A Phase 3 interventional study of Biospecimen Collection and Bone Marrow Aspiration in Rhabdomyosarcoma, sponsored by Children's Oncology Group. Recruiting at 87 sites in 2 countries. Open to participants aged Up to 50 Years. Per ClinicalTrials.gov, last updated 2026-08-25.
Sponsored by Children's Oncology Group · Phase 3, Interventional, and Treatment
This phase III trial compares higher dose chemotherapy, with vincristine, dactinomycin and cyclophosphamide, over a shorter amount of time to lower dose chemotherapy plus maintenance, with vincristine, dactinomycin, cyclophosphamide, irinotecan and vinorelbine, over a longer amount of time, along with standard of care surgery and radiation, in patients with newly diagnosed intermediate risk rhabdomyosarcoma. Vincristine and vinorelbine are in a class of medications called vinca alkaloids. They work by stopping tumor cells from growing and dividing and may kill them. Dactinomycin is a type of antibiotic that is only used in cancer chemotherapy (antineoplastic antibiotic). It works by damaging the cell's DNA and may kill tumor cells. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's DNA and may kill tumor cells. It may also lower the body's immune response. Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and DNA repair and may kill tumor cells. It is not yet known if the higher dose chemotherapy over a shorter amount of time or the lower dose chemotherapy with maintenance over a longer amount of time is more effective in the treatment of patient with newly diagnosed, intermediate risk rhabdomyosarcoma.
PRIMARY OBJECTIVE:
I. To determine if the event free survival (EFS) of patients with intermediate risk rhabdomyosarcoma (IR RMS) treated with surgery, radiotherapy, and vincristine, dactinomycin, cyclophosphamide (VAC) (2.2 g/m\^2/cycle cyclophosphamide) chemotherapy (regimen A) is better than that of patients treated with surgery, radiotherapy, and VAC alternating with vincristine, irinotecan (VI) (VAC/VI) (1.2 g/m\^2/cycle cyclophosphamide) chemotherapy plus 24 weeks of maintenance chemotherapy with vinorelbine and cyclophosphamide (regimen B).
SECONDARY OBJECTIVES:
I. To determine if the overall survival (OS) of patients with IR RMS treated with surgery and/or radiotherapy, and VAC (2.2 g/m\^2/cycle cyclophosphamide) chemotherapy (regimen A) is better than the OS of patients treated with surgery, radiotherapy, and VAC alternating with VI (VAC/VI) (1.2 g/m\^2/cycle cyclophosphamide) plus 24 weeks of maintenance chemotherapy with vinorelbine and cyclophosphamide (regimen B).
II. To compare clinician-reported treatment-related adverse event (AE) rates between two regimens.
III. To determine what proportion of patients deemed eligible for delayed primary excision (DPE) on retrospective central review undergo DPE and to assess the concordance of DPE eligibility between the central review and local site.
IV. To compare the 4-year local failure (LF) rate of patients deemed DPE-eligible by retrospective central review who undergo DPE with the 4-year LF rate of patients deemed DPE-eligible by central review who do not undergo DPE.
V. To determine the feasibility of reporting diagnostic tumor molecular features identified via the Molecular Characterization Initiative (MCI) within 6 weeks of treatment initiation for clinical group III patients.
EXPLORATORY OBJECTIVES:
I. To prospectively evaluate the following somatic molecular features (PAX3 or PAX7 and FOXO1 fusion, MYCN amplification, TP53 mutation, MYOD1 mutation, CDK4 amplification) via MCI and determine their association with EFS and OS.
II. To explore the relationship between methylation patterns in IR RMS and EFS and OS.
III. To test the use of digital pathology/artificial intelligence to refine the diagnosis of IR RMS.
IV. To assess the differential impact of regimen intensity on gonadal toxicity experienced by patients.
V. To determine the proportion of patients having fertility discussions and fertility preservation procedures prior to starting treatment.
VI. To collect biospecimens for patient-derived xenograft (PDX) RMS model generation.
VII. To bank biospecimens for future research. VIII. To evaluate the association between Household Material Hardship (HMH) measures and EFS and OS.
