A Phase 3 interventional study of Cyclophosphamide and Dactinomycin in Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma and Embryonal Rhabdomyosarcoma, sponsored by National Cancer Institute (NCI). Active, not recruiting at 382 sites in 5 countries. Open to participants aged Up to 40 Years. Per ClinicalTrials.gov, last updated 2026-10-01.
Sponsored by National Cancer Institute (NCI) · Phase 3, Interventional, and Treatment
This randomized phase III trial studies how well combination chemotherapy (vincristine sulfate, dactinomycin, cyclophosphamide alternated with vincristine sulfate and irinotecan hydrochloride or vinorelbine) works compared to combination chemotherapy plus temsirolimus in treating patients with rhabdomyosarcoma (cancer that forms in the soft tissues, such as muscle), and has an intermediate chance of coming back after treatment (intermediate risk). Drugs used work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Combination chemotherapy and temsirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether chemotherapy plus temsirolimus is more effective than chemotherapy alone in treating patients with intermediate-risk rhabdomyosarcoma.
PRIMARY OBJECTIVE:
I. To compare the event-free survival (EFS) of patients with intermediate-risk (IR) rhabdomyosarcoma (RMS) treated with surgery, radiotherapy, and vincristine (vincristine sulfate), dactinomycin, and cyclophosphamide (VAC) alternating with vincristine and irinotecan (irinotecan hydrochloride) (VI) (VAC/VI) with maintenance to that of patients treated with surgery, radiotherapy and VAC/VI plus temsirolimus with maintenance.
SECONDARY OBJECTIVE:
I. To compare the overall survival (OS) of patients with IR RMS treated with surgery, radiotherapy, and VAC alternating with VI with maintenance to that of patients treated with surgery, radiotherapy and VAC/VI plus temsirolimus with maintenance.
EXPLORATORY OBJECTIVES:
I. To compare the outcome of patients based on their FOXO1 fusion gene partner, by evaluating PAX3 versus (vs) PAX7 in all patients found to be FOXO1 fusion positive.
II. To compare the outcome of patients based on their [F18]-fluorodeoxy-D-glucose-positron emission tomography (FDG-PET) response at week 9 (positive or negative), as assessed by Deauville criteria (5-point).
III. To estimate the frequency of patients with circulating tumor deoxyribonucleic acid (DNA) (ctDNA) at diagnosis and subsequent time-points, and explore whether tumor-specific somatic variants are detectable in the ctDNA.
IV. To compare the outcome of patients (VAC/VI with or without temsirolimus) who have received maintenance therapy on ARST1431 to those who received VAC/VI on ARST0531.
OUTLINE:
FEASIBILITY PHASE (THE FEASIBILITY PHASE IS COMPLETE, EFFECTIVE WITH AMENDMENT #1A): (\< 21 years old): This is a dose-escalation study of temsirolimus.
Patients receive vincristine sulfate intravenously (IV) over 1 minute on day 1 of weeks 1-13, 16, 17, 19, 20, 22-26, 28, 31-34, 37, 38, and 40, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, irinotecan hydrochloride IV over 90 minutes on days 1-5 of weeks 4, 10, 16, 19, 25, 31, and 37, and temsirolimus IV over 30-60 minutes on days 1, 8, and 15. Patients also undergo radiation therapy (RT) beginning week 13 for up to 6.5 weeks. Courses with temsirolimus repeat every 21 days for 12 weeks in the absence of disease progression or unacceptable toxicity.
EFFICACY PHASE: Patients are randomized to Regimen A or B. Patients with disease that is ARMS FOXO1 fusion negative (stage I, group I/II, stage 1, group III [orbit] or stage II, group I/II) are assigned to Regimen C.
REGIMEN A (VAC/VI): Patients receive vincristine sulfate IV over 1 minute on day 1 of weeks 1-13, 16, 17, 19, 20, 22-26, 28, 31-34, 37, 38, and 40, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, irinotecan hydrochloride IV over 90 minutes on days 1-5 of weeks 4, 10, 16, 19, 25, 31, and 37. Patients also undergo primary site RT beginning at week 13 or metastatic site RT beginning at week 43 for up to 6.5 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients then receive vinorelbine IV over 6-10 minutes on days 1, 8, and 15 and cyclophosphamide orally (PO) once daily (QD) on days 1-28. Cycles repeats every 28 days for 24 weeks in the absence of disease progression or unacceptable toxicity.
