New combination therapy aims to outsmart aggressive childhood brain cancer

NCT ID NCT00392327

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

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

Summary

This phase 3 trial investigates whether adding carboplatin and isotretinoin to standard radiation and chemotherapy improves outcomes for children and young adults with high-risk medulloblastoma, a fast-growing brain tumor. Participants receive radiation therapy along with several chemotherapy drugs, including carboplatin, which may make tumor cells more sensitive to radiation. The study compares different treatment regimens to see which is more effective at preventing the cancer from returning.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Carboplatin, cisplatin, cyclophosphamide, vincristine, isotretinoin
What this could lead to: If successful, this combination could improve long-term survival for children with high-risk medulloblastoma, a rare and aggressive brain tumor.
What could go wrong: This is an early-phase trial with a limited number of participants. The added drugs may increase side effects, and the results may not apply to all patients.

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

379 people

The number who actually took part.

Started

May 2007

Finished

Mar 2026

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

3 to 22 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: * Age greater than or equal to 3 and less than 22 years at the time of diagnosis * Newly diagnosed, previously untreated: (1) M0 medulloblastoma with \> 1.5 cm\^2 residual; (2) M+ medulloblastoma; patients with diffusely anaplastic medulloblastoma are eligible regardless of M-stage or residual tumor * As of amendment # 2, enrollment of patients with supratentorial PNET has been discontinued * All patients with M4 disease are not eligible * A pre-operative magnetic resonance imaging (MRI) scan of the brain with and without contrast is required; NOTE: computed tomography (CT) scans are NOT sufficient for study eligibility since radiation therapy planning and response will be based on MRI scans only * Post-operative head MRI scan with and without contrast (preferably within 72 hours post-surgery); for patients who undergo stereotactic biopsy only, either a pre or post-operative MRI is sufficient; for patients with M2 and M3 disease, a post-op MRI is strongly encouraged, but not mandatory * Spinal MRI imaging with and without gadolinium is required within 10 days of surgery if done pre-operatively or within 28 days of surgery if done post-operatively; for posterior fossa tumors, pre-operative MRI scans are preferred because surgically-induced inflammation/blood can be difficult to distinguish from tumor * Lumbar cerebrospinal fluid (CSF) cytology examination must be obtained pre-operatively or within 31 days following surgery; the optimal time for obtaining CSF is prior to surgery or 1-3 weeks following surgery; ventricular CSF (either pre- or post-op) may be used only if a post-operative spinal tap is contraindicated; if a spinal tap is contraindicated and there is no ventricular CSF available, then CSF cytology can be waived for patients with supratentorial tumors or if there is documentation of spinal subarachnoid metastases (M3); patients who are categorized as M1 must have either an intra-operative positive CSF (via lumbar puncture at the end of the procedure) or a positive lumbar CSF obtained \> 7 days post-operatively (to rule out surgically induced false positives) * Patients must have a Karnofsky performance level of \>= 30 for patients \> 16 years of age or a Lansky performance scale of \>= 30 for patients =\< 16 years of age and life expectancy \> 8 weeks * No previous chemotherapy or radiation therapy * Corticosteroids should not be used during chemotherapy administration as an antiemetic because of their effect on the blood-brain barrier * Clinically significant drug interactions have been reported when using vincristine with strong CYP450 3A4 inhibitors and inducers. Selected strong inhibitors of cytochrome P450, family 3, subfamily A, polypeptide 4 (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 (vincristine sulfate) * The clinical outcome and significance of CYP450 interactions with cyclophosphamide are less clear. CYP450 3A4 stimulators or inhibitors should be avoided or used with great caution; aprepitant also interacts with CYP3A4 and should be used with caution with etoposide or vincristine chemotherapy * Cisplatin should be used with caution with nephrotoxic drug; aminoglycoside should be avoided or used with caution during or shortly after cisplatin administration and concomitant use with amphotericin B should probably also be avoided; patients receiving cisplatin and other potentially ototoxic drugs such as aminoglycoside or loop diuretics concomitantly should be closely monitored for signs of ototoxicity * In patients receiving cisplatin and phenytoin or fosphenytoin, serum concentrations of phenytoin may decrease. Carbamazepine concentration may also decrease with concomitant use. Plasma levels of anticonvulsant agents should be monitored and doses adjusted during therapy with cisplatin * No other experimental therapy is permitted while on study * 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: * 0.8 mg/dL (2 to \< 6 years of age) * 1.0 mg/dL (6 to \< 10 years of age) * 1.2 mg/dL (10 to \< 13 years of age) * 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to \< 16 years of age) * 1.7 mg/dL (male) or 1.4 mg/dL (female) (\>= 16 years of age) * Total bilirubin \< 1.5 x upper limit of normal (ULN) for age * Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase \[AST\]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 2.5 x upper limit of normal (ULN) for age; for patients on anti-seizure medications, SGOT (AST) or SGPT (ALT) must be \< 5 x ULN * Absolute neutrophil count (ANC) \>= 1,000/uL * Platelets \>= 100,000/uL (untransfused) * Hemoglobin \>= 8 g/dl (may be transfused) * There is information indicating a risk of fetal or teratogenic toxicity with this treatment. Female patients who are post-menarchal must have a negative pregnancy test; lactating female patients must agree not to breast-feed while on this trial; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method * 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

