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

RecruitingPhase 2

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

Who can join

180 Days – 22 Years · All sexes

Full eligibility criteria
Inclusion Criteria:

* 180 days- \< 22 years (at time of study enrollment)
* Patient must have a body surface area of ≥ 0.3 m\^2
* Patients with progressive, relapsed, or recurrent LCH with measurable disease at study entry

  * Patients must have had histologic verification of LCH (from either original diagnosis or relapse/progression) at the time of study entry

    * Tissue confirmation of relapse is recommended but not required.
    * Pathology report must be submitted for central confirmation of diagnosis within 7 days of enrollment.
    * Formalin-fixed paraffin-embedded (FFPE) blocks or unstained slides (initial diagnosis and/or subsequent biopsies) will be required for retrospective central confirmation of diagnosis and molecular studies
    * Patients with mixed histiocytic disorders (e.g. LCH with juvenile xanthogranuloma) may be included
  * Patients must have measurable disease
  * Patients must have progressive or refractory disease or experience relapse after at least one previous systemic treatment strategy
  * Pathogenic somatic mutation detected in genes encoding tyrosine kinase receptors (CSFR1, ERBB3 or ALK), RAS or RAF (may be from original or subsequent biopsy or peripheral blood/bone marrow aspirate). Clinical mutation reports may include quantitative polymerase chain reaction (PCR) (e.g. BRAFV600E) and/or Sanger or next generation sequencing. Immunohistochemistry (e.g. VE1 antibody for BRAFV600E) alone is not sufficient
* Participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet, which may be taken by mouth or other enteral route such as nasogastric, jejunostomy, or gastric tube
* Karnofsky \>= 50% for patients \> 16 years of age and Lansky \>= 50% for patients =\< 16 years of age
* 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
* Myelosuppressive chemotherapy: Patients must not have received within 14 days of entry onto this study
* Investigational agent or any other anticancer therapy not defined above: Patients must not have received any investigational agent or any other anticancer therapy (including MAPK pathway inhibitor) for at least 14 days prior to planned start of tovorafenib (DAY101)
* Radiation therapy (RT): Patient must not have received RT within 2 weeks after the last dose fraction of RT
* Patients must have fully recovered from any prior surgery
* Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, targeted inhibitor, and/or radiotherapy with toxicities reduced to grade 1 or less (Common Terminology Criteria for Adverse Events \[CTCAE\] version 5.0)
* Steroids: =\< 0.5 mg/kg/day of prednisone equivalent (maximum 20 mg/day) averaged during the month prior to study enrollment is permissible
* Strong inducers or inhibitors of CYP2C8 are prohibited for 14 days before the first dose of tovorafenib (DAY101) and from planned administration for the duration of study participation
* Medications that are breast cancer resistant protein (BCRP) substrates that have a narrow therapeutic index are prohibited for 14 days before the first dose of tovorafenib (DAY101) and for the duration of study participation
* Peripheral absolute neutrophil count (ANC) \>= 750/uL unless secondary to bone marrow involvement, in such cases bone marrow involvement must be documented (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* Platelet count \>= 75,000/uL (unsupported/without transfusion within the past 7 days) (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* Patients with marrow disease must have platelet count of \>= 75,000/uL (transfusion support allowed) and must not be refractory to platelet transfusions. Bone marrow involvement must be documented
* Hemoglobin \>= 8 g/dL (unsupported/without transfusion within the past 7 days). Patients with marrow disease must have hemoglobin \>= 8 g/dL (transfusion support allowed). Bone marrow involvement must be documented
* Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g., Neulasta \[registered trademark\]) or 7 days for short-acting growth factor
* A serum creatinine based on age/sex as follows (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)

  * Age: 6 months to \< 1 year; Maximum Serum Creatinine (mg/dL):= 0.5 mg/dl (male and female)
  * Age: 1 to \< 2 years; Maximum Serum Creatinine (mg/dL): = 0.6 mg/dl (male and female)
  * Age: 2 to \< 6 years; Maximum Serum Creatinine (mg/dL): = 0.8 mg/dl (male and female)
  * Age: 6 to \< 10 years; Maximum Serum Creatinine (mg/dL): = 1.0 mg/dl (male and female)
  * Age: 10 to \< 13 years; Maximum Serum Creatinine (mg/dL): = 1.2 mg/dl (male and female)
  * Age: 13 to \< 16 years; Maximum Serum Creatinine (mg/dL): = 1.5 mg/dl (male) and 1.4 mg/dl (female)
  * Age: \>= 16 years; Maximum Serum Creatinine (mg/dL): = 1.7 mg/dl (male) and 1.4 mg/dl (female)
  * OR- a 24 hour urine creatinine clearance \>= 50 mL/min/1.73 m\^2
  * OR- a glomerular filtration rate (GFR) \>= 50 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)
  * Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility
* Bilirubin (sum of conjugated + unconjugated) =\< 1.5 x upper limit of normal (ULN) for age (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* Alanine aminotransferase (ALT) =\< 3 x ULN for age (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* Serum albumin \>= 2 g/dl must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
* For patients with liver disease caused by their histiocytic disorder (as evaluated on radiographic imaging or biopsy): patients may be enrolled with abnormal bilirubin, aspartate aminotransferase (AST), ALT and albumin with documentation of histiocytic liver disease
* Fractional shortening (FS) of \>= 25% or ejection fraction of \>= 50%, as determined by echocardiography or multigated acquisition scan (MUGA) within 28 days prior to study enrollment. Depending on institutional standard, either FS or left ventricular ejection fraction (LVEF) is adequate for enrollment if only one value is measured; if both values are measured, then both values must meet criteria above (must be obtained within 28 days prior to enrollment and start of protocol therapy) (repeat if necessary)
* No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry \> 94% if there is clinical indication for determination; unless it is due to underlying pulmonary LCH
* Central Nervous System Function Defined As:

