford University — Palo Alto, California, United States
University of California Davis Comprehensive Cancer Center — Sacramento, California, United States
Rady Children's Hospital - San Diego — San Diego, California, United States
Naval Medical Center -San Diego — San Diego, California, United States
UCSF Medical Center-Mission Bay — San Francisco, California, United States
Children's Hospital Colorado — Aurora, Colorado, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center — Denver, Colorado, United States
Connecticut Children's Medical Center — Hartford, Connecticut, United States
Yale University — New Haven, Connecticut, United States
Alfred I duPont Hospital for Children — Wilmington, Delaware, United States
MedStar Georgetown University Hospital — Washington D.C., District of Columbia, United States
Children's National Medical Center — Washington D.C., District of Columbia, United States
Broward Health Medical Center — Fort Lauderdale, Florida, United States
Golisano Children's Hospital of Southwest Florida — Fort Myers, Florida, United States
UF Health Cancer Institute - Gainesville — Gainesville, Florida, United States
Memorial Regional Hospital/Joe DiMaggio Children's Hospital — Hollywood, Florida, United States
Nemours Children's Clinic-Jacksonville — Jacksonville, Florida, United States
University of Miami Miller School of Medicine-Sylvester Cancer Center — Miami, Florida, United States
Nicklaus Children's Hospital — Miami, Florida, United States
AdventHealth Orlando — Orlando, Florida, United States
Arnold Palmer Hospital for Children — Orlando, Florida, United States
Nemours Children's Hospital — Orlando, Florida, United States
Nemours Children's Clinic - Pensacola — Pensacola, Florida, United States
Sacred Heart Hospital — Pensacola, Florida, United States
Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States
Saint Joseph's Hospital/Children's Hospital-Tampa — Tampa, Florida, United States
Saint Mary's Medical Center — West Palm Beach, Florida, United States
Children's Healthcare of Atlanta - Arthur M Blank Hospital — Atlanta, Georgia, United States
Atrium Health Navicent — Macon, Georgia, United States
Memorial Health University Medical Center — Savannah, Georgia, United States
Kapiolani Medical Center for Women and Children — Honolulu, Hawaii, United States
Saint Luke's Cancer Institute - Boise — Boise, Idaho, United States
Lurie Children's Hospital-Chicago — Chicago, Illinois, United States
University of Illinois — Chicago, Illinois, United States
University of Chicago Comprehensive Cancer Center — Chicago, Illinois, United States
Loyola University Medical Center — Maywood, Illinois, United States
Advocate Children's Hospital-Oak Lawn — Oak Lawn, Illinois, United States
Advocate Children's Hospital-Park Ridge — Park Ridge, Illinois, United States
OSF Children's Hospital of Illinois — Peoria, Illinois, United States
Southern Illinois University School of Medicine — Springfield, Illinois, United States
Northwestern Medicine Central DuPage Hospital — Winfield, Illinois, United States
Riley Hospital for Children — Indianapolis, Indiana, United States
Ascension Saint Vincent Indianapolis Hospital — Indianapolis, Indiana, United States
Blank Children's Hospital — Des Moines, Iowa, United States
University of Iowa/Holden Comprehensive Cancer Center — Iowa City, Iowa, United States
Wesley Medical Center — Wichita, Kansas, United States
University of Kentucky/Markey Cancer Center — Lexington, Kentucky, United States
Norton Children's Hospital — Louisville, Kentucky, United States
Children's Hospital New Orleans — New Orleans, Louisiana, United States
Ochsner Medical Center Jefferson — New Orleans, Louisiana, United States
Maine Children's Cancer Program — Scarborough, Maine, United States
University of Maryland/Greenebaum Cancer Center — Baltimore, Maryland, United States
Sinai Hospital of Baltimore — Baltimore, Maryland, United States
Johns Hopkins University/Sidney Kimmel Cancer Center — Baltimore, Maryland, United States
Massachusetts General Hospital Cancer Center — Boston, Massachusetts, United States
Dana-Farber Cancer Institute — Boston, Massachusetts, United States
UMass Memorial Medical Center - University Campus — Worcester, Massachusetts, United States
C S Mott Children's Hospital — Ann Arbor, Michigan, United States
Children's Hospital of Michigan — Detroit, Michigan, United States
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States
Bronson Methodist Hospital — Kalamazoo, Michigan, United States
Corewell Health Children's — Royal Oak, Michigan, United States
Children's Hospitals and Clinics of Minnesota - Minneapolis — Minneapolis, Minnesota, United States
University of Minnesota/Masonic Cancer Center — Minneapolis, Minnesota, United States
Mayo Clinic in Rochester — Rochester, Minnesota, United States
University of Mississippi Medical Center — Jackson, Mississippi, United States
Children's Mercy Hospitals and Clinics — Kansas City, Missouri, United States
Cardinal Glennon Children's Medical Center — St Louis, Missouri, United States
Washington University School of Medicine — St Louis, Missouri, United States
Mercy Hospital Saint Louis — St Louis, Missouri, United States
Children's Hospital and Medical Center of Omaha — Omaha, Nebraska, United States
University of Nebraska Medical Center — Omaha, Nebraska, United States
Alliance for Childhood Diseases/Cure 4 the Kids Foundation — Las Vegas, Nevada, United States
Summerlin Hospital Medical Center — Las Vegas, Nevada, United States
Renown Regional Medical Center — Reno, Nevada, United States
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center — Lebanon, New Hampshire, United States
Hackensack University Medical Center — Hackensack, New Jersey, United States
Morristown Medical Center — Morristown, New Jersey, United States
Jersey Shore Medical Center — Neptune City, New Jersey, United States
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital — New Brunswick, New Jersey, United States
Newark Beth Israel Medical Center — Newark, New Jersey, United States
University of New Mexico Cancer Center — Albuquerque, New Mexico, United States
