New hope for kids with brain tumors: drug combo trial launches

NCT ID NCT04576117

Disease control Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jul 02, 2026 · Updated 2 times

Summary

This Phase 3 trial tests whether adding vinblastine to selumetinib works better than selumetinib alone for children and young adults with recurrent or progressive low-grade glioma, a common brain cancer. About 300 participants aged 2 to 25 will be enrolled. The goal is to improve tumor control and delay disease progression.

What this could mean

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

Active substance: Selumetinib and vinblastine
What this could lead to: If successful, this could provide a more effective treatment option for children and young adults with recurrent low-grade glioma, potentially slowing or stopping tumor growth.
What could go wrong: This is an early-stage Phase 3 trial, and the combination may not prove more effective than selumetinib alone. Side effects from chemotherapy (vinblastine) could be significant.

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

About 300 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2021

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

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

Ages

2 to 25 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: * Feasibility phase: patients must be \>= 2 years and =\< 21 years of age at the time of enrollment * Efficacy phase: patients must be \>= 2 years and =\< 25 years of age at the time of enrollment * All patients \> 21 years of age at the time of enrollment must have had initial diagnosis of low-grade glioma by 21 years of age * Patients must have a body surface area (BSA) of \>= 0.5 m\^2 at enrollment * Patients must have eligibility confirmed by rapid central pathology and central molecular screening reviews performed on APEC14B1 * Non-neurofibromatosis type 1 (non-NF1), non-tuberous sclerosis complex (non-TSC) low-grade glioma (LGG) without a BRAFV600E or IDH1 mutation * Patients must have progressive or recurrent LGG. Note: Biopsy may be at either initial diagnosis or recurrence * Patients must have measurable disease, defined as having a two-dimensional measurable tumor volume of \>= 1 cm\^2 * Tumor size will be measured to include both solid and cystic components of the tumor (whether or not tumor is enhancing) + fluid attenuated inversion recovery (FLAIR) signal * Eligible histologies will include all tumors considered low-grade glioma or low-grade astrocytoma (World Health Organization \[WHO\] grade 1 and II) by the WHO Classification of Tumors of the Central Nervous System - 4th Edition Revised, with the exception of subependymal giant cell astrocytoma * Patients with metastatic disease or multiple independent primary LGGs are eligible * Patients must be progressive or recurrent after having been treated with at least one prior tumor-directed therapy before enrollment * Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study * Myelosuppressive chemotherapy: Must not have received within 2 weeks of entry onto this study (4 weeks if prior nitrosourea); * Biologic (anti-neoplastic agent): At least 7 days since the completion of therapy with a biologic agent; * Radiation therapy (RT): \>= 2 weeks (wks) for local palliative RT (small port); \>= 6 months must have elapsed if prior craniospinal RT or if \>= 50% radiation of pelvis; \>= 6 wks must have elapsed if other substantial bone marrow (BM) radiation; * Antibodies: \>= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to =\< grade 1; * MEK inhibitor or vinblastine: Must not have received treatment with a MEK inhibitor or vinblastine within 6 months of study enrollment * Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^ 2 or a serum creatinine based on age/sex as follows (within 7 days prior to enrollment): * 2 to \< 6 years: 0.8 mg/dL (male) 0.8 mg/dL (female) * 6 to \< 10 years: 1 mg/dL (male) 1 mg/dL (female) * 10 to \< 13 years: 1.2 mg/dL (male) 1.2 mg/dL (female) * 13 to \< 16 years: 1.5 mg/dL (male) 1.4 mg/dL (female) * \>= 16 years: 1.7 mg/dL (male) 1.4 mg/dL (female) * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed on study regardless of their total and indirect \[unconjugated\] bilirubin levels as long as their direct \[conjugated\] bilirubin is \< 3.1 mg/dL) * Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L (within 7 days prior to enrollment) * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L * Albumin \>= 2 g/L (within 7 days prior to enrollment) * Left ventricular ejection fraction (LVEF) \>= 53% (or institutional normal; if the LVEF result is given as a range of values, then the upper value of the range will be used) by echocardiogram (within 4 weeks prior to enrollment) * Corrected QT interval (QTc interval) =\< 450 msec by electrocardiogram (EKG) (within 