  * Patients with seizure disorder may be enrolled if well controlled
  * Central nervous system (CNS) toxicity =\< Grade 2
* Human immunodeficiency virus (HIV) infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial unless antiretroviral therapy interacts with the metabolism of tovorafenib (DAY101) and cannot safely be changed to antivirals that do not interact with study medication
* All patients and/or their parent(s) 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:

* LCH arising along with other hematologic malignancy (e.g. mixed LCH with acute lymphoblastic leukemia) or any history of non-histiocytic malignancy
* Disease scenarios as below will be excluded

  * Skin-limited disease
  * Gastrointestinal (GI) tract involvement only (those that have disease that can be determined by endoscopic biopsies only)
  * LCH-associated neurodegeneration (LCH-ND) without parenchymal lesions or other systemic lesions
* Patients with activating mutations in MAP2K1 are not eligible for this study due to drug target specificity. Mutation status will be submitted to study team within 7 days of enrollment
* Refractory nausea and vomiting, malabsorption, or external biliary shunt that would preclude adequate absorption of tovorafenib (DAY101)
* Uncontrolled systemic bacterial, viral, or fungal infection
* Major surgical procedure or significant traumatic injury within 14 days prior to study enrollment, or anticipation of need for major surgical procedure during the course of the study. Placement of a vascular access device or minor surgery is permitted within fourteen (14) days of study enrollment (provided that the wound has healed)
* History of significant bowel resection that would preclude adequate absorption or other significant malabsorptive disease
* Ophthalmologic considerations: Patients with known significant ophthalmologic conditions or known risk factors for retinal vein occlusion (RVO) or central serous retinopathy (CSR) are not eligible
* History of solid organ or hematopoietic bone marrow transplantation
* Clinically significant active cardiovascular disease, or history of myocardial infarction, or deep vein thrombosis/pulmonary embolism within 6 months prior to enrollment, ongoing cardiomyopathy, or current prolonged QT interval \> 440 ms based on triplicate electrocardiogram (ECG) average
* History of Grade \>= 2 CNS hemorrhage or history of any CNS hemorrhage within 28 days of study entry
* History of any drug reaction with eosinophilia and systemic symptoms (DRESS) syndrome or Stevens Johnsons syndrome (SJS) or who are allergic to tovorafenib (DAY101) or any of its components
* CTCAE version (V.) 5.0 Grade 3 symptomatic creatinine kinase (CPK) elevation (\> 5 x ULN)
* Female patients who are pregnant are ineligible. A pregnancy test is required for female patients of childbearing potential
* Lactating females who plan to breastfeed their infants are ineligible
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation are ineligible. Women of childbearing potential must use non-hormonal contraception during tovorafenib treatment and for at least 28 days after the last dose. Men should use effective contraception and must not father a child while taking tovorafenib and for 14 days after the last dose

About the study

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.

What is being tested

Sponsor: National Cancer Institute (NCI) · Participants: 48 · Started: Mar 28, 2024

Contact the study team

Official record on ClinicalTrials.gov — NCT05828069

Locations in the U.S.