Get updates

Get notified about this study

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

anaplastic/large cell medulloblastoma medulloblastoma

As listed by the trial registrant

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

Anaplastic medulloblastoma Medulloblastoma

Contacts and locations

Locations

AdventHealth Orlando

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

Oak Lawn, Illinois, 60453, United States
Advocate Children's Hospital-Park Ridge

Park Ridge, Illinois, 60068, United States
Advocate Lutheran General Hospital

Park Ridge, Illinois, 60068, United States
Albany Medical Center

Albany, New York, 12208, United States
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
Banner Children's at Desert

Mesa, Arizona, 85202, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, 77030, United States
Baystate Medical Center

Springfield, Massachusetts, 01199, United States
Blank Children's Hospital

Des Moines, Iowa, 50309, United States
British Columbia Children's Hospital

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

Kalamazoo, Michigan, 49007, 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
Carilion Children's

Roanoke, Virginia, 24014, 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 Medical Center of Akron

Akron, Ohio, 44308, United States
Children's Hospital New Orleans

New Orleans, Louisiana, 70118, United States
Children's Hospital and Medical Center of Omaha

Omaha, Nebraska, 68114, 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 San Antonio

San Antonio, Texas, 78207, United States
Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, 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
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center

Lebanon, New Hampshire, 03756, United States
Dayton Children's Hospital

Dayton, Ohio, 45404, United States
Dell Children's Medical Center of Central Texas

Austin, Texas, 78723, United States
Driscoll Children's Hospital

Corpus Christi, Texas, 78411, United States
Dutch Childhood Oncology Group

Utrecht, 3584 CS, Netherlands
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
Henry Ford Health Saint John Hospital

Detroit, Michigan, 48236, 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-Oakland

Oakland, California, 94611, United States
Kalamazoo Center for Medical Studies

Kalamazoo, Michigan, 49008, United States
Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96826, United States
Laura and Isaac Perlmutter Cancer Center at NYU Langone

New York, New York, 10016, United States
Legacy Emanuel Children's Hospital

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

Portland, Oregon, 97227, United States
Lehigh Valley Hospital - Muhlenberg

Bethlehem, Pennsylvania, 18017, United States
Lehigh Valley Hospital-Cedar Crest

Allentown, Pennsylvania, 18103, United States
Loma Linda University Medical Center

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

Maywood, Illinois, 60153, United States
Lucile Packard Children's Hospital Stanford University

Palo Alto, California, 94304, United States
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Torrance, California, 90502, United States
Lurie Children's Hospital-Chicago