4 weeks prior to enrollment) * Absolute neutrophil count \>= 1,000/uL (unsupported) (within 7 days prior to enrollment) * Platelets \>= 100,000/uL (unsupported) (within 7 days prior to enrollment) * Hemoglobin \>= 8 g/dL (may be supported) (within 7 days prior to enrollment) * Patients with a known seizure disorder should be stable and should not have experienced a significant increase in seizure frequency within 2 weeks prior to enrollment * Stable neurological examination for \>= 1 week * HYPERTENSION: * Patients 2-17 years of age must have a blood pressure that is =\< 95th percentile for age, height, and sex at the time of enrollment (with or without the use of anti-hypertensive medications); * Patients \>= 18 years of age must have a blood pressure =\< 130/80 mmHg at the time of enrollment (with or without the use of anti-hypertensive medications) * Note for patients of all ages: Adequate blood pressure can be achieved using medication for the treatment of hypertension * All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to enrollment * For all patients, an MRI of the brain (with orbital cuts for optic pathway tumors) and/or spine (depending on the site\[s\] of primary disease) with and without contrast must be performed within 4 weeks prior to enrollment * Note: If surgical resection or biopsy is performed at the time of progression or recurrence, a post-operative MRI is required * Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age * Patients must have the ability to swallow whole capsules Exclusion Criteria: * Prior therapy with vinblastine and/or a MEK inhibitor is permitted, with the following exceptions: * Patients must not have had progressive disease while on therapy with vinblastine or a MEK inhibitor; * Patients must not have discontinued vinblastine or selumetinib due to toxicity * Patients with a concurrent malignancy or history of treatment (other than surgery) for another tumor within the last year are ineligible * Patients with diffuse intrinsic pontine tumors as seen on MRI (\> 2/3 of pons involvement on imaging) are not eligible even if biopsy reveals grade I/II histology * Patients may not be receiving any other investigational agents * Patients must not have known hypersensitivity to selumetinib, vinblastine, or similar compounds * CYP3A4 agents: Patients must not have received fluconazole or drugs that are strong inducers or inhibitors of CYP3A4 within 7 days prior to study enrollment * Patients with any serious medical or psychiatric illness/condition, including substance use disorders or ophthalmological conditions, likely in the judgment of the investigator to interfere or limit compliance with study requirements/treatment * Patients who, in the opinion of the investigator, are not able to comply with the study procedures are not eligible * PRE-EXISTING CONDITIONS (CARDIAC): * Known genetic disorder that increases risk for coronary artery disease. Note: The presence of dyslipidemia in a family with a history of myocardial infarction is not in itself an exclusion unless there is a known genetic disorder documented; * Symptomatic heart failure * New York Heart Association (NYHA) class II-IV prior or current cardiomyopathy * Severe valvular heart disease * History of atrial fibrillation * PRE-EXISTING CONDITIONS (OPHTHALMOLOGIC CONDITIONS): * Current or past history of central serous retinopathy * Current or past history of retinal vein occlusion or retinal detachment * Patients with uncontrolled glaucoma * If checking pressure is clinically indicated, patients with intraocular pressure (IOP) \> 22 mmHg or upper limit of normal (ULN) adjusted by age are not eligible * Any multivitamin containing vitamin E must be stopped prior to study enrollment even if it contains less than 100% of the daily recommended dosing for vitamin E * Surgery within 2 weeks prior to enrollment, with the exception of a surgical biopsy, placement of a vascular access device or cerebrospinal fluid (CSF) diverting procedure such as endoscopic third ventriculostomy (ETV) and ventriculoperitoneal (VP) shunt * Note: Patients must have healed from any prior surgery * Patients who have an uncontrolled infection are not eligible * Female patients who are pregnant are not eligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential * Lactating females who plan to breastfeed their infants * 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 12 weeks after stopping study therapy are not eligible * Note: Women of child-bearing potential and males with sexual partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) should use effective methods of contraception for the duration of the study and for 12 weeks after stopping study therapy to avoid pregnancy and/or potential adverse effects on the developing embryo * 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.

