New hope for kids with rare immune cell cancer: drug trial launches

NCT ID NCT05828069

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

First seen Jun 24, 2026 · Last updated Aug 14, 2026 · Updated 13 times

Summary

This study tests a drug called tovorafenib (DAY101) in children and young adults (ages 6 months to 22 years) with Langerhans cell histiocytosis that has returned or not responded to treatment. The drug works by blocking enzymes that help cancer cells grow. Researchers will check safety, side effects, and how well the drug shrinks tumors. About 48 people will take part.

What this could mean

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

Active substance: tovorafenib (DAY101)
What this could lead to: If this works, it could offer a new treatment option for children and young adults with Langerhans cell histiocytosis that has come back or not responded to other therapies.
What could go wrong: This is a Phase 2 trial with only 48 participants, so results are still early. The drug may not work for everyone, and side effects like liver problems or skin reactions are possible.

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

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Conditions

The condition(s) this trial relates to.

Histiocytosis, Langerhans-Cell Langerhans cell histiocytosis

As listed by the trial registrant

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

RECURRENT LANGERHANS CELL HISTIOCYTOSIS REFRACTORY LANGERHANS CELL HISTIOCYTOSIS

Contacts and locations

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Locations

Albany Medical Center
RECRUITING

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

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

Las Vegas, Nevada, 89135, United States

Contact Email: •••••@•••••
Arkansas Children's Hospital
RECRUITING

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

Orlando, Florida, 32806, United States
BI-LO Charities Children's Cancer Center
RECRUITING

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

Houston, Texas, 77030, United States
Blank Children's Hospital
RECRUITING

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

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

Kalamazoo, Michigan, 49007, United States
CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)
RECRUITING

Québec, G1V 4G2, Canada
Cardinal Glennon Children's Medical Center
RECRUITING

St Louis, Missouri, 63104, United States
Centre Hospitalier Universitaire Sainte-Justine
RECRUITING

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

Sherbrooke, Quebec, J1H 5N4, Canada
Children's Healthcare of Atlanta - Arthur M Blank Hospital
RECRUITING

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

London, Ontario, N6A 5W9, Canada
Children's Hospital Colorado
RECRUITING

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

Los Angeles, California, 90027, United States
Children's Hospital New Orleans
RECRUITING

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

Omaha, Nebraska, 68114, United States
Children's Hospital of Alabama
RECRUITING

Birmingham, Alabama, 35233, United States

Contact Email: •••••@•••••
Children's Hospital of Orange County
RECRUITING

Orange, California, 92868, United States

Contact Email: •••••@•••••
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States

Contact Email: •••••@•••••
Children's Hospital of Pittsburgh of UPMC
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
Children's Hospital of San Antonio
RECRUITING

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

Norfolk, Virginia, 23507, United States

Contact Email: •••••@•••••
Children's Hospital of Wisconsin
RECRUITING

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

Minneapolis, Minnesota, 55404, United States
Children's National Medical Center
RECRUITING

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

Hartford, Connecticut, 06106, United States
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
RECRUITING

Grand Rapids, Michigan, 49503, United States
Dayton Children's Hospital
RECRUITING

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

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

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

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

Knoxville, Tennessee, 37916, United States
El Paso Children's Hospital
RECRUITING

El Paso, Texas, 79905, United States
Geisinger Medical Center
RECRUITING

Danville, Pennsylvania, 17822, United States

Contact Email: •••••@•••••
Golisano Children's Hospital of Southwest Florida
RECRUITING

Fort Myers, Florida, 33908, United States
Hackensack University Medical Center
RECRUITING

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

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

Halifax, Nova Scotia, B3K 6R8, Canada
Johns Hopkins All Children's Hospital
RECRUITING

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

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

Downey, California, 90242, United States
Kaiser Permanente-Oakland
RECRUITING

Oakland, California, 94611, United States
Kapiolani Medical Center for Women and Children
RECRUITING

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

New York, New York, 10016, United States

Contact Email: •••••@•••••
Loma Linda University Medical Center
RECRUITING

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

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

Chicago, Illinois, 60611, United States
Maimonides Medical Center
RECRUITING

Brooklyn, New York, 11219, United States
McMaster Children's Hospital at Hamilton Health Sciences
SUSPENDED