REGIMEN B (VAC/VI TEMSIROLIMUS): Patients receive vincristine sulfate IV over 1 minute on day 1 of weeks 1-13, 16, 17, 19, 20, 22-26, 28, 31-34, 37, 38, and 40, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, irinotecan hydrochloride IV over 90 minutes on days 1-5 of weeks 4, 10, 16, 19, 25, 31, and 37 and temsirolimus IV over 30-60 minutes on day 1 of weeks 1-12 and 21-42. Patients also undergo RT as in Regimen A. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients then receive vinorelbine IV over 6-10 minutes on days 1, 8, and 15 and cyclophosphamide PO QD on days 1-28. Cycles repeats every 28 days for 24 weeks in the absence of disease progression or unacceptable toxicity.
MAINTENANCE PHASE (Patients in Regimen A or Regimen B): Patients receive vinorelbine IV over 6-10 minutes on days 1, 8, and 15 and cyclophosphamide orally (PO) once daily (QD) on days 1-28. Treatment repeats every 28 days for up to 6 cycles.
REGIMEN C (FOXO1 FUSION NEGATIVE): Patients receive vincristine sulfate IV over 1 minute on day 1 of weeks 1-10 and 13-22, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 4, 7, 10, 13, 16, 19, and 22, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 4, 7, and 10. Patients undergo RT beginning at week 13 for up to 6.5 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for 2 years, every 6 months for 1 year, and then annually for up to 10 years.
9 studies on the registry are indexed under Rhabdomyosarcoma, Embryonal; 3 are open to participants now.
Browse Rhabdomyosarcoma, Embryonal studies →National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.
Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) 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.
RMS types included under embryonal rhabdomyosarcoma (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 2013 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); classification of alveolar rhabdomyosarcoma (ARMS) in the 2013 WHO classification is the same as in the ICR and includes classic and solid variants
ERMS
ARMS:
or radioisotope (GFR) >= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/gender as follows:
Exclusion Criteria:
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.
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
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.
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.
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.
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.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
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.
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.
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.
Patients receive vincristine sulfate IV over 1 minute on day 1 of weeks 1-13, 16, 17, 19, 20, 22-26, 28, 31-34, 37, 38, and 40, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, irinotecan hydrochloride IV over 90 minutes on days 1-5 of weeks 4, 10, 16, 19, 25, 31, and 37. Patients also undergo primary site RT beginning at week 13 or metastatic site RT beginning at week 43 for up to 6.5 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients then receive vinorelbine IV over 6-10 minutes on days 1, 8, and 15 and cyclophosphamide PO QD on days 1-28. Cycles repeats every 28 days for 24 weeks in the absence of disease progression or unacceptable toxicity.
Drug: Cyclophosphamide · Biological: Dactinomycin · Drug: Irinotecan Hydrochloride · Other: Laboratory Biomarker Analysis · Other: Questionnaire Administration · Radiation: Radiation Therapy · Drug: Vincristine Sulfate
Patients receive vincristine sulfate IV over 1 minute on day 1 of weeks 1-13, 16, 17, 19, 20, 22-26, 28, 31-34, 37, 38, and 40, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, irinotecan hydrochloride IV over 90 minutes on days 1-5 of weeks 4, 10, 16, 19, 25, 31, and 37 and temsirolimus IV over 30-60 minutes on day 1 of weeks 1-12 and 21-42. Patients also undergo RT as in Regimen A. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients then receive vinorelbine IV over 6-10 minutes on days 1, 8, and 15 and cyclophosphamide PO QD on days 1-28. Cycles repeats every 28 days for 24 weeks in the absence of disease progression or unacceptable toxicity.