grade II glioma low grade glioma low-grade astrocytoma

As listed by the trial registrant

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

Recurrent low grade astrocytoma Recurrent who grade 2 glioma Refractory low grade astrocytoma Refractory low grade glioma Refractory who grade 1 glioma

Contacts and locations

Locations

Albany Medical Center

Albany, New York, 12208, United States
Alfred I duPont Hospital for Children

Wilmington, Delaware, 19803, 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
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
Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada
Centre Hospitalier Universitaire de Sherbrooke-Fleurimont

Sherbrooke, Quebec, J1H 5N4, 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 Michigan

Detroit, Michigan, 48201, United States
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
Connecticut Children's Medical Center

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

Fort Worth, Texas, 76104, United States
Corewell Health Children's

Royal Oak, Michigan, 48073, United States
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States
Dana-Farber Cancer Institute

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

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

Austin, Texas, 78723, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
East Carolina University

Greenville, North Carolina, 27834, United States
East Tennessee Childrens Hospital

Knoxville, Tennessee, 37916, United States
Golisano Children's Hospital of Southwest Florida

Fort Myers, Florida, 33908, United States
Hospital for Sick Children

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

Halifax, Nova Scotia, B3K 6R8, Canada
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
Laura and Isaac Perlmutter Cancer Center at NYU Langone

New York, New York, 10016, United States
Loma Linda University Medical Center

Loma Linda, California, 92354, United States
Lucile Packard Children's Hospital Stanford University

Palo Alto, California, 94304, United States
Lurie Children's Hospital-Chicago

Chicago, Illinois, 60611, United States
Madigan Army Medical Center

Tacoma, Washington, 98431, United States
Maine Children's Cancer Program

Scarborough, Maine, 04074, United States
Mayo Clinic in Rochester

Rochester, Minnesota, 55905, 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
Morristown Medical Center

Morristown, New Jersey, 07960, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Nemours Children's Clinic-Jacksonville

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

Orlando, Florida, 32827, United States
New York Medical College

Valhalla, New York, 10595, United States
Norton Children's Hospital

Louisville, Kentucky, 40202, United States
Oregon Health and Science University

Portland, Oregon, 97239, United States
Primary Children's Hospital

Salt Lake City, Utah, 84113, United States
Prisma Health Richland Hospital

Columbia, South Carolina, 29203, United States
Providence Sacred Heart Medical Center and Children's Hospital

Spokane, Washington, 99204, 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 Christopher's Hospital for Children

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

Tampa, Florida, 33607, United States
Saint Luke's Cancer Institute - Boise

Boise, Idaho, 83712, United States
Sanford Broadway Medical Center

Fargo, North Dakota, 58122, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
State University of New York Upstate Medical University

Syracuse, New York, 13210, 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
UF Health Cancer Institute - Gainesville

Gainesville, Florida, 32610, United States
UT MD Anderson Cancer Center

Houston, Texas, 77030, United States
UT Southwestern/Simmons Cancer Center-Dallas

Dallas, Texas, 75390, United States
University of Alberta Hospital

Edmonton, Alberta, T6G 2B7, Canada
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 Minnesota/Masonic Cancer Center

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

Jackson, Mississippi, 39216, United States
University of Nebraska Medical Center

Omaha, Nebraska, 68198, 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 Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States
University of Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, 53792, United States
VCU Massey Comprehensive Cancer Center

Richmond, Virginia, 23298, 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
West Virginia University Healthcare

Morgantown, West Virginia, 26506, United States
Yale University

New Haven, Connecticut, 06520, United States

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