RecruitingPhase 2NCT05828069

A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

Phase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis

Sponsor

National Cancer Institute (NCI)

Enrollment

48 participants

Start Date

Mar 28, 2024

Study Type

INTERVENTIONAL

Conditions

Recurrent Langerhans Cell Histiocytosis Refractory Langerhans Cell Histiocytosis

Summary

This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back (relapsed) after previous treatment, or does not respond to therapy (refractory). Langerhans cell histiocytosis is a type of disease that occurs when the body makes too many immature Langerhans cells (a type of white blood cell). When these cells build up, they can form tumors in certain tissues and organs including bones, skin, lungs and pituitary gland and can damage them. This tumor is more common in children and young adults. DAY101 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Using DAY101 may be effective in treating patients with relapsed or refractory Langerhans cell histiocytosis.

Eligibility

Min Age: 180 DaysMax Age: 22 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a procedure called Biospecimen Collection, a procedure called Bone Marrow Aspiration, and others for people with recurrent langerhans cell histiocytosis and refractory langerhans cell histiocytosis. The study is currently recruiting participants at 119 locations. People eligible for this study include aged 180 Days to 22 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

PROCEDUREBiospecimen Collection

Undergo collection of blood and urine samples

PROCEDUREBone Marrow Aspiration

Undergo bone marrow aspiration

PROCEDUREBone Marrow Biopsy

Undergo bone marrow biopsy

PROCEDUREComputed Tomography

Undergo CT

PROCEDUREEchocardiography Test

Undergo ECHO

PROCEDUREFDG-Positron Emission Tomography and Computed Tomography Scan

Undergo FDG-PET imaging

PROCEDURELumbar Puncture

Undergo lumbar puncture

PROCEDUREMultigated Acquisition Scan

Undergo MUGA

DRUGTovorafenib

Given PO

Locations(120)

Children's Hospital of Alabama

Birmingham, Alabama, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Kaiser Permanente Downey Medical Center

Downey, California, United States

Loma Linda University Medical Center

Loma Linda, California, United States

Miller Children's and Women's Hospital Long Beach

Long Beach, California, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

Valley Children's Hospital

Madera, California, United States

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Kaiser Permanente-Oakland

Oakland, California, United States

Children's Hospital of Orange County

Orange, California, United States

Lucile Packard Children's Hospital Stanford University

Palo Alto, California, United States

UCSF Medical Center-Mission Bay

San Francisco, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center

Denver, Colorado, United States

Connecticut Children's Medical Center

Hartford, Connecticut, United States

Yale University

New Haven, Connecticut, United States

Alfred I duPont Hospital for Children

Wilmington, Delaware, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Golisano Children's Hospital of Southwest Florida

Fort Myers, Florida, United States

UF Health Cancer Institute - Gainesville

Gainesville, Florida, United States

Memorial Regional Hospital/Joe DiMaggio Children's Hospital

Hollywood, Florida, United States

Nemours Children's Clinic-Jacksonville

Jacksonville, Florida, United States

University of Miami Miller School of Medicine-Sylvester Cancer Center

Miami, Florida, United States

Nicklaus Children's Hospital

Miami, Florida, United States

Arnold Palmer Hospital for Children

Orlando, Florida, United States

Nemours Children's Hospital

Orlando, Florida, United States

Nemours Children's Clinic - Pensacola

Pensacola, Florida, United States

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Saint Joseph's Hospital/Children's Hospital-Tampa

Tampa, Florida, United States

Children's Healthcare of Atlanta - Arthur M Blank Hospital

Atlanta, Georgia, United States

Memorial Health University Medical Center

Savannah, Georgia, United States

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, United States

Lurie Children's Hospital-Chicago

Chicago, Illinois, United States

University of Chicago Comprehensive Cancer Center

Chicago, Illinois, United States

OSF Children's Hospital of Illinois

Peoria, Illinois, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

Blank Children's Hospital

Des Moines, Iowa, United States

University of Iowa/Holden Comprehensive Cancer Center

Iowa City, Iowa, United States

University of Kentucky/Markey Cancer Center

Lexington, Kentucky, United States

Children's Hospital New Orleans

New Orleans, Louisiana, United States

Ochsner Medical Center Jefferson

New Orleans, Louisiana, United States

Sinai Hospital of Baltimore

Baltimore, Maryland, United States

Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, United States

Michigan State University

East Lansing, Michigan, United States

Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Bronson Methodist Hospital

Kalamazoo, Michigan, United States

Children's Hospitals and Clinics of Minnesota - Minneapolis

Minneapolis, Minnesota, United States

University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, United States

University of Mississippi Medical Center

Jackson, Mississippi, United States

Cardinal Glennon Children's Medical Center

St Louis, Missouri, United States

Washington University School of Medicine

St Louis, Missouri, United States

Mercy Hospital Saint Louis

St Louis, Missouri, United States

Children's Hospital and Medical Center of Omaha

Omaha, Nebraska, United States

University of Nebraska Medical Center

Omaha, Nebraska, United States

Alliance for Childhood Diseases/Cure 4 the Kids Foundation

Las Vegas, Nevada, United States

Summerlin Hospital Medical Center

Las Vegas, Nevada, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital

New Brunswick, New Jersey, United States

Saint Joseph's Regional Medical Center

Paterson, New Jersey, United States

Albany Medical Center

Albany, New York, United States

Maimonides Medical Center

Brooklyn, New York, United States

The Steven and Alexandra Cohen Children's Medical Center of New York

New Hyde Park, New York, United States

Laura and Isaac Perlmutter Cancer Center at NYU Langone