Drug: Cyclophosphamide · Biological: Dactinomycin · Drug: Irinotecan Hydrochloride · Other: Laboratory Biomarker Analysis · Other: Questionnaire Administration · Radiation: Radiation Therapy · Drug: Temsirolimus · Drug: Vincristine Sulfate · Drug: Vinorelbine
Patients receive vincristine sulfate IV over 1 minute on day 1 of weeks 1-10 and 13-22, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 4, 7, 10, 13, 16, 19, and 22, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 4, 7, and 10. Patients undergo RT beginning at week 13 for up to 6.5 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.
Drug: Cyclophosphamide · Biological: Dactinomycin · Other: Laboratory Biomarker Analysis · Other: Questionnaire Administration · Radiation: Radiation Therapy · Drug: Vincristine Sulfate
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
Correlative studies
Ancillary studies
Undergo RT
Also known as: Cancer Radiotherapy, Energy Type, ENERGY_TYPE, Irradiate, Irradiated, Irradiation, Radiation, Radiation Therapy, NOS, Radiotherapeutics, Radiotherapy, RT, Therapy, Radiation
Given IV
Also known as: CCI 779, CCI-779, CCI-779 Rapamycin Analog, CCI779, Cell Cycle Inhibitor 779, Rapamycin Analog, Rapamycin Analog CCI-779, Torisel
Given IV
Also known as: Kyocristine, Leurocristine Sulfate, Leurocristine, sulfate, Oncovin, Vincasar, Vincosid, Vincrex, Vincristine, sulfate
Given IV
Also known as: Dihydroxydeoxynorvinkaleukoblastine
Event-free Survival (EFS)
The Kaplan-Meier method will used to estimate 3-year EFS between the randomized treatment groups (Regimen A (VAC/VI) vs. Regimen B (VAC/VI/Temsirolimus)) using the log-rank test. EFS event is defined as the time from study entry until first occurrence of progression, relapse, second malignancy, or death.
Time frame: Up to 3 years from study entry
Overall Survival (OS)
The Kaplan-Meier method will used to estimate 3-year OS between the randomized treatment groups (Regimen A (VAC/VI) vs. Regimen B (VAC/VI/Temsirolimus)) using the log-rank test. OS event is defined as the time from study entry until death.
Time frame: Up to 3 years from study entry
Frequency of Circulating Tumor DNA (ctDNA) at Diagnosis and Subsequent Time-points
Will determine the frequency with which of ctDNA is detected in patients with intermediate-risk (IR) rhabdomyosarcoma (RMS) at diagnosis and how these levels change over time with. In addition, the level of ctDNA as a percent of total cell-free DNA will be summarized by reporting the median and range. Since no prior data are available on the rate of positivity nor the change over time, the analysis will be purely descriptive. Changes over time will be plotted to visually determine and compare the trajectories of ctDNA during the early and late stages of therapy.
Time frame: Up to 10 years
Patient Outcome Based on Their Forkhead Box O1 Protein (FOXO1) Partner, by Evaluating PAX3 vs. PAX7 in All Patients Found to be FOXO1 Fusion Positive
Time frame: Up to 10 years
Patient Outcome Based on Their [F18]-Fluorodeoxy-D-glucose-positron Emission Tomography (FDG-positron Emission Tomography [PET]) Response
Assessed by Deauville Criteria (5-point).
Time frame: At week 9
Patient Outcomes Based on (Vincristine, Dactinomycin, and Cyclophosphamide [VAC]/Vincristine and Irinotecan [VI] With or Without Temsirolimus) Who Have Received Maintenance Therapy on ARST1431 to Those Who Received VAC/VI on ARST0531
The two-sample log-rank test will be performed.
Time frame: Up to 10 years
This study was opened for enrollment on May 23, 2016, and was temporarily closed to accrual on September 23, 2016 for feasibility review. The study was re-opened for enrollment on January 2, 2018. The study was temporarily closed again on August 22, 2018 and re-opened on December 31, 2018 with the addition of maintenance therapy. The study is closed to accrual since 1/6/2022.
