Alveolar RhabdomyosarcomaPhase 3NCT02567435
Combination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma
A Phase 3 study testing Cyclophosphamide, Dactinomycin and Irinotecan Hydrochloride in people with Alveolar Rhabdomyosarcoma and Botryoid-Type Embryonal Rhabdomyosarcoma, run by National Cancer Institute (NCI) at 382 sites in 5 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages Up to 40 years
- Conditions the study is for
- Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma, Embryonal Rhabdomyosarcoma
- What is being tested?
- Cyclophosphamide, Dactinomycin and Irinotecan Hydrochloride
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 325 participants
- Where
- 382 sitesin 5 countries
- Study dates
- 1 Jun 2016to 27 Oct 2026
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Feasibility Phase: Patients must be < 21 years of age at the time of enrollment; please note: the feasibility phase is complete, effective with amendment #1
- Efficacy Phase: Patients must be =< 40 years of age at the time of enrollment
- Patients with newly diagnosed RMS of any subtype, except adult-type pleomorphic, based upon institutional histopathologic classification, are eligible to enroll on the study based upon stage, group, and age, as below
- 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
- Stage 1, group III (non-orbit)
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- Stage 3, group I/II
- Stage 2/3, group III
- Stage 4, group IV, < 10 years old
- ARMS:
- Stages 1-3, groups I-III
- Specimen Submission: Patients must have sufficient tissue available for the required biology study
- Lansky performance status score >= 50 for patients =< 16 years of age; Karnofsky performance status score >= 50 for patients > 16 years of age
- Peripheral absolute neutrophil count (ANC) >= 750/uL
- Platelet count >= 75,000/uL
- Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
- 1 month to < 6 months old: 0.4 mg/dl (male), 0.4 mg/dl (female)
- 6 months to < 1 year old: 0.5 mg/dl (male), 0.5 mg/dl (female)
- 1 to < 2 years old: 0.6 mg/dl (male), 0.6 mg/dl (female)
- 2 to < 6 years old: 0.8 mg/dl (male), 0.8 mg/dl (female)
- 6 to < 10 years old: 1 mg/dl (male), 1 mg/dl (female)
- 10 to < 13 years old: 1.2 mg/dl (male), 1.2 mg/dl (female)
- 13 to < 16 years old: 1.5 mg/dl (male), 1.4 mg/dl (female)
- >= 16 years old: 1.7 mg/dl (male), 1.4 mg/dl (female)
- Patients with an elevated serum creatinine due to obstructive hydronephrosis secondary to tumor are still eligible; however, patients with urinary tract obstruction by tumor must have unimpeded urinary flow established via diversion (i.e. percutaneous nephrostomies or ureteric stents) of the urinary tract
- Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
- 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.
Who cannot join
- Patients who have previously received temsirolimus, another mTOR inhibitor, or any other investigational agent
- Patients who have received any chemotherapy (excluding steroids) and/or RT prior to this enrollment
- Patients with uncontrolled hyperglycemia
- Patients with uncontrolled hyperlipidemia
- Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for at least 3 months after treatment is completed
- Female patients who are pregnant are not eligible since fetal toxicities or teratogenic effects have been noted for several of the study drugs; Note: A pregnancy test is required for female patients of childbearing potential prior to study entry
- Lactating females who plan to breastfeed their infants are not eligible
Trial locations and contacts
382 sites in 5 countries.