AlabamaChildren's Hospital of Alabama, Birmingham
ArkansasArkansas Children's Hospital, Little Rock
CaliforniaKaiser Permanente Downey Medical Center, Downey
Loma Linda University Medical Center, Loma Linda
Miller Children's and Women's Hospital Long Beach, Long Beach
Children's Hospital Los Angeles, Los Angeles
Kaiser Permanente-Oakland, Oakland
UCSF Benioff Children's Hospital Oakland, Oakland
Children's Hospital of Orange County, Orange
Lucile Packard Children's Hospital Stanford University, Palo Alto
UCSF Medical Center-Mission Bay, San Francisco
ColoradoChildren's Hospital Colorado, Aurora
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center, Denver
ConnecticutConnecticut Children's Medical Center, Hartford
Yale University, New Haven
DelawareAlfred I duPont Hospital for Children, Wilmington
District of ColumbiaChildren's National Medical Center, Washington D.C.
FloridaGolisano Children's Hospital of Southwest Florida, Fort Myers
UF Health Cancer Institute - Gainesville, Gainesville
Memorial Regional Hospital/Joe DiMaggio Children's Hospital, Hollywood
Nemours Children's Clinic-Jacksonville, Jacksonville
Nicklaus Children's Hospital, Miami
University of Miami Miller School of Medicine-Sylvester Cancer Center, Miami
Arnold Palmer Hospital for Children, Orlando
Nemours Children's Hospital, Orlando
Nemours Children's Clinic - Pensacola, Pensacola
Johns Hopkins All Children's Hospital, St. Petersburg
Saint Joseph's Hospital/Children's Hospital-Tampa, Tampa
GeorgiaChildren's Healthcare of Atlanta - Arthur M Blank Hospital, Atlanta
Memorial Health University Medical Center, Savannah
HawaiiKapiolani Medical Center for Women and Children, Honolulu
IllinoisLurie Children's Hospital-Chicago, Chicago
University of Chicago Comprehensive Cancer Center, Chicago
OSF Children's Hospital of Illinois, Peoria
IndianaRiley Hospital for Children, Indianapolis
IowaBlank Children's Hospital, Des Moines
University of Iowa/Holden Comprehensive Cancer Center, Iowa City
KentuckyUniversity of Kentucky/Markey Cancer Center, Lexington
LouisianaChildren's Hospital New Orleans, New Orleans
Ochsner Medical Center Jefferson, New Orleans
MarylandJohns Hopkins University/Sidney Kimmel Cancer Center, Baltimore
Sinai Hospital of Baltimore, Baltimore
MichiganCorewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital, Grand Rapids
Bronson Methodist Hospital, Kalamazoo
MinnesotaChildren's Hospitals and Clinics of Minnesota - Minneapolis, Minneapolis
MississippiUniversity of Mississippi Medical Center, Jackson
MissouriCardinal Glennon Children's Medical Center, St Louis
Mercy Hospital Saint Louis, St Louis
Washington University School of Medicine, St Louis
NebraskaChildren's Hospital and Medical Center of Omaha, Omaha
University of Nebraska Medical Center, Omaha
NevadaAlliance for Childhood Diseases/Cure 4 the Kids Foundation, Las Vegas
Summerlin Hospital Medical Center, Las Vegas
New JerseyHackensack University Medical Center, Hackensack
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital, New Brunswick
Saint Joseph's Regional Medical Center, Paterson
New YorkAlbany Medical Center, Albany
Maimonides Medical Center, Brooklyn
The Steven and Alexandra Cohen Children's Medical Center of New York, New Hyde Park
Laura and Isaac Perlmutter Cancer Center at NYU Langone, New York
Memorial Sloan Kettering Cancer Center, New York
Mount Sinai Hospital, New York
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center, New York
NYP/Weill Cornell Medical Center, New York
State University of New York Upstate Medical University, Syracuse
Montefiore Medical Center - Moses Campus, The Bronx
North CarolinaMission Hospital, Asheville
Duke University Medical Center, Durham
East Carolina University, Greenville
Wake Forest University Health Sciences, Winston-Salem
OhioRainbow Babies and Childrens Hospital, Cleveland
Nationwide Children's Hospital, Columbus
Dayton Children's Hospital, Dayton
OklahomaUniversity of Oklahoma Health Sciences Center, Oklahoma City
OregonOregon Health and Science University, Portland
PennsylvaniaGeisinger Medical Center, Danville
Children's Hospital of Philadelphia, Philadelphia
Saint Christopher's Hospital for Children, Philadelphia
Children's Hospital of Pittsburgh of UPMC, Pittsburgh
South CarolinaPrisma Health Richland Hospital, Columbia
BI-LO Charities Children's Cancer Center, Greenville
South DakotaSanford USD Medical Center - Sioux Falls, Sioux Falls
TennesseeEast Tennessee Childrens Hospital, Knoxville
The Children's Hospital at TriStar Centennial, Nashville
Vanderbilt University/Ingram Cancer Center, Nashville
TexasDell Children's Medical Center of Central Texas, Austin
UT Southwestern/Simmons Cancer Center-Dallas, Dallas
El Paso Children's Hospital, El Paso
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center, Houston
UT MD Anderson Cancer Center, Houston
Children's Hospital of San Antonio, San Antonio
Methodist Children's Hospital of South Texas, San Antonio
University of Texas Health Science Center at San Antonio, San Antonio
Scott and White Memorial Hospital, Temple
UtahPrimary Children's Hospital, Salt Lake City
VirginiaUniversity of Virginia Cancer Center, Charlottesville
Children's Hospital of The King's Daughters, Norfolk
VCU Massey Comprehensive Cancer Center, Richmond
WashingtonSeattle Children's Hospital, Seattle
Providence Sacred Heart Medical Center and Children's Hospital, Spokane
West VirginiaWest Virginia University Charleston Division, Charleston
WisconsinSaint Vincent Hospital Cancer Center Green Bay, Green Bay
Children's Hospital of Wisconsin, Milwaukee

Conditions

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.