New hope for toddlers with brain cancer: stem cell rescue trial

NCT ID NCT00336024

Disease control Sponsor: Children's Oncology Group Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at 91 children under 3 years old with aggressive brain tumors. It compared two intense chemotherapy plans, one with and one without methotrexate, followed by a stem cell transplant. The goal was to see which approach led to more complete tumor disappearance.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

91 people

The number who actually took part.

Started

Oct 2007

Finished

Sep 2025

Lead sponsor

A research network

The lead sponsor is a research network or cooperative group.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 2 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * High-risk medulloblastoma defined by any of the following: * \> 1.5 cm\^2 residual disease for any medulloblastoma histology, or * Lumbar cerebral spinal fluid (CSF) cytology positive for tumor cells by analysis of fluid collected either before definitive surgery or at least 10 days after definitive surgery unless contraindicated, or * Magnetic resonance imaging (MRI) evidence of M2 or M3 metastatic disease, or * M4 disease * Supratentorial primitive neuroectodermal tumor (PNET) (any M-stage) will be eligible for study entry * Children less than 8 months of age at the time of definitive surgery with or without measurable radiographic residual tumor with M0 stage medulloblastoma will be eligible for study entry * Patients with anaplastic medulloblastoma are eligible regardless of M-stage or residual tumor * Patients with M0 classic, non-desmoplastic medulloblastoma (R1) with radiographically measurable residual disease \< 1.5 cm\^2 are eligible * Cranial MRI (with and without gadolinium) must be done pre-operatively; post-operatively, cranial MRI (with and without gadolinium) must be done, preferably within 48 hours of surgery; entire spinal MRI must be obtained either pre-operatively (with gadolinium) or post-operatively (at least 10 days following surgery) prior to study enrollment (with and without gadolinium); patients with MRI evidence of spinal disease are eligible for this study * Evaluation of lumbar CSF cytology (cytospin preparation for microscopic evaluation) must be performed either pre-operatively or at least 10 days after definitive surgery unless contraindicated * Patient must have a life expectancy \> 8 weeks * Patient must have received no prior radiation therapy or chemotherapy other than corticosteroids; corticosteroids are allowable for all patients * Creatinine clearance or radioisotope glomerular filtration rate \>= 60 mL/min * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age, and * Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase \[AST\]) and serum glutamic pyruvic transaminase (SGPT) (alanine transaminase \[ALT\]) \< 2 x ULN for age * Shortening fraction \>= 27% by echocardiogram, or * Ejection fraction \>= 47% by radionuclide angiogram * No evidence of dyspnea at rest * Pulse oximetry \> 94% on room air * Peripheral absolute neutrophil count (ANC) \> 1,000/uL * Platelet count \> 100,000/uL (transfusion independent) * Hemoglobin greater than 8 g/dL (may have received red blood cell \[RBC\] transfusions allowed) * Hold trimethoprim/sulfamethoxazole (Bactrim) on the day of high-dose methotrexate (HDMTX) infusion and continue to hold until the methotrexate level is less than 0.1 micromolar (1 x 10-7 M) * Avoid probenecid, penicillins, cephalosporins, aspirin, proton pump inhibitors and nonsteroidal anti-inflammatory drug (NSAIDS) on the day of methotrexate and continue until the methotrexate level is less than 0.1 micromolar (1 x 10-7 M) as renal excretion of methotrexate is inhibited by these agents * Avoid IV contrast media, urinary acidifiers, phenytoin, and fosphenytoin on the day of methotrexate and until the methotrexate level is less than 0.1 micromolar (1 x 10-7 M) * Concurrent use of enzyme inducing anticonvulsants (e.g. phenytoin, phenobarbital, and carbamazepine) should be avoided * Clinically significant drug interactions have been reported when using vincristine with strong cytochrome P450 (CYP450) family 3 subfamily A member 4 (3A4) inhibitors and inducers; selected strong inhibitors of cytochrome P450 3A4 include azole antifungals, such as fluconazole, voriconazole, itraconazole, ketoconazole, and strong inducers include drugs such as rifampin, phenytoin, phenobarbitol, carbamazepine, and St. John's wort; the use of these drugs should be avoided with vincristine * 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

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Conditions

The condition(s) this trial relates to.

anaplastic/large cell medulloblastoma medulloblastoma supratentorial primitive neuroectodermal tumor

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Anaplastic medulloblastoma Medulloblastoma Supratentorial embryonal tumor, not otherwise specified Untreated childhood supratentorial primitive neuroectodermal tumor