327 sites in 5 countries
United States300 sites
Providence Regional Cancer System-Aberdeen
Aberdeen, Washington, 98520
Children's Hospital Medical Center of Akron
Akron, Ohio, 44308
Albany Medical Center
Albany, New York, 12208
Presbyterian Hospital
Albuquerque, New Mexico, 87106
Lehigh Valley Hospital-Cedar Crest
Allentown, Pennsylvania, 18103
Texas Tech University Health Sciences Center-Amarillo
Amarillo, Texas, 79106
Kaiser Permanente-Anaheim
Anaheim, California, 92806
Anchorage Associates in Radiation Medicine
Anchorage, Alaska, 98508
Anchorage Radiation Therapy Center
Anchorage, Alaska, 99504
Alaska Breast Care and Surgery LLC
Anchorage, Alaska, 99508
Alaska Oncology and Hematology LLC
Anchorage, Alaska, 99508
Alaska Women's Cancer Care
Anchorage, Alaska, 99508
Anchorage Oncology Centre
Anchorage, Alaska, 99508
Katmai Oncology Group
Anchorage, Alaska, 99508
Providence Alaska Medical Center
Anchorage, Alaska, 99508
C S Mott Children's Hospital
Ann Arbor, Michigan, 48109
University of Michigan Rogel Cancer Center
Ann Arbor, Michigan, 48109
PCR Oncology
Arroyo Grande, California, 93420
Mission Hospital
Asheville, North Carolina, 28801
Children's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, 30329
Augusta University Medical Center
Augusta, Georgia, 30912
Children's Hospital Colorado
Aurora, Colorado, 80045
Dell Children's Medical Center of Central Texas
Austin, Texas, 78723
University of Maryland/Greenebaum Cancer Center
Baltimore, Maryland, 21201
Sinai Hospital of Baltimore
Baltimore, Maryland, 21215
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287
Eastern Maine Medical Center
Bangor, Maine, 04401
Bronson Battle Creek
Battle Creek, Michigan, 49017
Overlake Medical Center
Bellevue, Washington, 98004
Kaiser Permanente-Bellflower
Bellflower, California, 90706
PeaceHealth Saint Joseph Medical Center
Bellingham, Washington, 98225
Saint Charles Health System
Bend, Oregon, 97701
Central Vermont Medical Center/National Life Cancer Treatment
Berlin Corners, Vermont, 05602
Walter Reed National Military Medical Center
Bethesda, Maryland, 20889-5600
Lehigh Valley Hospital - Muhlenberg
Bethlehem, Pennsylvania, 18017
Children's Hospital of Alabama
Birmingham, Alabama, 35233
Prisma Health Cancer Institute - Spartanburg
Boiling Springs, South Carolina, 29316
Saint Luke's Cancer Institute - Boise
Boise, Idaho, 83712
Tufts Children's Hospital
Boston, Massachusetts, 02111
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
United Hospital Center
Bridgeport, West Virginia, 26330
Maimonides Medical Center
Brooklyn, New York, 11219
Roswell Park Cancer Institute
Buffalo, New York, 14263
Providence Saint Joseph Medical Center/Disney Family Cancer Center
Burbank, California, 91505
University of Vermont Medical Center
Burlington, Vermont, 05401
University of Vermont and State Agricultural College
Burlington, Vermont, 05405
Carson Tahoe Regional Medical Center
Carson City, Nevada, 89703
Providence Regional Cancer System-Centralia
Centralia, Washington, 98531
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27599
Medical University of South Carolina
Charleston, South Carolina, 29425
Vandalia Health/CAMC Institute for Academic Medicine
Charleston, West Virginia, 25304
Carolinas Medical Center/Levine Cancer Institute
Charlotte, North Carolina, 28203
Novant Health Presbyterian Medical Center
Charlotte, North Carolina, 28204
Novant Health Carolina Surgical - Randolph
Charlotte, North Carolina, 28207
Oncology Specialists of Charlotte
Charlotte, North Carolina, 28207
Southern Oncology Specialists-Charlotte
Charlotte, North Carolina, 28262
University of Virginia Cancer Center
Charlottesville, Virginia, 22908
T C Thompson Children's Hospital
Chattanooga, Tennessee, 37403
Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611
John H Stroger Jr Hospital of Cook County
Chicago, Illinois, 60612
Rush MD Anderson Cancer Center
Chicago, Illinois, 60612
University of Illinois
Chicago, Illinois, 60612
University of Chicago Comprehensive Cancer Center
Chicago, Illinois, 60637
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Siteman Cancer Center at Saint Peters Hospital
City of Saint Peters, Missouri, 63376
Clackamas Radiation Oncology Center
