Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma
Launched by CHILDREN'S ONCOLOGY GROUP · Mar 28, 2022
Trial Information
Current as of July 22, 2025
Recruiting
Keywords
ClinConnect Summary
This clinical trial is studying the use of chemotherapy to treat children and young adults with newly diagnosed very low-risk and low-risk forms of rhabdomyosarcoma, a type of cancer that affects soft tissues. The goal is to maintain good treatment outcomes while reducing the amount of chemotherapy needed. Participants will receive a specific treatment plan involving two chemotherapy drugs, vincristine and dactinomycin, for 24 weeks. The study also aims to understand how patients with different types of rhabdomyosarcoma respond to treatment and whether those with certain DNA mutations need more intensive therapy for better results.
To be eligible for this trial, patients must be 21 years old or younger and have a newly diagnosed case of specific types of rhabdomyosarcoma. They should not have received any prior chemotherapy or radiation for cancer. Throughout the trial, participants will undergo regular health assessments to monitor their response to treatment and any side effects. Parents and guardians will need to provide consent for their children to participate. This study is currently recruiting participants, and it aims to help improve treatment strategies for young patients facing this type of cancer.
Gender
ALL
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.
About Children's Oncology Group
The Children's Oncology Group (COG) is a leading national organization dedicated to improving the care and outcomes of children with cancer through collaborative research and clinical trials. Comprising a network of pediatric oncology experts, COG focuses on developing innovative treatment protocols, advancing scientific knowledge, and enhancing the quality of life for young patients. By fostering interdisciplinary collaboration and utilizing a comprehensive approach to childhood cancer, COG aims to translate research findings into effective therapies, ultimately striving for a cure for all children diagnosed with cancer.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
New Haven, Connecticut, United States
Durham, North Carolina, United States
Cleveland, Ohio, United States
Philadelphia, Pennsylvania, United States
Buffalo, New York, United States
Saint Louis, Missouri, United States
Detroit, Michigan, United States
Providence, Rhode Island, United States
Dallas, Texas, United States
Kalamazoo, Michigan, United States
Maywood, Illinois, United States
Boston, Massachusetts, United States
Loma Linda, California, United States
Newark, New Jersey, United States
Valhalla, New York, United States
Winnipeg, Manitoba, Canada
Hackensack, New Jersey, United States
Edmonton, Alberta, Canada
Vancouver, British Columbia, Canada
Peoria, Illinois, United States
Wichita, Kansas, United States
Oklahoma City, Oklahoma, United States
Temple, Texas, United States
Toronto, Ontario, Canada
Orange, California, United States
Scarborough, Maine, United States
Akron, Ohio, United States
Lubbock, Texas, United States
Norfolk, Virginia, United States
Randwick, New South Wales, Australia
New York, New York, United States
Baltimore, Maryland, United States
Little Rock, Arkansas, United States
Des Moines, Iowa, United States
Jackson, Mississippi, United States
Minneapolis, Minnesota, United States
Austin, Texas, United States
Corpus Christi, Texas, United States
Calgary, Alberta, Canada
Quebec, , Canada
Hershey, Pennsylvania, United States
Charlottesville, Virginia, United States
Houston, Texas, United States
Tacoma, Washington, United States
San Antonio, Texas, United States
Springfield, Illinois, United States
Cincinnati, Ohio, United States
San Antonio, Texas, United States
Boston, Massachusetts, United States
Los Angeles, California, United States
Rochester, New York, United States
Danville, Pennsylvania, United States
San Diego, California, United States
Washington, District Of Columbia, United States
Orlando, Florida, United States
Savannah, Georgia, United States
Chicago, Illinois, United States
Chicago, Illinois, United States
Iowa City, Iowa, United States
New Orleans, Louisiana, United States
Baltimore, Maryland, United States
Baltimore, Maryland, United States
Minneapolis, Minnesota, United States