Albany Medical Center — Albany, New York, United States
Roswell Park Cancer Institute — Buffalo, New York, United States
The Steven and Alexandra Cohen Children's Medical Center of New York — New Hyde Park, New York, United States
Laura and Isaac Perlmutter Cancer Center at NYU Langone — New York, New York, United States
Mount Sinai Hospital — New York, New York, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center — New York, New York, United States
Memorial Sloan Kettering Cancer Center — New York, New York, United States
University of Rochester — Rochester, New York, United States
State University of New York Upstate Medical University — Syracuse, New York, United States
Montefiore Medical Center - Moses Campus — The Bronx, New York, United States
New York Medical College — Valhalla, New York, United States
UNC Lineberger Comprehensive Cancer Center — Chapel Hill, North Carolina, United States
Carolinas Medical Center/Levine Cancer Institute — Charlotte, North Carolina, United States
Novant Health Presbyterian Medical Center — Charlotte, North Carolina, United States
Duke University Medical Center — Durham, North Carolina, United States
East Carolina University — Greenville, North Carolina, United States
Wake Forest University Health Sciences — Winston-Salem, North Carolina, United States
Sanford Broadway Medical Center — Fargo, North Dakota, United States
Children's Hospital Medical Center of Akron — Akron, Ohio, United States
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
Rainbow Babies and Childrens Hospital — Cleveland, Ohio, United States
Cleveland Clinic Foundation — Cleveland, Ohio, United States
Nationwide Children's Hospital — Columbus, Ohio, United States
Dayton Children's Hospital — Dayton, Ohio, United States
ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital — Toledo, Ohio, United States
University of Oklahoma Health Sciences Center — Oklahoma City, Oklahoma, United States
Oregon Health and Science University — Portland, Oregon, United States
Lehigh Valley Hospital-Cedar Crest — Allentown, Pennsylvania, United States
Geisinger Medical Center — Danville, Pennsylvania, United States
Penn State Children's Hospital — Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Saint Christopher's Hospital for Children — Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
Rhode Island Hospital — Providence, Rhode Island, United States
Medical University of South Carolina — Charleston, South Carolina, United States
Prisma Health Richland Hospital — Columbia, South Carolina, United States
BI-LO Charities Children's Cancer Center — Greenville, South Carolina, United States
Sanford USD Medical Center - Sioux Falls — Sioux Falls, South Dakota, United States
East Tennessee Childrens Hospital — Knoxville, Tennessee, United States
Saint Jude Children's Research Hospital — Memphis, Tennessee, United States
The Children's Hospital at TriStar Centennial — Nashville, Tennessee, United States
Vanderbilt University/Ingram Cancer Center — Nashville, Tennessee, United States
Dell Children's Medical Center of Central Texas — Austin, Texas, United States
Driscoll Children's Hospital — Corpus Christi, Texas, United States
Medical City Dallas Hospital — Dallas, Texas, United States
UT Southwestern/Simmons Cancer Center-Dallas — Dallas, Texas, United States
El Paso Children's Hospital — El Paso, Texas, United States
Cook Children's Medical Center — Fort Worth, Texas, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center — Houston, Texas, United States
UT MD Anderson Cancer Center — Houston, Texas, United States
Covenant Children's Hospital — Lubbock, Texas, United States
UMC Cancer Center / UMC Health System — Lubbock, Texas, United States
Children's Hospital of San Antonio — San Antonio, Texas, United States
Methodist Children's Hospital of South Texas — San Antonio, Texas, United States
University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States
Scott and White Memorial Hospital — Temple, Texas, United States
Primary Children's Hospital — Salt Lake City, Utah, United States
University of Virginia Cancer Center — Charlottesville, Virginia, United States
Inova Fairfax Hospital — Falls Church, Virginia, United States
Children's Hospital of The King's Daughters — Norfolk, Virginia, United States
VCU Massey Comprehensive Cancer Center — Richmond, Virginia, United States
Carilion Children's — Roanoke, Virginia, United States
Seattle Children's Hospital — Seattle, Washington, United States
Providence Sacred Heart Medical Center and Children's Hospital — Spokane, Washington, United States
Mary Bridge Children's Hospital and Health Center — Tacoma, Washington, United States
Madigan Army Medical Center — Tacoma, Washington, United States
West Virginia University Charleston Division — Charleston, West Virginia, United States
University of Wisconsin Carbone Cancer Center - University Hospital — Madison, Wisconsin, United States
Marshfield Medical Center-Marshfield — Marshfield, Wisconsin, United States
Children's Hospital of Wisconsin — Milwaukee, Wisconsin, United States
Sydney Children's Hospital — Randwick, New South Wales, Australia
The Children's Hospital at Westmead — Westmead, New South Wales, Australia
Perth Children's Hospital — Perth, Western Australia, Australia
Alberta Children's Hospital — Calgary, Alberta, Canada
University of Alberta Hospital — Edmonton, Alberta, Canada
British Columbia Children's Hospital — Vancouver, British Columbia, Canada
CancerCare Manitoba — Winnipeg, Manitoba, Canada
IWK Health Centre — Halifax, Nova Scotia, Canada
Hospital for Sick Children — Toronto, Ontario, Canada
The Montreal Children's Hospital of the MUHC — Montreal, Quebec, Canada
Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec, Canada
Centre Hospitalier Universitaire de Sherbrooke-Fleurimont — Sherbrooke, Quebec, Canada
CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL) — Québec, Canada
Starship Children's Hospital — Grafton, Auckland, New Zealand
Christchurch Hospital — Christchurch, New Zealand

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Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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