Contacts and locations

Locations

AdventHealth Orlando

Orlando, Florida, 32803, United States
Advocate Children's Hospital-Oak Lawn

Oak Lawn, Illinois, 60453, United States
Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada
Alfred I duPont Hospital for Children

Wilmington, Delaware, 19803, United States
Alliance for Childhood Diseases/Cure 4 the Kids Foundation

Las Vegas, Nevada, 89135, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202-3591, United States
Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States
Ascension Saint Vincent Indianapolis Hospital

Indianapolis, Indiana, 46260, United States
BI-LO Charities Children's Cancer Center

Greenville, South Carolina, 29605, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, 77030, United States
British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada
Bronson Methodist Hospital

Kalamazoo, Michigan, 49007, United States
Broward Health Medical Center

Fort Lauderdale, Florida, 33316, United States
C S Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States
CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)

Québec, G1V 4G2, Canada
CancerCare Manitoba

Winnipeg, Manitoba, R3E 0V9, Canada
Cardinal Glennon Children's Medical Center

St Louis, Missouri, 63104, United States
Carolinas Medical Center/Levine Cancer Institute

Charlotte, North Carolina, 28203, United States
Cedars Sinai Medical Center

Los Angeles, California, 90048, United States
Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada
Children's Healthcare of Atlanta - Arthur M Blank Hospital

Atlanta, Georgia, 30329, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital of Alabama

Birmingham, Alabama, 35233, United States
Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada
Children's Hospital of Orange County

Orange, California, 92868, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States
Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Children's Hospitals and Clinics of Minnesota - Minneapolis

Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States
Cook Children's Medical Center

Fort Worth, Texas, 76104, United States
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States
Covenant Children's Hospital

Lubbock, Texas, 79410, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Dayton Children's Hospital

Dayton, Ohio, 45404, United States
Driscoll Children's Hospital

Corpus Christi, Texas, 78411, United States
East Tennessee Childrens Hospital

Knoxville, Tennessee, 37916, United States
Eastern Maine Medical Center

Bangor, Maine, 04401, United States
Geisinger Medical Center

Danville, Pennsylvania, 17822, United States
Golisano Children's Hospital of Southwest Florida

Fort Myers, Florida, 33908, United States
Greenville Cancer Treatment Center

Greenville, South Carolina, 29605, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada
IWK Health Centre

Halifax, Nova Scotia, B3K 6R8, Canada
Inova Fairfax Hospital

Falls Church, Virginia, 22042, United States
Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States
Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, 21287, United States
Kaiser Permanente Downey Medical Center

Downey, California, 90242, United States
Kalamazoo Center for Medical Studies

Kalamazoo, Michigan, 49008, United States
Kingston Health Sciences Centre

Kingston, Ontario, K7L 2V7, Canada
Laura and Isaac Perlmutter Cancer Center at NYU Langone

New York, New York, 10016, United States
Lee Memorial Health System

Fort Myers, Florida, 33901, United States
Legacy Emanuel Children's Hospital

Portland, Oregon, 97227, United States
Legacy Emanuel Hospital and Health Center

Portland, Oregon, 97227, United States
Loma Linda University Medical Center

Loma Linda, California, 92354, United States
Loyola University Medical Center

Maywood, Illinois, 60153, United States
Lurie Children's Hospital-Chicago

Chicago, Illinois, 60611, United States
Marshfield Medical Center-Marshfield

Marshfield, Wisconsin, 54449, United States
Mary Bridge Children's Hospital and Health Center

Tacoma, Washington, 98405, United States
Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States
McMaster Children's Hospital at Hamilton Health Sciences

Hamilton, Ontario, L8N 3Z5, Canada
Medical City Dallas Hospital

Dallas, Texas, 75230, United States
Medical University of South Carolina

Charleston, South Carolina, 29425, United States
Memorial Health University Medical Center

Savannah, Georgia, 31404, United States
Memorial Regional Hospital/Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States
Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States
Mercy Children's Hospital

Toledo, Ohio, 43608, United States
Mercy Hospital Saint Louis

St Louis, Missouri, 63141, United States
Methodist Children's Hospital of South Texas

San Antonio, Texas, 78229, United States
Michigan State University

East Lansing, Michigan, 48823, United States
Miller Children's and Women's Hospital Long Beach

Long Beach, California, 90806, United States
Montefiore Medical Center - Moses Campus