OUTLINE: Patients are randomized to 1 of 2 regimens.
REGIMEN A:
CYCLES 1-4, 8, 12: Patients receive vincristine intravenously (IV) on days 1, 8 and 15 of each cycle, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of each cycle, and cyclophosphamide IV over 1-6 hours on day 1 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.
CYCLES 5, 9, 10, 13, 14: Patients receive vincristine IV on day 1 of each cycle, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of each cycle, and cyclophosphamide IV over 1-6 hours on day 1 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.
CYCLE 6: Patients receive vincristine IV on day 1 and cyclophosphamide IV over 1-6 hours on day 1. Cycle 6 continues for 21 days in the absence of disease progression or unacceptable toxicity.
CYCLE 7: Patients receive vincristine IV on days 1, 8 and 15 and cyclophosphamide IV over 1-6 hours on day 1. Cycle 7 continues for 21 days in the absence of disease progression or unacceptable toxicity.
Patients may undergo surgical resection during cycle 4 (week 12), radiation to the primary site during cycle 5 and 6 and/or radiation to distant metastatic sites during cycle 14. Patients do not receive dactinomycin during radiation. Patients undergo computed tomography (CT) scan and/or magnetic resonance imaging (MRI) and blood sample collection throughout the study. Patients may undergo lymph node biopsy during screening and/or fludeoxyglucose (FDG) positron emission tomography (PET) scan, bone scan, bone marrow biopsy and aspiration, lumbar puncture with cerebrospinal fluid sample collection throughout the study.
REGIMEN B:
CYCLES 1, 3, 8: Patients receive vincristine IV on days 1, 8 and 15 of each cycle, dactinomycin IV over 15 or 10-15 minutes on day 1 of each cycle, and cyclophosphamide IV over 1-6 hours on day 1 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.
CYCLES 2, 4, 11: Patients receive vincristine IV on days 1, 8 and 15 of each cycle and irinotecan IV over 90 minutes on days 1-5 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.
CYCLES 5, 10, 12, 14: Patients receive vincristine IV on day 1 of each cycle, dactinomycin IV over 15 or 10-15 minutes on day 1 of each cycle, and cyclophosphamide IV over 1-6 hours on day 1 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.
CYCLES 6, 7, 9, 13: Patients receive vincristine IV on days 1 and 8 of each cycle and irinotecan IV over 90 minutes on days 1-5 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Patients may undergo surgical resection during cycle 4 (week 12), radiation to the primary site during cycle 5 and 6 and/or radiation to distant metastatic sites during cycle 14. Patients do not receive dactinomycin during radiation.
MAINTENANCE: Patients receive vinorelbine IV over 6-10 minutes on days 1, 8 and 15 of each cycle and cyclophosphamide orally (PO) once daily (QD) on days 1-28 of each cycle. Cycles repeat every 28 days for 6 cycles in the absence of disease progression or unacceptable toxicity.
Patients undergo CT scan and/or MRI and blood sample collection throughout the study. Patients may undergo lymph node biopsy during screening and/or FDG PET scan, bone scan, bone marrow biopsy and aspiration, lumbar puncture with cerebrospinal fluid sample collection throughout the study.
After completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for years 2 and 3 then every 6 months for year 4 and 5.
238 studies on the registry are indexed under Rhabdomyosarcoma; 49 are open to participants now.
This study's planned enrollment of 342 is above the median of 42 across 202 interventional studies indexed under Rhabdomyosarcoma.
Browse Rhabdomyosarcoma studies →Children's Oncology Group is the lead sponsor of 448 studies on the registry; 36 are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 11 (92%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patients with newly diagnosed soft tissue RMS of any subtype, except adult-type pleomorphic, based upon institutional histopathologic classification, are eligible to enroll on the study based upon FOXO1 fusion status, Stage, Intergroup Rhabdomyosarcoma Study (IRS) group, and age, as below. FOXO1 fusion status must be determined prior to enrollment. RMS types included under embryonal rhabdomyosarcoma (ERMS) include those which are reclassified in the 2020 World Health Organization (WHO) classification as ERMS (typical, dense and botryoid variants) and spindle cell/sclerosing RMS (encompassing the historical spindle cell ERMS variant and the newly recognized sclerosing RMS variant). Classification of alveolar Rhabdomyosarcoma (ARMS) in the 2020 WHO Classification is the same as in the International Classification of Rhabdomyosarcoma (ICR) and includes classic and solid variants.