Clackamas, Oregon, 97015
Providence Cancer Institute Clackamas Clinic
Clackamas, Oregon, 97015
Rainbow Babies and Childrens Hospital
Cleveland, Ohio, 44106
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Prisma Health Cancer Institute - Laurens
Clinton, South Carolina, 29325
University of Missouri Children's Hospital
Columbia, Missouri, 65212
Prisma Health Richland Hospital
Columbia, South Carolina, 29203
Nationwide Children's Hospital
Columbus, Ohio, 43205
Bay Area Hospital
Coos Bay, Oregon, 97420
Driscoll Children's Hospital
Corpus Christi, Texas, 78411
Siteman Cancer Center at West County Hospital
Creve Coeur, Missouri, 63141
Medical City Dallas Hospital
Dallas, Texas, 75230
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, 75390
Geisinger Medical Center
Danville, Pennsylvania, 17822
Dayton Children's Hospital
Dayton, Ohio, 45404
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Denver, Colorado, 80218
Blank Children's Hospital
Des Moines, Iowa, 50309
Iowa Methodist Medical Center
Des Moines, Iowa, 50309
Iowa Lutheran Hospital
Des Moines, Iowa, 50316
Wayne State University/Karmanos Cancer Institute
Detroit, Michigan, 48201
Henry Ford Health Saint John Hospital
Detroit, Michigan, 48236
Kaiser Permanente Downey Medical Center
Downey, California, 90242
Essentia Health Cancer Center
Duluth, Minnesota, 55805
Duke University Medical Center
Durham, North Carolina, 27710
Prisma Health Cancer Institute - Easley
Easley, South Carolina, 29640
Michigan State University
East Lansing, Michigan, 48823
Swedish Cancer Institute-Edmonds
Edmonds, Washington, 98026
El Paso Children's Hospital
El Paso, Texas, 79905
Providence Regional Cancer Partnership
Everett, Washington, 98201
Inova Fairfax Hospital
Falls Church, Virginia, 22042
Sanford Broadway Medical Center
Fargo, North Dakota, 58122
Hurley Medical Center
Flint, Michigan, 48503
Kaiser Permanente-Fontana
Fontana, California, 92335
Broward Health Medical Center
Fort Lauderdale, Florida, 33316
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida, 33908
Saint Luke's Cancer Institute - Fruitland
Fruitland, Idaho, 83619
UF Health Cancer Institute - Gainesville
Gainesville, Florida, 32610
Glens Falls Hospital
Glens Falls, New York, 12801
Corewell Health Grand Rapids Hospitals - Butterworth Hospital
Grand Rapids, Michigan, 49503
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Trinity Health Grand Rapids Hospital
Grand Rapids, Michigan, 49503
East Carolina University
Greenville, North Carolina, 27834
Saint Francis Hospital
Greenville, South Carolina, 29601
BI-LO Charities Children's Cancer Center
Greenville, South Carolina, 29605
Prisma Health Cancer Institute - Butternut
Greenville, South Carolina, 29605
Prisma Health Cancer Institute - Faris
Greenville, South Carolina, 29605
Saint Francis Cancer Center
Greenville, South Carolina, 29607
Prisma Health Cancer Institute - Eastside
Greenville, South Carolina, 29615
Prisma Health Cancer Institute - Greer
Greer, South Carolina, 29650
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Cancer and Blood Specialists-Henderson
Henderson, Nevada, 89052
Comprehensive Cancer Centers of Nevada - Henderson
Henderson, Nevada, 89052
Comprehensive Cancer Centers of Nevada-Horizon Ridge
Henderson, Nevada, 89052
Las Vegas Cancer Center-Henderson
Henderson, Nevada, 89052
Comprehensive Cancer Centers of Nevada-Southeast Henderson
Henderson, Nevada, 89074
Las Vegas Urology - Green Valley
Henderson, Nevada, 89074
Las Vegas Urology - Pebble
Henderson, Nevada, 89074
Oncology Las Vegas - Henderson
Henderson, Nevada, 89074
Urology Specialists of Nevada - Green Valley
Henderson, Nevada, 89074
Penn State Children's Hospital
Hershey, Pennsylvania, 17033
Memorial Regional Hospital/Joe DiMaggio Children's Hospital
Hollywood, Florida, 33021
Straub Clinic and Hospital
Honolulu, Hawaii, 96813
Kaiser Permanente Moanalua Medical Center
Honolulu, Hawaii, 96819
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96826
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, 77030
UT MD Anderson Cancer Center
Houston, Texas, 77030
Novant Health Cancer Institute - Huntersville
Huntersville, North Carolina, 28078
Novant Health Presbyterian Medical Center Huntersville