The Bronx, New York, 10467, United States
Morristown Medical Center

Morristown, New Jersey, 07960, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
NYU Langone Hospital - Long Island

Mineola, New York, 11501, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Naval Medical Center - Portsmouth

Portsmouth, Virginia, 23708-2197, United States
Nemours Children's Clinic - Orlando

Orlando, Florida, 32806, United States
Nemours Children's Clinic - Pensacola

Pensacola, Florida, 32504, United States
Nemours Children's Clinic-Jacksonville

Jacksonville, Florida, 32207, United States
Nemours Children's Hospital

Orlando, Florida, 32827, United States
Nevada Cancer Research Foundation NCORP

Las Vegas, Nevada, 89120, United States
New York Medical College

Valhalla, New York, 10595, United States
Newark Beth Israel Medical Center

Newark, New Jersey, 07112, United States
Nicklaus Children's Hospital

Miami, Florida, 33155, United States
Norton Children's Hospital

Louisville, Kentucky, 40202, United States
Novant Health Presbyterian Medical Center

Charlotte, North Carolina, 28204, United States
Oregon Health and Science University

Portland, Oregon, 97239, United States
Orlando Health Cancer Institute

Orlando, Florida, 32806, United States
Overlook Hospital

Summit, New Jersey, 07902, United States
Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States
Primary Children's Hospital

Salt Lake City, Utah, 84113, United States
Princess Margaret Hospital for Children

Perth, Western Australia, 6008, Australia
Prisma Health Richland Hospital

Columbia, South Carolina, 29203, United States
ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital

Toledo, Ohio, 43606, United States
Providence Sacred Heart Medical Center and Children's Hospital

Spokane, Washington, 99204, United States
Rady Children's Hospital - San Diego

San Diego, California, 92123, United States
Rainbow Babies and Childrens Hospital

Cleveland, Ohio, 44106, United States
Roswell Park Cancer Institute

Buffalo, New York, 14263, United States
Royal Children's Hospital

Parkville, Victoria, 3052, Australia
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital

New Brunswick, New Jersey, 08903, United States
Saint Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States
Saint Joseph's Hospital/Children's Hospital-Tampa

Tampa, Florida, 33607, United States
Saint Joseph's Regional Medical Center

Paterson, New Jersey, 07503, United States
Saint Luke's Cancer Institute - Boise

Boise, Idaho, 83712, United States
Saint Mary's Medical Center

West Palm Beach, Florida, 33407, United States
San Jorge Children's Hospital

San Juan, 00912, Puerto Rico
Sanford Broadway Medical Center

Fargo, North Dakota, 58122, United States
Sanford USD Medical Center - Sioux Falls

Sioux Falls, South Dakota, 57117-5134, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Stony Brook University Medical Center

Stony Brook, New York, 11794, United States
Sutter Medical Center Sacramento

Sacramento, California, 95816, United States
Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia
Texas Tech University Health Sciences Center-Amarillo

Amarillo, Texas, 79106, United States
The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia
The Montreal Children's Hospital of the MUHC

Montreal, Quebec, H3H 1P3, Canada
Tufts Children's Hospital

Boston, Massachusetts, 02111, United States
Tulane University School of Medicine

New Orleans, Louisiana, 70112, United States
UCSF Medical Center-Parnassus

San Francisco, California, 94143, United States
UNC Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, 27599, United States
University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States
University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States
University of California Davis Comprehensive Cancer Center

Sacramento, California, 95817, United States
University of Florida Health Science Center - Gainesville

Gainesville, Florida, 32610, United States
University of Hawaii Cancer Center

Honolulu, Hawaii, 96813, United States
University of Iowa/Holden Comprehensive Cancer Center

Iowa City, Iowa, 52242, United States
University of Kentucky/Markey Cancer Center

Lexington, Kentucky, 40536, United States
University of Miami Miller School of Medicine-Sylvester Cancer Center

Miami, Florida, 33136, United States
University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States
University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States
University of New Mexico Cancer Center

Albuquerque, New Mexico, 87106, United States
University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States
University of Rochester

Rochester, New York, 14642, United States
University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States
University of Vermont and State Agricultural College

Burlington, Vermont, 05405, United States
VCU Massey Comprehensive Cancer Center

Richmond, Virginia, 23298, United States
Valley Children's Hospital

Madera, California, 93636, United States
Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States
Walter Reed National Military Medical Center

Bethesda, Maryland, 20889-5600, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States

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