FOXO1 fusion negative (FN)
FOXO1 fusion positive (FP)
Please note the following:
Patients who are \< 10 years old with distant disease (Stage 4) who have institutional molecular testing indicating fusion negative (FN) RMS but are later found to have fusion positive (FP) RMS by MCI or other testing will be considered to have metastatic FP disease and will go off study
A serum creatinine based on age/sex as follows:
OR a (GFR) ≥ 70 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard).
For adult patients (aged 18 years or older):
(All laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated. Laboratory studies must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment. Laboratory tests need not be repeated if therapy starts within seven (7) days of their most recent prior assessment. If the result of a laboratory study that is repeated at any time post-enrollment and prior to the start of protocol therapy is outside the limits for eligibility, then the evaluation must be rechecked within 48 hours prior to initiating protocol therapy. The results of the recheck must be within the limits for eligibility to proceed. If the result of the recheck is outside the limits of eligibility, the patient may not receive protocol therapy and will be considered off protocol therapy.)
Total ≤ 1.5 x (ULN) for age (All laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated. Laboratory studies must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment. Laboratory tests need not be repeated if therapy starts within seven (7) days of their most recent prior assessment. If the result of a laboratory study that is repeated at any time post-enrollment and prior to the start of protocol therapy is outside the limits for eligibility, then the evaluation must be rechecked within 48 hours prior to initiating protocol therapy. The results of the recheck must be within the limits for eligibility to proceed. If the result of the recheck is outside the limits of eligibility, the patient may not receive protocol therapy and will be considered off protocol therapy.)
Serum glutamic pyruvic transaminase () (alanine aminotransferase [ALT]) ≤ 135 U/L (All laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated. Laboratory studies must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment. Laboratory tests need not be repeated if therapy starts within seven (7) days of their most recent prior assessment. If the result of a laboratory study that is repeated at any time post-enrollment and prior to the start of protocol therapy is outside the limits for eligibility, then the evaluation must be rechecked within 48 hours prior to initiating protocol therapy. The results of the recheck must be within the limits for eligibility to proceed. If the result of the recheck is outside the limits of eligibility, the patient may not receive protocol therapy and will be considered off protocol therapy.)
Exclusion Criteria:
Previous or concurrent cancer(s) that is/was being treated with chemotherapy and/or radiation.
Patients with central nervous system involvement of RMS as defined below:
Patients who have received any chemotherapy (excluding ) and/or radiation therapy for RMS prior to enrollment. Note: the following exception:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.
Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Brain metastases
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Leptomeningeal disease
Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.
Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Reproductive potential
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
See Detailed Description for Regimen A.
Procedure: Biospecimen Collection · Procedure: Bone Marrow Aspiration · Procedure: Bone Marrow Biopsy · Procedure: Bone Scan · Procedure: Computed Tomography · Drug: Cyclophosphamide · Biological: Dactinomycin · Procedure: Lumbar Puncture · Procedure: Lymph Node Biopsy · Procedure: Magnetic Resonance Imaging · Procedure: Positron Emission Tomography · Radiation: Radiation Therapy · Procedure: Resection · Other: Survey Administration · Drug: Vincristine Sulfate
See Detailed Description for Regimen B.