| Milestone | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) | Regimen C (FOXO1 Fusion Negative, VAC/VA) | Feasibility (VAC/VI/Temsirolimus) |
|---|---|---|---|---|
| Started | 156 | 156 | 3 | 10 |
| Completed | 150 | 147 | 3 | 9 |
| Not completed | 6 | 9 | 0 | 1 |
| Withdrew: Death | 1 | 1 | 0 | 0 |
| Withdrew: Ineligible | 1 | 3 | 0 | 0 |
| Withdrew: Withdrawal of consent for any further data submissions | 4 | 5 | 0 | 1 |
The Kaplan-Meier method will used to estimate 3-year EFS between the randomized treatment groups (Regimen A (VAC/VI) vs. Regimen B (VAC/VI/Temsirolimus)) using the log-rank test. EFS event is defined as the time from study entry until first occurrence of progression, relapse, second malignancy, or death.
| Percentage of participants | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) |
|---|---|---|
| Event-free Survival (EFS) | 64.80 (55.5 to 74.1) | 66.80 (57.5 to 76.2) |
The Kaplan-Meier method will used to estimate 3-year OS between the randomized treatment groups (Regimen A (VAC/VI) vs. Regimen B (VAC/VI/Temsirolimus)) using the log-rank test. OS event is defined as the time from study entry until death.
| Percentage of participants | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) |
|---|---|---|
| Overall Survival (OS) | 78.70 (70.9 to 86.4) | 77.80 (69.7 to 85.9) |
Will determine the frequency with which of ctDNA is detected in patients with intermediate-risk (IR) rhabdomyosarcoma (RMS) at diagnosis and how these levels change over time with. In addition, the level of ctDNA as a percent of total cell-free DNA will be summarized by reporting the median and range. Since no prior data are available on the rate of positivity nor the change over time, the analysis will be purely descriptive. Changes over time will be plotted to visually determine and compare the trajectories of ctDNA during the early and late stages of therapy.
Results for this outcome have not been posted.
Results for this outcome have not been posted.
Assessed by Deauville Criteria (5-point).
Results for this outcome have not been posted.
The two-sample log-rank test will be performed.
Results for this outcome have not been posted.
Collected over Serious and Other (Not Including Serious) Adverse Events monitored/assessed up to 66 weeks from study entry. All-Cause Mortality monitored/assessed up to 7 years and 4 months from study entry.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Regimen A (VAC/VI) | 33/155 (21.3%) | 17/155 (11%) | 139/155 (89.7%) |
| Regimen B (VAC/VI/Temsirolimus) | 33/153 (21.6%) | 77/153 (50.3%) | 141/153 (92.2%) |
| Regimen C (FOXO1 Fusion Negative, VAC/VA) | 0/3 (0%) | 0/3 (0%) | 1/3 (33.3%) |
| Feasibility (VAC/VI/Temsirolimus) | 4/10 (40%) | 6/10 (60%) | 8/10 (80%) |
| Event | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) | Regimen C (FOXO1 Fusion Negative, VAC/VA) | Feasibility (VAC/VI/Temsirolimus) |
|---|---|---|---|---|
| Mucositis oralGastrointestinal disorders | 3/155 | 35/153 | 0/3 | 2/10 |
| AnaphylaxisImmune system disorders | 0/155 | 0/153 | 0/3 | 1/10 |
| DehydrationMetabolism and nutrition disorders | 0/155 | 13/153 | 0/3 | 1/10 |
| Dermatitis radiationInjury, poisoning and procedural complications | 0/155 | 0/153 | 0/3 | 1/10 |
| DiarrheaGastrointestinal disorders | 1/155 | 7/153 | 0/3 | 1/10 |
| HyponatremiaMetabolism and nutrition disorders | 0/155 | 0/153 | 0/3 | 1/10 |
| Lung infectionInfections and infestations | 0/155 | 1/153 | 0/3 | 1/10 |
| PneumonitisRespiratory, thoracic and mediastinal disorders | 0/155 | 1/153 | 0/3 | 1/10 |
| SepsisInfections and infestations | 1/155 | 4/153 | 0/3 | 1/10 |