Huntersville, North Carolina, 28078
Southern Oncology Specialists-Huntersville
Huntersville, North Carolina, 28078
Riley Hospital for Children
Indianapolis, Indiana, 46202
Ascension Saint Vincent Indianapolis Hospital
Indianapolis, Indiana, 46260
University of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa, 52242
Swedish Cancer Institute-Issaquah
Issaquah, Washington, 98029
University of Mississippi Medical Center
Jackson, Mississippi, 39216
Nemours Children's Clinic-Jacksonville
Jacksonville, Florida, 32207
Bronson Methodist Hospital
Kalamazoo, Michigan, 49007
West Michigan Cancer Center
Kalamazoo, Michigan, 49007
Beacon Kalamazoo
Kalamazoo, Michigan, 49048
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108
Kadlec Clinic Hematology and Oncology
Kennewick, Washington, 99336
Kingman Regional Medical Center
Kingman, Arizona, 86401
East Tennessee Childrens Hospital
Knoxville, Tennessee, 37916
Gundersen Lutheran Medical Center
La Crosse, Wisconsin, 54601
Providence Regional Cancer System-Lacey
Lacey, Washington, 98503
University of Michigan Health - Sparrow Lansing
Lansing, Michigan, 48912
Las Vegas Urology - Pecos
Las Vegas, Nevada, 89074
Desert West Surgery
Las Vegas, Nevada, 89102
OptumCare Cancer Care at Charleston
Las Vegas, Nevada, 89102
University Medical Center of Southern Nevada
Las Vegas, Nevada, 89102
Cancer and Blood Specialists-Shadow
Las Vegas, Nevada, 89106
Radiation Oncology Centers of Nevada Central
Las Vegas, Nevada, 89106
Urology Specialists of Nevada - Central
Las Vegas, Nevada, 89106
HealthCare Partners Medical Group Oncology/Hematology-Maryland Parkway
Las Vegas, Nevada, 89109
Sunrise Hospital and Medical Center
Las Vegas, Nevada, 89109
HealthCare Partners Medical Group Oncology/Hematology-San Martin
Las Vegas, Nevada, 89113
Las Vegas Urology - Sunset
Las Vegas, Nevada, 89113
Urology Specialists of Nevada - Southwest
Las Vegas, Nevada, 89113
Radiation Oncology Centers of Nevada Southeast
Las Vegas, Nevada, 89119
Cancer Therapy and Integrative Medicine
Las Vegas, Nevada, 89121
Ann M Wierman MD LTD
Las Vegas, Nevada, 89128
Cancer and Blood Specialists-Tenaya
Las Vegas, Nevada, 89128
Comprehensive Cancer Centers of Nevada - Northwest
Las Vegas, Nevada, 89128
HealthCare Partners Medical Group Oncology/Hematology-Tenaya
Las Vegas, Nevada, 89128
Las Vegas Urology - Cathedral Rock
Las Vegas, Nevada, 89128
Las Vegas Urology - Smoke Ranch
Las Vegas, Nevada, 89128
Oncology Las Vegas - Tenaya
Las Vegas, Nevada, 89128
OptumCare Cancer Care at MountainView
Las Vegas, Nevada, 89128
Urology Specialists of Nevada - Northwest
Las Vegas, Nevada, 89128
Alliance for Childhood Diseases/Cure 4 the Kids Foundation
Las Vegas, Nevada, 89135
Comprehensive Cancer Centers of Nevada - Town Center
Las Vegas, Nevada, 89144
Comprehensive Cancer Centers of Nevada-Summerlin
Las Vegas, Nevada, 89144
Summerlin Hospital Medical Center
Las Vegas, Nevada, 89144
Las Vegas Cancer Center-Medical Center
Las Vegas, Nevada, 89148-2405
Comprehensive Cancer Centers of Nevada
Las Vegas, Nevada, 89148
HealthCare Partners Medical Group Oncology/Hematology-Centennial Hills
Las Vegas, Nevada, 89149
Comprehensive Cancer Centers of Nevada - Central Valley
Las Vegas, Nevada, 89169
University Cancer Center
Las Vegas, Nevada, 89169
OptumCare Cancer Care at Fort Apache
Las Vegas, Nevada, 89183
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
Lebanon, New Hampshire, 03756
University of Kentucky/Markey Cancer Center
Lexington, Kentucky, 40536
Arkansas Children's Hospital
Little Rock, Arkansas, 72202-3591
Loma Linda University Medical Center
Loma Linda, California, 92354
PeaceHealth Saint John Medical Center
Longview, Washington, 98632
Children's Hospital Los Angeles
Los Angeles, California, 90027
Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, 90027
Mattel Children's Hospital UCLA
Los Angeles, California, 90095
UCLA / Jonsson Comprehensive Cancer Center
Los Angeles, California, 90095
Norton Children's Hospital
Louisville, Kentucky, 40202
Palms West Radiation Therapy
Loxahatchee Groves, Florida, 33470
Covenant Children's Hospital
Lubbock, Texas, 79410
UMC Cancer Center / UMC Health System
Lubbock, Texas, 79415
Texas Tech University Health Sciences Center-Lubbock
