New drug combo shows promise in tough brain cancer trial

NCT ID NCT03581292

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

First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 2 times

Summary

This study tested a new drug called veliparib, given with radiation and chemotherapy, for children and young adults with a fast-growing brain tumor (high-grade glioma). The goal was to see if adding veliparib helps keep the cancer from coming back longer than standard treatment. The trial enrolled 38 people aged 3 to 25 with specific tumor types.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

38 people

The number who actually took part.

Started

Nov 2018

Expected to finish

Oct 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

3 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: * Stratum 1 (IDH wild-type): Patients must be \>= 3 years of age and =\< 21 years of age at the time of enrollment * Please note Stratum 1 was closed to accrual on January 24, 2020 * Stratum 2 (IDH mutant): Patients must be \>= 3 years of age and =\< 25 years of age at the time of enrollment * Patients must have eligibility confirmed by rapid central pathology and central molecular screening reviews performed on APEC14B1: * Newly-diagnosed high-grade glioma such as anaplastic astrocytoma or glioblastoma * Negative results for H3 K27M by immunohistochemistry (IHC) * Negative results for BRAFV600 mutation by next-generation sequencing (NGS) * Patients must have histological verification of diagnosis. Patients with M+ disease (defined as evidence of neuraxis dissemination) are not eligible. Cerebrospinal fluid (CSF) cytology is not required but may be obtained if clinically indicated prior to study enrollment. If cytology is positive, the patient would be considered to have metastatic disease and would, therefore, be ineligible * Pre-operative and post-operative brain magnetic resonance imaging (MRI) with and without contrast must be obtained. The requirement for a post-operative MRI is waived for patients who undergo biopsy only. A spine MRI is not required, but may be obtained if clinically indicated. If the spine MRI is positive, the patient would be considered to have M+ disease (defined as neuraxis dissemination) and would be ineligible * 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 * Peripheral absolute neutrophil count (ANC) \>= 1,000/uL (within 7 days prior to enrollment) * Platelet count \>= 100,000/uL (transfusion independent) (within 7 days prior to enrollment) * Hemoglobin \>= 8.0 gm/dL (can be transfused) (within 7 days prior to enrollment) * 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 (within 7 days prior to enrollment): * 3 to \< 6 years: 0.8 (male and female) maximum serum creatinine (mg/dL) * 6 to \< 10 years: 1 (male and female) maximum serum creatinine (mg/dL) * 10 to \< 13 years: 1.2 (male and female) maximum serum creatinine (mg/dL) * 13 to \< 16 years: 1.5 (male), 1.4 (female) maximum serum creatinine (mg/dL) * \>= 16 years: 1.7 (male), 1.4 (female) maximum serum creatinine (mg/dL) * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) * Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L * Patients with seizure disorder may be enrolled if seizures are well-controlled (i.e., patients must not have required rescue medications for uncontrolled seizures within 14 days prior to enrollment) * Patients must be enrolled and protocol therapy must be projected to begin no later than 31 days after definitive surgery (Day 0). If a biopsy only was performed, the biopsy date will be considered the date of definitive surgery. For patients who have a biopsy or incomplete resection at diagnosis followed by additional surgery, the date of the last resection will be considered the date of definitive surgery * 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 Exclusion Criteria: * Patients with the following histologies: * Diffuse astrocytoma (grade 2) * Oligodendrogliomas (any grade) * Pleomorphic xanthoastrocytoma (PXA, any grade) * Patients with primary tumor location of brainstem or spinal cord * Patients with M+ disease (defined as neuraxis dissemination either by imaging or by cytology) * Patients with treatment-related acute myeloid leukemia (AML) (t-AML)/myelodysplastic syndrome (MDS) or with features suggestive of AML/MDS * Prior allogenic bone marrow transplant or double umbilical cord blood transplantation * Patients must not have received any prior tumor-directed therapy including radiation therapy, chemotherapy (tumor-directed therapy), molecularly targeted agents, or immunotherapy for the treatment of HGG other than surgical intervention and/or corticosteroids * Lumbar CSF cytology is not required, but may be performed if clinically indicated prior to study enrollment. If lumbar CSF cytology is positive, the patient is considered to have M+ disease and is ineligible * Note: False positive cytology can occur within 10 days of surgery * Patients with gliomatosis cerebri type 1 or 2 * Patients who are not able to receive protocol specified radiation therapy * Patients must not be currently receiving other anti-cancer agents * Patients with known constitutional mismatch repair deficiency syndrome (CMMR-D)/biallelic mismatch repair deficiency (bMMRD) * Female patients who are pregnant are ineligible due to risks of fetal and teratogenic adverse events as seen in animal/human studies * Lactating females are not eligible unless they have agreed not to breastfeed their infants * Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained * Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation and for 6 months after the last dose of protocol-specified chemotherapy