Hamilton, Ontario, L8N 3Z5, Canada
Memorial Health University Medical Center
RECRUITING

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

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

New York, New York, 10065, United States
Mercy Hospital Saint Louis
RECRUITING

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

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

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

Long Beach, California, 90806, United States
Mission Hospital
RECRUITING

Asheville, North Carolina, 28801, United States
Montefiore Medical Center - Moses Campus
SUSPENDED

The Bronx, New York, 10467, United States
Mount Sinai Hospital
RECRUITING

New York, New York, 10029, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
RECRUITING

New York, New York, 10032, United States

Contact Email: •••••@•••••
NYP/Weill Cornell Medical Center
RECRUITING

New York, New York, 10065, United States
Nationwide Children's Hospital
RECRUITING

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

Pensacola, Florida, 32504, United States

Contact Email: •••••@•••••
Nemours Children's Clinic-Jacksonville
RECRUITING

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

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

Valhalla, New York, 10595, United States
Nicklaus Children's Hospital
RECRUITING

Miami, Florida, 33155, United States
OSF Children's Hospital of Illinois
RECRUITING

Peoria, Illinois, 61637, United States

Contact Email: •••••@•••••
Ochsner Medical Center Jefferson
RECRUITING

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

Portland, Oregon, 97239, United States

Contact Email: •••••@•••••
Perth Children's Hospital
SUSPENDED

Perth, Western Australia, 6009, Australia
Primary Children's Hospital
RECRUITING

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

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

Spokane, Washington, 99204, United States
Queensland Children's Hospital
SUSPENDED

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

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

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

Denver, Colorado, 80218, United States

Contact Email: •••••@•••••
Royal Children's Hospital
SUSPENDED

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

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

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

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

Paterson, New Jersey, 07503, United States
Saint Vincent Hospital Cancer Center Green Bay
RECRUITING

Green Bay, Wisconsin, 54301, United States
Sanford USD Medical Center - Sioux Falls
RECRUITING

Sioux Falls, South Dakota, 57117-5134, United States

Contact Email: •••••@•••••
Scott and White Memorial Hospital
RECRUITING

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

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

Baltimore, Maryland, 21215, United States
State University of New York Upstate Medical University
RECRUITING

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

Las Vegas, Nevada, 89144, United States

Contact Email: •••••@•••••
Sydney Children's Hospital
SUSPENDED

Randwick, New South Wales, 2031, Australia
The Children's Hospital at TriStar Centennial
RECRUITING

Nashville, Tennessee, 37203, United States
The Steven and Alexandra Cohen Children's Medical Center of New York
RECRUITING

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

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

San Francisco, California, 94158, United States

Contact Email: •••••@•••••
UF Health Cancer Institute - Gainesville
RECRUITING

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

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

Dallas, Texas, 75390, United States

Contact Email: •••••@•••••
University of Alberta Hospital
RECRUITING

Edmonton, Alberta, T6G 2B7, Canada

Contact Email: •••••@•••••
University of Chicago Comprehensive Cancer Center
RECRUITING

Chicago, Illinois, 60637, United States

Contact Email: •••••@•••••
University of Iowa/Holden Comprehensive Cancer Center
RECRUITING

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

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

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

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

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

Omaha, Nebraska, 68198, United States
University of Oklahoma Health Sciences Center
RECRUITING

Oklahoma City, Oklahoma, 73104, United States
University of Texas Health Science Center at San Antonio
RECRUITING

San Antonio, Texas, 78229, United States

Contact Email: •••••@•••••
University of Virginia Cancer Center
RECRUITING

Charlottesville, Virginia, 22908, United States

Contact Email: •••••@•••••
VCU Massey Comprehensive Cancer Center
RECRUITING

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

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

Nashville, Tennessee, 37232, United States
Wake Forest University Health Sciences
RECRUITING

Winston-Salem, North Carolina, 27157, United States
Washington University School of Medicine
RECRUITING

St Louis, Missouri, 63110, United States

Contact Email: •••••@•••••
West Virginia University Charleston Division
RECRUITING

Charleston, West Virginia, 25304, United States
Yale University
RECRUITING

New Haven, Connecticut, 06520, United States

Contact Email: •••••@•••••

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