Omaha, Nebraska, United States
New York, New York, United States
New York, New York, United States
Syracuse, New York, United States
Chapel Hill, North Carolina, United States
Winston Salem, North Carolina, United States
Fargo, North Dakota, United States
Nashville, Tennessee, United States
Birmingham, Alabama, United States
Hartford, Connecticut, United States
Indianapolis, Indiana, United States
Lexington, Kentucky, United States
Charlotte, North Carolina, United States
Allentown, Pennsylvania, United States
Falls Church, Virginia, United States
Washington, District Of Columbia, United States
Honolulu, Hawaii, United States
Portland, Oregon, United States
Pensacola, Florida, United States
Grand Rapids, Michigan, United States
Cleveland, Ohio, United States
Phoenix, Arizona, United States
Oakland, California, United States
San Diego, California, United States
Aurora, Colorado, United States
Wilmington, Delaware, United States
Fort Lauderdale, Florida, United States
Hollywood, Florida, United States
Jacksonville, Florida, United States
Miami, Florida, United States
Miami, Florida, United States
Tampa, Florida, United States
West Palm Beach, Florida, United States
Chicago, Illinois, United States
Ann Arbor, Michigan, United States
Saint Louis, Missouri, United States
New Brunswick, New Jersey, United States
Albany, New York, United States
New Hyde Park, New York, United States
Charlotte, North Carolina, United States
Greenville, North Carolina, United States
Columbus, Ohio, United States
Dayton, Ohio, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Sioux Falls, South Dakota, United States
Knoxville, Tennessee, United States
Dallas, Texas, United States
Fort Worth, Texas, United States
Houston, Texas, United States
Salt Lake City, Utah, United States
Richmond, Virginia, United States
Seattle, Washington, United States
Spokane, Washington, United States
Tacoma, Washington, United States
Montreal, Quebec, Canada
Grafton, Auckland, New Zealand
Christchurch, , New Zealand
Downey, California, United States
Long Beach, California, United States
Madera, California, United States
Oakland, California, United States
Palo Alto, California, United States
San Francisco, California, United States
Denver, Colorado, United States
Fort Myers, Florida, United States
Gainesville, Florida, United States
Orlando, Florida, United States
Orlando, Florida, United States
Saint Petersburg, Florida, United States
Atlanta, Georgia, United States
Oak Lawn, Illinois, United States
Indianapolis, Indiana, United States
Louisville, Kentucky, United States
Rochester, Minnesota, United States
Kansas City, Missouri, United States
Saint Louis, Missouri, United States
Omaha, Nebraska, United States
Las Vegas, Nevada, United States
Bronx, New York, United States
Toledo, Ohio, United States
Columbia, South Carolina, United States
Greenville, South Carolina, United States
Memphis, Tennessee, United States
Roanoke, Virginia, United States
Madison, Wisconsin, United States
Marshfield, Wisconsin, United States
Milwaukee, Wisconsin, United States
Westmead, New South Wales, Australia
Montreal, Quebec, Canada
Little Rock, Arkansas, United States
Los Angeles, California, United States
Worcester, Massachusetts, United States
Reno, Nevada, United States
Albuquerque, New Mexico, United States
Mobile, Alabama, United States
Los Angeles, California, United States
Pensacola, Florida, United States
Boise, Idaho, United States
Royal Oak, Michigan, United States
Charleston, West Virginia, United States
Sherbrooke, Quebec, Canada
Park Ridge, Illinois, United States
Las Vegas, Nevada, United States
San Antonio, Texas, United States
Perth, Western Australia, Australia
Winfield, Illinois, United States
Halifax, Nova Scotia, Canada
Nashville, Tennessee, United States
Lubbock, Texas, United States
El Paso, Texas, United States
Saint Louis, Missouri, United States
Indianapolis, Indiana, United States
Lebanon, New Hampshire, United States
West Palm Beach, Florida, United States
Quebec, , Canada
Madison, Wisconsin, United States
Grand Rapids, Michigan, United States
Royal Oak, Michigan, United States
Atlanta, Georgia, United States
Patients applied
Trial Officials
Josephine H Haduong
Principal Investigator
Children's Oncology Group
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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