Chicago, Illinois, 60611, United States
M D Anderson Cancer Center

Houston, Texas, 77030, United States
Madigan Army Medical Center

Tacoma, Washington, 98431, United States
Marshfield Medical Center-Marshfield

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

Tacoma, Washington, 98405, United States
Massachusetts General Hospital Cancer Center

Boston, Massachusetts, 02114, United States
Mayo Clinic in Rochester

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

Hamilton, Ontario, L8N 3Z5, Canada
MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States
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
Mount Sinai Hospital

New York, New York, 10029, 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
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
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
Overlook Hospital

Summit, New Jersey, 07902, United States
Penn State Children's Hospital

Hershey, Pennsylvania, 17033, 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 Alaska Medical Center

Anchorage, Alaska, 99508, 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
Riley Hospital for Children

Indianapolis, Indiana, 46202, United States
Roswell Park Cancer Institute

Buffalo, New York, 14263, United States
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital

New Brunswick, New Jersey, 08903, United States
Saint Barnabas Medical Center

Livingston, New Jersey, 07039, 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 Jude Midwest Affiliate

Peoria, Illinois, 61637, 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
Saint Peter's University Hospital

New Brunswick, New Jersey, 08901, United States
Saint Vincent Hospital Cancer Center Green Bay

Green Bay, Wisconsin, 54301, United States
Sanford Broadway Medical Center

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

Sioux Falls, South Dakota, 57117-5134, United States
Saskatoon Cancer Centre

Saskatoon, Saskatchewan, S7N 4H4, Canada
Scott and White Memorial Hospital

Temple, Texas, 76508, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Southern Illinois University School of Medicine

Springfield, Illinois, 62702, United States
State University of New York Upstate Medical University

Syracuse, New York, 13210, United States
Stony Brook University Medical Center

Stony Brook, New York, 11794, United States
Summerlin Hospital Medical Center

Las Vegas, Nevada, 89144, United States
Sunrise Hospital and Medical Center

Las Vegas, Nevada, 89109, United States
Sutter Medical Center Sacramento

Sacramento, California, 95816, United States
T C Thompson Children's Hospital

Chattanooga, Tennessee, 37403, United States
Tampa General Hospital

Tampa, Florida, 33606, United States
Texas Tech University Health Sciences Center-Amarillo

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

Nashville, Tennessee, 37203, United States
The Montreal Children's Hospital of the MUHC

Montreal, Quebec, H3H 1P3, Canada
The Steven and Alexandra Cohen Children's Medical Center of New York

New Hyde Park, New York, 11040, United States
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States
UCSF Medical Center-Mission Bay

San Francisco, California, 94158, United States
UCSF Medical Center-Parnassus

San Francisco, California, 94143, United States
UMC Cancer Center / UMC Health System

Lubbock, Texas, 79415, United States
UNC Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, 27599, United States
UT Southwestern/Simmons Cancer Center-Dallas

Dallas, Texas, 75390, United States
University Medical Center of Southern Nevada

Las Vegas, Nevada, 89102, United States
University Pediatric Hospital

San Juan, 00926, Puerto Rico
University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States
University of Alberta Hospital

Edmonton, Alberta, T6G 2B7, Canada
University of California Davis Comprehensive Cancer Center

Sacramento, California, 95817, United States
University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637, United States
University of Illinois

Chicago, Illinois, 60612, 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 Missouri Children's Hospital

Columbia, Missouri, 65212, 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
Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, 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
Wayne State University/Karmanos Cancer Institute

Detroit, Michigan, 48201, United States
West Virginia University Charleston Division

Charleston, West Virginia, 25304, United States
West Virginia University Healthcare

Morgantown, West Virginia, 26506, United States
Women's and Children's Hospital-Adelaide

North Adelaide, South Australia, 5006, Australia
Yale University

New Haven, Connecticut, 06520, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.