Clinical trialNCT05828069RecruitingPhase 2Interventional

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

SponsorNational Cancer Institute (NCI)Enrollment48Recurrent Langerhans Cell HistiocytosisRefractory Langerhans Cell Histiocytosis

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Carl E Allen
Children's Oncology Group
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

120 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Australia

Sydney Children'S Hospital
Randwick, New South Wales, Australia
Suspended
Queensland Children'S Hospital
South Brisbane, Queensland, Australia
Suspended
Royal Children'S Hospital
Parkville, Victoria, Australia
Suspended
Perth Children'S Hospital
Perth, Western Australia, Australia
Suspended

Canada

University of Alberta Hospital
Edmonton, Alberta, Canada
Sarah J. McKillop
Recruiting
British Columbia Children'S Hospital
Vancouver, British Columbia, Canada
Rebecca J. Deyell
Recruiting
Iwk Health Centre
Halifax, Nova Scotia, Canada
Suspended
Mcmaster Children'S Hospital at Hamilton Health Sciences
Hamilton, Ontario, Canada
Suspended
Children'S Hospital
London, Ontario, Canada
Suspended
Hospital for Sick Children
Toronto, Ontario, Canada
Suspended
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Monia Marzouki
Recruiting
Centre Hospitalier Universitaire de Sherbrooke-Fleurimont
Sherbrooke, Quebec, Canada
Josee Brossard
Recruiting
Chu de Quebec-Centre Hospitalier de L'Universite Laval (chul)
Québec, Canada
Bruno Michon
Recruiting

United States

Children'S Hospital of Alabama
Birmingham, Alabama, United States
Matthew A. Kutny
Recruiting
Arkansas Children'S Hospital
Little Rock, Arkansas, United States
Michael W. Bishop
Recruiting
Kaiser Permanente Downey Medical Center
Downey, California, United States
Robert M. Cooper
Recruiting
Loma Linda University Medical Center
Loma Linda, California, United States
Albert Kheradpour
Recruiting
Miller Children'S and Women'S Hospital Long Beach
Long Beach, California, United States
Jacqueline N. Casillas
Recruiting
Children'S Hospital Los Angeles
Los Angeles, California, United States
Rebecca S. Parker
Recruiting
Valley Children'S Hospital
Madera, California, United States
Active, not recruiting
Kaiser Permanente-Oakland
Oakland, California, United States
Aarati V. Rao
Recruiting
Ucsf Benioff Children'S Hospital Oakland
Oakland, California, United States
Jennifer G. Michlitsch
Recruiting
Children'S Hospital of Orange County
Orange, California, United States
Elyssa M. Rubin
Recruiting
Lucile Packard Children'S Hospital Stanford University
Palo Alto, California, United States
Jay Michael S. Balagtas
Recruiting
Ucsf Medical Center-Mission Bay
San Francisco, California, United States
Natalie L. Wu
Recruiting
Children'S Hospital Colorado
Aurora, Colorado, United States
Timothy P. Garrington
Recruiting
Rocky Mountain Hospital for Children-Presbyterian Saint Luke'S Medical Center
Denver, Colorado, United States
Florence Choo
Recruiting
Connecticut Children'S Medical Center
Hartford, Connecticut, United States
Michael S. Isakoff
Recruiting
Yale University
New Haven, Connecticut, United States
Farzana Pashankar
Recruiting
Alfred I Dupont Hospital for Children
Wilmington, Delaware, United States
Vibhuti Agarwal
Recruiting
Children'S National Medical Center
Washington D.C., District of Columbia, United States
Jeffrey S. Dome
Recruiting
Golisano Children'S Hospital of Southwest Florida
Fort Myers, Florida, United States
Emad K. Salman
Recruiting
Uf Health Cancer Institute - Gainesville
Gainesville, Florida, United States
Brian Stover
Recruiting
Memorial Regional Hospital/joe Dimaggio Children'S Hospital
Hollywood, Florida, United States
Iftikhar Hanif
Recruiting
Nemours Children'S Clinic-Jacksonville
Jacksonville, Florida, United States
Vibhuti Agarwal
Recruiting
Nicklaus Children'S Hospital
Miami, Florida, United States
Maggie E. Fader
Recruiting
University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, Florida, United States
Warren Alperstein
Recruiting
Arnold Palmer Hospital for Children
Orlando, Florida, United States
Zhongbo Hu
Recruiting
Nemours Children'S Hospital
Orlando, Florida, United States
Vibhuti Agarwal
Recruiting
Nemours Children'S Clinic - Pensacola
Pensacola, Florida, United States
Jeffrey H. Schwartz
Recruiting

80 more sites on the registry record.ClinicalTrials.gov ↗

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Tovorafenib →

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 28 Mar 2024
Primary completion: 30 Sept 2028
Completion: 30 Sept 2028
Last update posted: 19 Aug 2026
Registered enrollment: 48
Registry id: NCT05828069

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.