Lubbock, Texas, 79430
Atrium Health Navicent
Macon, Georgia, 31201
Valley Children's Hospital
Madera, California, 93636
University of Wisconsin Carbone Cancer Center - University Hospital
Madison, Wisconsin, 53792
Marshfield Medical Center-Marshfield
Marshfield, Wisconsin, 54449
WVUH-Berkely Medical Center
Martinsburg, West Virginia, 25401
Matthews Radiation Oncology Center
Matthews, North Carolina, 28105
Novant Health Cancer Institute - Matthews
Matthews, North Carolina, 28105
Loyola University Medical Center
Maywood, Illinois, 60153
Saint Luke's Cancer Institute - Meridian
Meridian, Idaho, 83642
Banner Children's at Desert
Mesa, Arizona, 85202
University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, Florida, 33136
Nicklaus Children's Hospital
Miami, Florida, 33155
Miami Cancer Institute
Miami, Florida, 33176
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
NYU Langone Hospital - Long Island
Mineola, New York, 11501
Children's Hospitals and Clinics of Minnesota - Minneapolis
Minneapolis, Minnesota, 55404
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455
Saint Patrick Hospital - Community Hospital
Missoula, Montana, 59802
USA Health Strada Patient Care Center
Mobile, Alabama, 36604
Novant Health Cancer Institute - Mooresville
Mooresville, North Carolina, 28117
West Virginia University Healthcare
Morgantown, West Virginia, 26506
Morristown Medical Center
Morristown, New Jersey, 07960
Trinity Health Muskegon Hospital
Muskegon, Michigan, 49444
Saint Luke's Cancer Institute - Nampa
Nampa, Idaho, 83687
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, 37203
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, 37232
Saint Peter's University Hospital
New Brunswick, New Jersey, 08901
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital
New Brunswick, New Jersey, 08903
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08903
Smilow Cancer Center/Yale-New Haven Hospital
New Haven, Connecticut, 06510
Yale University
New Haven, Connecticut, 06520
The Steven and Alexandra Cohen Children's Medical Center of New York
New Hyde Park, New York, 11040
Children's Hospital New Orleans
New Orleans, Louisiana, 70118
Ochsner Medical Center Jefferson
New Orleans, Louisiana, 70121
Laura and Isaac Perlmutter Cancer Center at NYU Langone
New York, New York, 10016
Mount Sinai Hospital
New York, New York, 10029
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York, New York, 10032
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Newark Beth Israel Medical Center
Newark, New Jersey, 07112
Providence Newberg Medical Center
Newberg, Oregon, 97132
Corewell Health Lakeland Hospitals - Niles Hospital
Niles, Michigan, 49120
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
Advocate Children's Hospital-Oak Lawn
Oak Lawn, Illinois, 60453
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Kaiser Permanente-Oakland
Oakland, California, 94611
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Children's Hospital and Medical Center of Omaha
Omaha, Nebraska, 68114
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Children's Hospital of Orange County
Orange, California, 92868
AdventHealth Orlando
Orlando, Florida, 32803
Arnold Palmer Hospital for Children
Orlando, Florida, 32806
Nemours Children's Hospital
Orlando, Florida, 32827
Hope Cancer Care of Nevada-Pahrump
Pahrump, Nevada, 89048
Lucile Packard Children's Hospital Stanford University
Palo Alto, California, 94304
Advocate Children's Hospital-Park Ridge
Park Ridge, Illinois, 60068
Camden Clark Medical Center
Parkersburg, West Virginia, 26101
Saint Joseph's Regional Medical Center
Paterson, New Jersey, 07503
Nemours Children's Clinic - Pensacola
Pensacola, Florida, 32504
Sacred Heart Hospital
Pensacola, Florida, 32504
OSF Children's Hospital of Illinois
Peoria, Illinois, 61637
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Saint Christopher's Hospital for Children
Philadelphia, Pennsylvania, 19134
Phoenix Childrens Hospital
Phoenix, Arizona, 85016
Mayo Clinic Hospital in Arizona
Phoenix, Arizona, 85054
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Huron Medical Center PC
Port Huron, Michigan, 48060
Lake Huron Medical Center