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Conditions

The condition(s) this trial relates to.

anaplastic astrocytoma astrocytoma (excluding glioblastoma) glioblastoma glioma malignant glioma

As listed by the trial registrant

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

Anaplastic astrocytoma Glioblastoma Malignant glioma

Contacts and locations

Locations

AdventHealth Orlando

Orlando, Florida, 32803, United States
Albany Medical Center

Albany, New York, 12208, 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
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
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
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 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 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
Christchurch Hospital

Christchurch, 8011, New Zealand
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 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
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
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
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
HIMA San Pablo Oncologic Hospital

Caguas, 00726, Puerto Rico
Henry Ford Health Saint John Hospital

Detroit, Michigan, 48236, United States
IWK Health Centre

Halifax, Nova Scotia, B3K 6R8, Canada
Janeway Child Health Centre

St. John's, Newfoundland and Labrador, A1B 3V6, Canada
John Hunter Children's Hospital

Hunter Regional Mail Centre, New South Wales, 2310, Australia
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
Kaiser Permanente-Oakland

Oakland, California, 94611, 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
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
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
Mattel Children's Hospital UCLA

Los Angeles, California, 90095, 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 Hospital Saint Louis

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

San Antonio, Texas, 78229, United States
Miller Children's and Women's Hospital Long Beach

Long Beach, California, 90806, United States
Mission Hospital

Asheville, North Carolina, 28801, United States
Monash Medical Center-Clayton Campus

Clayton, Victoria, 3168, Australia
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
Nationwide Children's Hospital

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

Portsmouth, Virginia, 23708-2197, 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
Nicklaus Children's Hospital

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

Louisville, Kentucky, 40202, United States
OSF Children's Hospital of Illinois

Peoria, Illinois, 61637, United States
Ochsner Medical Center Jefferson

New Orleans, Louisiana, 70121, United States
Oregon Health and Science University

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

Perth, Western Australia, 6009, Australia
Phoenix Childrens Hospital

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

Salt Lake City, Utah, 84113, United States
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
Queensland Children's Hospital

South Brisbane, Queensland, 4101, Australia
Rainbow Babies and Childrens Hospital

Cleveland, Ohio, 44106, United States
Renown Regional Medical Center

Reno, Nevada, 89502, United States
Rhode Island Hospital

Providence, Rhode Island, 02903, United States
Riley Hospital for Children

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

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

Parkville, Victoria, 3052, Australia
Saint Joseph's Hospital/Children's Hospital-Tampa

Tampa, Florida, 33607, United States
Saint Jude Children's Research Hospital

Memphis, Tennessee, 38105, 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
Sanford Broadway Medical Center

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

Sioux Falls, South Dakota, 57117-5134, United States
Scott and White Memorial Hospital

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

Seattle, Washington, 98105, United States
Starship Children's Hospital

Grafton, Auckland, 1145, New Zealand
State University of New York Upstate Medical University

Syracuse, New York, 13210, United States
Summerlin Hospital Medical Center

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

Las Vegas, Nevada, 89109, United States
Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia
T C Thompson Children's Hospital

Chattanooga, Tennessee, 37403, United States
Tampa General Hospital

Tampa, Florida, 33606, United States
The Children's Hospital at TriStar Centennial

Nashville, Tennessee, 37203, 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
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States
UMass Memorial Medical Center - University Campus

Worcester, Massachusetts, 01655, United States
UNC Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, 27599, United States
USA Health Strada Patient Care Center

Mobile, Alabama, 36604, United States
UT MD Anderson Cancer Center

Houston, Texas, 77030, 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 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 Nebraska Medical Center

Omaha, Nebraska, 68198, 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 Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, 53792, 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 Healthcare

Morgantown, West Virginia, 26506, United States
Yale University

New Haven, Connecticut, 06520, United States

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