Procedure: Biospecimen Collection · Procedure: Bone Marrow Aspiration · Procedure: Bone Marrow Biopsy · Procedure: Bone Scan · Procedure: Computed Tomography · Drug: Cyclophosphamide · Biological: Dactinomycin · Drug: Irinotecan Hydrochloride · Procedure: Lumbar Puncture · Procedure: Lymph Node Biopsy · Procedure: Magnetic Resonance Imaging · Procedure: Positron Emission Tomography · Radiation: Radiation Therapy · Procedure: Resection · Other: Survey Administration · Drug: Vincristine Sulfate · Drug: Vinorelbine Tartrate
Undergo blood and cerebrospinal fluid sample collection
Also known as: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
Undergo bone marrow aspiration
Undergo bone marrow biopsy
Also known as: Biopsy of Bone Marrow, Biopsy, Bone Marrow
Undergo bone scan
Also known as: Bone Scintigraphy
Undergo CT scan
Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography
Given IV and PO
Also known as: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518, B-518, B518, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamide Monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Frindovyx, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR 138719, WR- 138719, WR-138719, WR138719
Given IV
Also known as: Actinomycin A IV, Actinomycin C1, Actinomycin D, Actinomycin I1, Actinomycin IV, Actinomycin X 1, Actinomycin-[thr-val-pro-sar-meval], Cosmegen, DACT, Dactinomycine, Lyovac Cosmegen, Meractinomycin
Given IV
Also known as: Campto, Camptosar, Camptothecin 11, Camptothecin-11, CPT 11, CPT-11, CPT11, Irinomedac, Irinotecan Hydrochloride Trihydrate, Irinotecan Monohydrochloride Trihydrate, U 101440E, U-101440E, U101440E
Undergo lumbar puncture
Also known as: LP, Spinal Tap
Undergo lymph node biopsy
Also known as: Biopsy of Lymph Node
Undergo MRI
Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI
Undergo FDG PET scan
Also known as: Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure), Positron Emission Tomography Scan, Positron-Emission Tomography, PT
Undergo radiation therapy
Also known as: Cancer Radiotherapy, Energy Type, ENERGY_TYPE, Irradiate, Irradiated, Irradiation, Radiation, Radiation Therapy, NOS, Radiotherapeutics, Radiotherapy, RT, Therapy, Radiation
Undergo resection surgery
Also known as: Surgical Resection
Ancillary studies
Given IV
Also known as: Kyocristine, Leurocristine Sulfate, Leurocristine, sulfate, Oncovin, Vincasar, Vincosid, Vincrex, Vincristine, sulfate
Given IV
Also known as: Biovelbin, Eunades, KW 2307, KW-2307, KW2307, Navelbine, Navelbine Ditartrate, NVB, Vinorelbine Ditartrate
Event free survival (EFS)
Will be estimated using the Kaplan-Meier method and will be compared between the randomized treatment groups using the stratified log-rank test.
Time frame: From randomization until the first occurrence of progression or relapse, second malignancy, or death, assessed up to 5 years
Overall survival (OS)
Will be estimated using the Kaplan-Meier method and will be compared between the randomized treatment groups using the stratified log-rank test.
Time frame: From randomization to death from any cause, assessed up to 4 years
Clinician-reported adverse events (AEs)
Clinician-reported treatment-related grade 3 or higher AEs will be reported using Common Terminology Criteria for Adverse Events version 5.0. Comparison of toxicities will be conducted using Fisher's exact test. The maximum grade for each toxicity will be recorded for each patient. Averages and confidence intervals for these toxicity frequencies will be provided.
Time frame: Up to 5 years
Proportion of patients undergoing delayed primary excision (DPE) among patients deemed to be DPE eligible on retrospective central review
Eligibility for DPE will be established through a retrospective central review of diagnostic and pre-local control imaging. Among the patients deemed eligible for DPE, the proportion of those who undergo DPE will be determined along with 95% confidence interval. Cohen's kappa will be used to assess the concordance between site reviews and central reviews. 95% confidence of kappa statistics will be provided.
Time frame: Up to week 12 of treatment
Local failure rate for patients deemed eligible for DPE
Cumulative incidence curves will be used to present the local failure rates over time.
Time frame: Up to 5 years
Percentage of molecular biomarker testing that is resulted within the 6-week timeframe
Assessing the feasibility of reporting diagnostic tumor molecular features in patients with clinical group III intermediate risk rhabdomyosarcoma via the molecular characterization initiative (MCI) among the first 50 Clinical Group III patients who initiated treatment and consent to MCI. If 14 or more patients cannot have diagnostic tumor molecular features identified via MCI within 6 weeks of treatment, this aim will be considered not feasible.