| Skin infectionInfections and infestations | 0/155 | 1/153 | 0/3 | 1/10 |
| Event | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) | Regimen C (FOXO1 Fusion Negative, VAC/VA) | Feasibility (VAC/VI/Temsirolimus) |
|---|---|---|---|---|
| AnemiaBlood and lymphatic system disorders | 107/155 | 117/153 | 1/3 | 3/10 |
| Neutrophil count decreasedInvestigations | 107/155 | 109/153 | 1/3 | 6/10 |
| White blood cell decreasedInvestigations | 103/155 | 103/153 | 1/3 | 4/10 |
| Lymphocyte count decreasedInvestigations | 79/155 | 81/153 | 0/3 | 2/10 |
| Febrile neutropeniaBlood and lymphatic system disorders | 56/155 | 61/153 | 0/3 | 2/10 |
| Platelet count decreasedInvestigations | 49/155 | 61/153 | 0/3 | 2/10 |
| NauseaGastrointestinal disorders | 56/155 | 52/153 | 0/3 | 3/10 |
| Mucositis oralGastrointestinal disorders | 30/155 | 55/153 | 0/3 | 2/10 |
| Dermatitis radiationInjury, poisoning and procedural complications | 17/155 | 18/153 | 1/3 | 0/10 |
| PainGeneral disorders | 5/155 | 8/153 | 1/3 | 1/10 |
| Age, Categorical(Participants) | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) | Regimen C (FOXO1 Fusion Negative, VAC/VA) | Feasibility (VAC/VI/Temsirolimus) | Total |
|---|---|---|---|---|---|
| <=18 years | 145 | 140 | 3 | 10 | 298 |
| Between 18 and 65 years | 11 | 16 | 0 | 0 | 27 |
| >=65 years | 0 | 0 | 0 | 0 | 0 |
| Age, Continuous(years) | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) | Regimen C (FOXO1 Fusion Negative, VAC/VA) | Feasibility (VAC/VI/Temsirolimus) | Total |
|---|---|---|---|---|---|
| Median | 6.57 (0.04 to 35.84) | 6.05 (0.35 to 32.91) | 5.71 (3.18 to 6.58) | 4.59 (0.17 to 14.37) | 6.34 (0.04 to 35.84) |
| Sex: Female, Male(Participants) | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) | Regimen C (FOXO1 Fusion Negative, VAC/VA) | Feasibility (VAC/VI/Temsirolimus) | Total |
|---|---|---|---|---|---|
| Female | 71 | 55 | 0 | 3 | 129 |
| Male | 85 | 101 | 3 | 7 | 196 |
| Ethnicity (NIH/OMB)(Participants) | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) | Regimen C (FOXO1 Fusion Negative, VAC/VA) | Feasibility (VAC/VI/Temsirolimus) | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 29 | 17 | 2 | 1 | 49 |
| Not Hispanic or Latino | 113 | 117 | 1 | 9 | 240 |
| Unknown or Not Reported | 14 | 22 | 0 | 0 | 36 |
| Race (NIH/OMB)(Participants) | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) | Regimen C (FOXO1 Fusion Negative, VAC/VA) | Feasibility (VAC/VI/Temsirolimus) | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 2 | 0 | 0 | 0 | 2 |
| Asian | 5 | 4 | 0 | 0 | 9 |
| Native Hawaiian or Other Pacific Islander | 0 | 1 | 0 | 0 | 1 |
| Black or African American | 20 | 16 | 0 | 1 | 37 |
| White | 99 | 106 | 2 | 8 | 215 |
| More than one race | 3 | 3 | 0 | 1 | 7 |
| Unknown or Not Reported | 27 | 26 | 1 | 0 | 54 |
| Region of Enrollment(participants) | Regimen A (VAC/VI) | Regimen B (VAC/VI/Temsirolimus) | Regimen C (FOXO1 Fusion Negative, VAC/VA) | Feasibility (VAC/VI/Temsirolimus) | Total |
|---|---|---|---|---|---|
| United States | 141 | 135 | 3 | 10 | 289 |
| Australia | 6 | 11 | 0 | 0 | 17 |
| Canada | 6 | 9 | 0 | 0 | 15 |
| New Zealand | 3 | 1 | 0 | 0 | 4 |
Showing the first 100 of 382 sites across 5 countries. Choose a country and city above to see the rest.
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jun 2016 to primary completion in Jun 2023, 84 months.
Results were first posted in Oct 2024, 1 year 3 months after primary completion.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Cyclophosphamide · Dactinomycin · Vincristine
National Cancer Institute (NCI) →