Port Huron, Michigan, 48060
Providence Portland Medical Center
Portland, Oregon, 97213
Providence Saint Vincent Medical Center
Portland, Oregon, 97225
Legacy Emanuel Children's Hospital
Portland, Oregon, 97227
Oregon Health and Science University
Portland, Oregon, 97239
Naval Medical Center - Portsmouth
Portsmouth, Virginia, 23708-2197
Rhode Island Hospital
Providence, Rhode Island, 02903
Corewell Health Reed City Hospital
Reed City, Michigan, 49677
Renown Regional Medical Center
Reno, Nevada, 89502
Saint Mary's Regional Medical Center
Reno, Nevada, 89503
Radiation Oncology Associates
Reno, Nevada, 89509
VCU Massey Comprehensive Cancer Center
Richmond, Virginia, 23298
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
University of Rochester
Rochester, New York, 14642
Kaiser Permanente-Roseville
Roseville, California, 95661
Corewell Health Children's
Royal Oak, Michigan, 48073
Sutter Medical Center Sacramento
Sacramento, California, 95816
University of California Davis Comprehensive Cancer Center
Sacramento, California, 95817
Corewell Health Lakeland Hospitals - Marie Yeager Cancer Center
Saint Joseph, Michigan, 49085
Corewell Health Lakeland Hospitals - Saint Joseph Hospital
Saint Joseph, Michigan, 49085
Novant Health Cancer Institute - Rowan
Salisbury, North Carolina, 28144
Rowan Regional Medical Center
Salisbury, North Carolina, 28144
Primary Children's Hospital
Salt Lake City, Utah, 84113
Children's Hospital of San Antonio
San Antonio, Texas, 78207
Methodist Children's Hospital of South Texas
San Antonio, Texas, 78229
University Hospital
San Antonio, Texas, 78229
University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
Kaiser Permanente-Rio San Diego
San Diego, California, 92108
Rady Children's Hospital - San Diego
San Diego, California, 92123
Naval Medical Center -San Diego
San Diego, California, 92134
UCSF Medical Center-Mission Bay
San Francisco, California, 94158
Kaiser Permanente Medical Center - Santa Clara
Santa Clara, California, 95051
Memorial Health University Medical Center
Savannah, Georgia, 31404
Maine Children's Cancer Program
Scarborough, Maine, 04074
Mayo Clinic in Arizona
Scottsdale, Arizona, 85259
Canada16 sites
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
University of Alberta Hospital
Edmonton, Alberta, T6G 2B7
IWK Health Centre
Halifax, Nova Scotia, B3K 6R8
McMaster Children's Hospital at Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5
Kingston Health Sciences Centre
Kingston, Ontario, K7L 2V7
Children's Hospital
London, Ontario, N6A 5W9
The Montreal Children's Hospital of the MUHC
Montreal, Quebec, H3H 1P3
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)
Québec, G1V 4G2
Jim Pattison Children's Hospital
Saskatoon, Saskatchewan, S7N 0W8
Saskatoon Cancer Centre
Saskatoon, Saskatchewan, S7N 4H4
Janeway Child Health Centre
St. John's, Newfoundland and Labrador, A1B 3V6
Hospital for Sick Children
Toronto, Ontario, M5G 1X8
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4
CancerCare Manitoba
Winnipeg, Manitoba, R3E 0V9
Australia6 sites
John Hunter Children's Hospital
Hunter Regional Mail Centre, New South Wales, 2310
Princess Margaret Hospital for Children
Perth, Western Australia, 6008
Perth Children's Hospital
Perth, Western Australia, 6009
Sydney Children's Hospital
Randwick, New South Wales, 2031
Queensland Children's Hospital
South Brisbane, Queensland, 4101
The Children's Hospital at Westmead
Westmead, New South Wales, 2145
Puerto Rico3 sites
HIMA San Pablo Oncologic Hospital
Caguas, 00726
San Jorge Children's Hospital
San Juan, 00912
University Pediatric Hospital
San Juan, 00926
New Zealand2 sites
Christchurch Hospital
Christchurch, 8011
Starship Children's Hospital
Grafton, Auckland, 1145
About this study
Official title: A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating With Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients With Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
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.
Read the detailed description
PRIMARY OBJECTIVE:
- 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:
- 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:
- 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.