Time frame: Up to cycle 3 (each cycle is 21 days)
Somatic molecular features TP53 mutation
Binary (1 = Mutation present, 0 = Mutation absent) no unit. Assessed via MCI to determine association with EFS and OS. EFS and OS will be estimated using KM method. The distribution will be compared by molecular features using log-rank test
Time frame: Up to 5 years
Somatic molecular features MYCN amplification
Binary (1 = Amplified, 0 = Not amplified) no unit. Assessed via MCI to determine association with EFS and OS. EFS and OS will be estimated using KM method. The distribution will be compared by molecular features using log-rank test.
Time frame: Up to 5 years
Somatic molecular features MYOD1 mutation
Binary (1 = Mutation present, 0 = Mutation absent) no unit. Assessed via MCI to determine association with EFS and OS. EFS and OS will be estimated using KM method. The distribution will be compared by molecular features using log-rank test.
Time frame: Up to 5 years
Somatic molecular features CDK4 amplification
Binary (1 = Not amplified, 0 = Amplified) no unit. Assessed via MCI to determine association with EFS and OS. EFS and OS will be estimated using KM method. The distribution will be compared by molecular features using log-rank test.
Time frame: Up to 5 years
Methylation patterns: EFS
Deoxyribonucleic acid (DNA) methylation data from rhabdomyosarcoma tumors collected during the conduct of ARST2531 will be analyzed. This data will be collected by array-based methods as part of MCI. The clinical significance of the rhabdomyosarcoma subsets determined in our DNA methylation studies will be analyzed. The association of DNA methylation subsets to EFS will be analyzed using the log-rank test and Cox model.
Time frame: Up to 5 years
Methylation patterns: OS
DNA methylation data from rhabdomyosarcoma tumors collected during the conduct of ARST2531 will be analyzed. This data will be collected by array-based methods as part of MCI. The clinical significance of the rhabdomyosarcoma subsets determined in our DNA methylation studies will be analyzed. The association of DNA methylation subsets to OS will be analyzed using the log-rank test and Cox model.
Time frame: Up to 5 years
Use of digital pathology/artificial intelligence: Risk predictions compared to EFS
Risk predictions will be recorded and ultimately compared to clinical outcomes, specifically 4-year EFS.
Time frame: 4 years from study enrollment
Use of digital pathology/artificial intelligence: Risk predictions compared to OS
Risk predictions will be recorded and ultimately compared to clinical outcomes, specifically 4-year OS.
Time frame: 4 years from study enrollment
Clinical practices of fertility discussions/procedures
Fertility consultation information (risk assessment, available fertility preservation options) will be collected and summarized using frequency and percentage. Frequency and percentage of eligible patients undergoing fertility preservation procedures will be summarized.
Time frame: Up to 5 years
Patient derived xenograft rhabdomyosarcoma model generation
Will viably collect tumor samples for generation of patient-derived xenograft models that can be used in future research studies to advance the understanding of rhabdomyosarcoma biology.
Time frame: Pre-treatment and cycle 3 (each cycle is 21 days)
Household material hardship (HMH)
HMH will be analyzed first as a binary variable (present/absent) and then as an ordinal (0-4) variable. Cox proportional hazard model will be used to assess the association between the binary HMH variable with EFS and OS. Then association between the ordinal measure and EFS and OS will be assessed using COX model.
Time frame: At time of survey completion, from enrollment until start of cycle 3 (each cycle is 21 days)
EFS by treatment arm within racial subgroups
Will be estimated using the Kaplan-Meier method. The corresponding 95% confidence interval (CI) will be estimated using Peto-peto method.
Time frame: Up to 5 years
EFS by treatment arm within ethnicity subgroups
Will be estimated using the Kaplan-Meier method. The corresponding 95% CI will be estimated using Peto-peto method.
Time frame: Up to 5 years
EFS by treatment arm within sex subgroups
Will be estimated using the Kaplan-Meier method. The corresponding 95% CI will be estimated using Peto-peto method.
Time frame: Up to 5 years
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Studies of the drug tested here: Cyclophosphamide