New drug targets tough childhood cancers with genetic flaws

NCT ID NCT03213678

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

First seen Apr 04, 2026 · Last updated Jun 22, 2026 · Updated 18 times

Summary

This phase 2 trial tested the drug samotolisib in 18 children and young adults with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that had spread, returned, or stopped responding to treatment. Only patients whose tumors had specific mutations in the TSC or PI3K/MTOR pathways were included. The goal was to see if samotolisib could shrink or control the cancer by blocking enzymes that help cancer cells grow.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Contacts and locations

Show contact details

Locations

AdventHealth Orlando

Orlando, Florida, 32803, 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
BI-LO Charities Children's Cancer Center

Greenville, South Carolina, 29605, United States
Banner Children's at Desert

Mesa, Arizona, 85202, United States
Banner University Medical Center - Tucson

Tucson, Arizona, 85719, 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
Bronson Methodist Hospital

Kalamazoo, Michigan, 49007, United States
C S Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States
Cardinal Glennon Children's Medical Center

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

Charlotte, North Carolina, 28203, United States
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 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 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
City of Hope Comprehensive Cancer Center

Duarte, California, 91010, United States
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, 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
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 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
Hackensack University Medical Center

Hackensack, New Jersey, 07601, 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
Kaiser Permanente-Oakland

Oakland, California, 94611, United States
Legacy Emanuel Children's Hospital

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
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
Mattel Children's Hospital UCLA

Los Angeles, California, 90095, United States
MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States
Medical City Dallas Hospital

Dallas, Texas, 75230, United States
Memorial Health University Medical Center

Savannah, Georgia, 31404, 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
Michigan State University Clinical Center

East Lansing, Michigan, 48824, 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
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
NYP/Weill Cornell Medical Center

New York, New York, 10065, 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
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
Novant Health Presbyterian Medical Center

Charlotte, North Carolina, 28204, United States
Ochsner Medical Center Jefferson

New Orleans, Louisiana, 70121, 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
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
Rainbow Babies and Childrens Hospital

Cleveland, Ohio, 44106, United States
Riley Hospital for Children

Indianapolis, Indiana, 46202, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center

Denver, Colorado, 80218, United States
Roswell Park Cancer Institute

Buffalo, New York, 14263, United States
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 Jude Midwest Affiliate

Peoria, Illinois, 61637, United States
Saint Luke's Cancer Institute - Boise

Boise, Idaho, 83712, United States
Saint Peter's University Hospital

New Brunswick, New Jersey, 08901, 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
Scott and White Memorial Hospital

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

Seattle, Washington, 98105, United States
Sinai Hospital of Baltimore

Baltimore, Maryland, 21215, 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
Summerlin Hospital Medical Center

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

Las Vegas, Nevada, 89109, United States
The Children's Hospital at TriStar Centennial

Nashville, Tennessee, 37203, United States
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
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 California Davis Comprehensive Cancer Center

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

Chicago, Illinois, 60637, United States
University of Florida Health Science Center - Gainesville

Gainesville, Florida, 32610, United States
University of Iowa/Holden Comprehensive Cancer Center

Iowa City, Iowa, 52242, 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 Vermont and State Agricultural College

Burlington, Vermont, 05405, United States
University of Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, 53792, United States
Valley Children's Hospital

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

Nashville, Tennessee, 37232, United States
Virginia Commonwealth University/Massey Cancer Center

Richmond, Virginia, 23298, 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

What this could mean

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

Active substance

samotolisib (LY3023414)

What this could lead to

If it works, this could point toward a new treatment option for children with certain hard-to-treat cancers that have specific genetic changes.

What could go wrong

This was a small, early-phase trial with only 18 participants, so results may not apply to all patients. The drug may cause serious side effects and may not shrink tumors effectively.

Conditions

The condition(s) this trial relates to.

cancer central nervous system cancer ependymoma Ewing sarcoma glioma hepatoblastoma Histiocytosis, Langerhans-Cell Langerhans cell histiocytosis medulloblastoma neuroblastoma Neuroectodermal Tumors, Primitive, Peripheral non-Hodgkin lymphoma osteosarcoma rhabdoid tumor rhabdomyosarcoma sarcoma Wilms tumor

As listed by the trial registrant

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