- Conditions
- Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma, Embryonal Rhabdomyosarcoma, Rhabdomyosarcoma, Sclerosing Rhabdomyosarcoma, Spindle Cell Rhabdomyosarcoma
- Being tested
- Cyclophosphamide, Dactinomycin, Irinotecan Hydrochloride, Laboratory Biomarker Analysis, Questionnaire Administration
- Medical subject headings
- Rhabdomyosarcoma, Alveolar, Rhabdomyosarcoma, Embryonal, Rhabdomyosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518
Given IV and PO
DactinomycinBiological
Also called Actinomycin A IV, Actinomycin C1, Actinomycin D, Actinomycin I1
Given IV
Also called Campto, Camptosar, Camptothecin 11, Camptothecin-11
Given IV
Laboratory Biomarker AnalysisOther
Correlative studies
Questionnaire AdministrationOther
Ancillary studies
Radiation TherapyRadiation
Also called Cancer Radiotherapy, Energy Type, ENERGY_TYPE, Irradiate
Undergo RT
TemsirolimusDrug
Also called CCI 779, CCI-779, CCI-779 Rapamycin Analog, CCI779
Given IV
Also called Kyocristine, Leurocristine Sulfate, Leurocristine, sulfate, Oncovin
Given IV
VinorelbineDrug
Also called Dihydroxydeoxynorvinkaleukoblastine
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Regimen A (VAC/VI) | Experimental | 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. Receives: Drug: Cyclophosphamide; Biological: Dactinomycin; Drug: Irinotecan Hydrochloride; Other: Laboratory Biomarker Analysis; Other: Questionnaire Administration; Radiation: Radiation Therapy; Drug: Vincristine Sulfate |
| Regimen B (VAC/VI/temsirolimus) | Experimental | 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. Receives: Drug: Cyclophosphamide; Biological: Dactinomycin; Drug: Irinotecan Hydrochloride; Other: Laboratory Biomarker Analysis; Other: Questionnaire Administration; Radiation: Radiation Therapy; Drug: Temsirolimus; Drug: Vincristine Sulfate; Drug: Vinorelbine |
| Regimen C (FOXO1 fusion negative, VAC/VA) | Experimental | 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. Receives: Drug: Cyclophosphamide; Biological: Dactinomycin; Other: Laboratory Biomarker Analysis; Other: Questionnaire Administration; Radiation: Radiation Therapy; Drug: Vincristine Sulfate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 3 years from study entry
Secondary outcomes (1)
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.
Measured: Up to 3 years from study entry
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2015 (estimated)
- Study start1 Jun 2016
- Primary completion30 Jun 2023
- Study completion27 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Alveolar RhabdomyosarcomaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 325 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Abha A Gupta, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02567435
- Sponsor's protocol ID
- NCI-2015-01644
- Other IDs
- NCI-2015-01644, ARST1431, ARST1431, ARST1431, U10CA180886
- Status last verified
- Apr 2026
- First submitted
- 2 Oct 2015
- Last updated
- 1 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Winsnes K, Bradley JA, Gao Z, Gupta AA, Rothstein DH, Shenoy A, Venkatramani R, Xue W, Arndt C. Intermediate Risk Rhabdomyosarcoma in Very Young Children (Less Than or Equal to 24 Months) Treated on the Prior Children's Oncology Group Protocol ARST1431. Pediatr Blood Cancer. 2026 Jun;73(6):e70293. doi: 10.1002/1545-5017.70293. Epub 2026 Mar 31.
PubMed 41914453Gupta AA, Xue W, Harrison DJ, Hawkins DS, Dasgupta R, Wolden S, Shulkin B, Qumseya A, Routh JC, MacDonald T, Feinberg S, Crompton B, Rudzinski ER, Arnold M, Venkatramani R. Addition of temsirolimus to chemotherapy in children, adolescents, and young adults with intermediate-risk rhabdomyosarcoma (ARST1431): a randomised, open-label, phase 3 trial from the Children's Oncology Group. Lancet Oncol. 2024 Jul;25(7):912-921. doi: 10.1016/S1470-2045(24)00255-9.
PubMed 38936378de Traux de Wardin H, Dermawan JK, Vanoli F, Jiang SC, Singer S, Chi P, Tap W, Wexler LH, Antonescu CR. NF1-Driven Rhabdomyosarcoma Phenotypes: A Comparative Clinical and Molecular Study of NF1-Mutant Rhabdomyosarcoma and NF1-Associated Malignant Triton Tumor. JCO Precis Oncol. 2024 Apr;8:e2300597. doi: 10.1200/PO.23.00597.
PubMed 38603649