Recruiting
Phase 2

Tovorafenib

Sponsor:

National Cancer Institute (NCI)

Code:

NCT05828069

Conditions

Recurrent Langerhans Cell Histiocytosis

Refractory Langerhans Cell Histiocytosis

Eligibility Criteria

Sex: All

Age: 0 - 22

Healthy Volunteers: Not accepted

Interventions

Biospecimen Collection

Bone Marrow Aspiration

Bone Marrow Biopsy

Computed Tomography

Echocardiography Test

Study Details

Brief 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.

Conditions

Recurrent Langerhans Cell Histiocytosis

Refractory Langerhans Cell Histiocytosis

Study ID

NCT05828069

Start date

Mar 28, 2024

Status verified date

Jul, 2026

Completion date

Sep 30, 2028

Anticipated

Primary completion date

Sep 30, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 22

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

48 participants

Anticipated

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (tovorafenib)

Patients receive tovorafenib PO QW on days 1, 8, 15, and 22 of each cycle. Cycles repeat every 28 days for 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo MUGA or ECHO scans, and FDG-PET or CT throughout the trial, and collection of blood and urine samples on study. Patients with suspicion of bone marrow and/or central nervous system involvement will also undergo bone marrow biopsy and aspiration and lumbar puncture on study and during follow up.

Interventions

Biospecimen Collection

Undergo collection of blood and urine samples

Bone Marrow Aspiration

Undergo bone marrow aspiration

Bone Marrow Biopsy

Undergo bone marrow biopsy

Computed Tomography

Undergo CT

Echocardiography Test

Undergo ECHO

FDG-Positron Emission Tomography and Computed Tomography Scan

Undergo FDG-PET imaging

Lumbar Puncture

Undergo lumbar puncture

Multigated Acquisition Scan

Undergo MUGA

Tovorafenib

Given PO

Primary outcome measure

  • Frequency of dose limiting toxicity (dose finding phase) [ Time Frame: Up to 28 days ]
  • Overall response rate (phase II) [ Time Frame: After 2 cycles of therapy (each cycle is 28 days) ]

Central Contacts and Locations

Locations

Children's Hospital of Alabama

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

Site Public Contact

205-638-9285oncologyresearch@peds.uab.edu

Principal Investigator:

Matthew A. Kutny

Arkansas Children's Hospital

Recruiting

Little Rock, Arkansas, United States, 72202-3591

Contacts

Site Public Contact

501-364-7373

Principal Investigator:

Michael W. Bishop

Kaiser Permanente Downey Medical Center

Recruiting

Downey, California, United States, 90242

Contacts

Site Public Contact

626-564-3455

Principal Investigator:

Robert M. Cooper

Loma Linda University Medical Center

Recruiting

Loma Linda, California, United States, 92354

Contacts

Site Public Contact

909-558-4050

Principal Investigator:

Albert Kheradpour

Miller Children's and Women's Hospital Long Beach

Recruiting

Long Beach, California, United States, 90806

Contacts

Site Public Contact

562-933-5600

Principal Investigator:

Jacqueline N. Casillas

Children's Hospital Los Angeles

Recruiting

Los Angeles, California, United States, 90027

Contacts

Site Public Contact

323-361-4110

Principal Investigator:

Rebecca S. Parker

UCSF Benioff Children's Hospital Oakland

Recruiting

Oakland, California, United States, 94609

Contacts

Site Public Contact

510-428-3264PedOncRschOAK@ucsf.edu

Principal Investigator:

Jennifer G. Michlitsch

Kaiser Permanente-Oakland

Recruiting

Oakland, California, United States, 94611

Contacts

Site Public Contact

877-642-4691Kpoct@kp.org

Principal Investigator:

Aarati V. Rao

Children's Hospital of Orange County

Recruiting

Orange, California, United States, 92868

Contacts

Site Public Contact

714-509-8646oncresearch@choc.org

Principal Investigator:

Elyssa M. Rubin

Lucile Packard Children's Hospital Stanford University

Recruiting

Palo Alto, California, United States, 94304

Contacts

Site Public Contact

800-694-0012ccto-office@stanford.edu

Principal Investigator:

Jay Michael S. Balagtas

UCSF Medical Center-Mission Bay

Recruiting

San Francisco, California, United States, 94158

Contacts

Site Public Contact

877-827-3222cancertrials@ucsf.edu

Principal Investigator:

Natalie L. Wu

Children's Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Site Public Contact

303-764-5056josh.b.gordon@nsmtp.kp.org

Principal Investigator:

Timothy P. Garrington

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

Recruiting

Denver, Colorado, United States, 80218

Contacts

Site Public Contact

303-832-2344PSGResearchSharedMailbox@HCAHealthcare.com

Principal Investigator:

Florence Choo

Connecticut Children's Medical Center

Recruiting

Hartford, Connecticut, United States, 06106

Contacts

Site Public Contact

860-545-9981

Principal Investigator:

Michael S. Isakoff

Yale University

Recruiting

New Haven, Connecticut, United States, 06520

Contacts

Site Public Contact

203-785-5702canceranswers@yale.edu

Principal Investigator:

Farzana Pashankar

Alfred I duPont Hospital for Children

Recruiting

Wilmington, Delaware, United States, 19803

Contacts

Site Public Contact

302-651-5572Allison.bruce@nemours.org

Principal Investigator:

Vibhuti Agarwal

Children's National Medical Center

Recruiting

Washington D.C., District of Columbia, United States, 20010

Contacts

Site Public Contact

202-476-2800OncCRC_OnCall@childrensnational.org

Principal Investigator:

Jeffrey S. Dome

Golisano Children's Hospital of Southwest Florida

Recruiting

Fort Myers, Florida, United States, 33908

Contacts

Site Public Contact

239-343-5333molly.arnstrom@leehealth.org

Principal Investigator:

Emad K. Salman

UF Health Cancer Institute - Gainesville

Recruiting

Gainesville, Florida, United States, 32610

Contacts

Site Public Contact

352-273-8010

Principal Investigator:

Brian Stover

Memorial Regional Hospital/Joe DiMaggio Children's Hospital

Recruiting

Hollywood, Florida, United States, 33021

Contacts

Site Public Contact

954-265-1847OHR@mhs.net

Principal Investigator:

Iftikhar Hanif

Nemours Children's Clinic-Jacksonville

Recruiting

Jacksonville, Florida, United States, 32207

Contacts

Site Public Contact

302-651-5572Allison.bruce@nemours.org

Principal Investigator:

Vibhuti Agarwal

University of Miami Miller School of Medicine-Sylvester Cancer Center

Recruiting

Miami, Florida, United States, 33136

Contacts

Site Public Contact

305-243-2647

Principal Investigator:

Warren Alperstein

Nicklaus Children's Hospital

Recruiting

Miami, Florida, United States, 33155

Contacts

Site Public Contact

888-624-2778

Principal Investigator:

Maggie E. Fader

Arnold Palmer Hospital for Children

Recruiting

Orlando, Florida, United States, 32806

Contacts

Site Public Contact

321-841-5357Jennifer.spinelli@orlandohealth.com

Principal Investigator:

Zhongbo Hu

Nemours Children's Hospital

Recruiting

Orlando, Florida, United States, 32827

Contacts

Site Public Contact

302-651-5572Allison.bruce@nemours.org

Principal Investigator:

Vibhuti Agarwal

Nemours Children's Clinic - Pensacola

Recruiting

Pensacola, Florida, United States, 32504

Contacts

Site Public Contact

helpdesk@childrensoncologygroup.org

Principal Investigator:

Jeffrey H. Schwartz

Johns Hopkins All Children's Hospital

Recruiting

St. Petersburg, Florida, United States, 33701

Contacts

Site Public Contact

727-767-4784Ashley.Repp@jhmi.edu

Principal Investigator:

Jennifer B. Dean

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

Recruiting

Tampa, Florida, United States, 33607

Contacts

Site Public Contact

813-357-0849jennifer.manns@baycare.org

Principal Investigator:

Don E. Eslin

Children's Healthcare of Atlanta - Arthur M Blank Hospital

Recruiting

Atlanta, Georgia, United States, 30329

Contacts

Site Public Contact

404-785-0232Olivia.Floyd@choa.org

Principal Investigator:

Kathryn S. Sutton

Memorial Health University Medical Center

Recruiting

Savannah, Georgia, United States, 31404

Contacts

Site Public Contact

912-350-7887Lorraine.OHara@hcahealthcare.com

Principal Investigator:

Andrew L. Pendleton

Kapiolani Medical Center for Women and Children

Recruiting

Honolulu, Hawaii, United States, 96826

Contacts

Site Public Contact

808-983-6090

Principal Investigator:

Wade T. Kyono

Lurie Children's Hospital-Chicago

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Site Public Contact

773-880-4562

Principal Investigator:

Joanna L. Weinstein

University of Chicago Comprehensive Cancer Center

Recruiting

Chicago, Illinois, United States, 60637

Contacts

Site Public Contact

773-702-8222cancerclinicaltrials@bsd.uchicago.edu

Principal Investigator:

Perry C. Morocco

OSF Children's Hospital of Illinois

Recruiting

Peoria, Illinois, United States, 61637

Contacts

Site Public Contact

309-624-4945ChildrensHospitalofIllinois@osfhealthcare.org

Principal Investigator:

Prerna Kumar

Riley Hospital for Children

Recruiting

Indianapolis, Indiana, United States, 46202

Contacts

Site Public Contact

800-248-1199

Principal Investigator:

Jennifer A. Belsky

Blank Children's Hospital

Recruiting

Des Moines, Iowa, United States, 50309

Contacts

Site Public Contact

515-241-8912samantha.mallory@unitypoint.org

Principal Investigator:

Samantha L. Mallory

University of Iowa/Holden Comprehensive Cancer Center

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Site Public Contact

800-237-1225

Principal Investigator:

Andrew P. Groves

University of Kentucky/Markey Cancer Center

Recruiting

Lexington, Kentucky, United States, 40536

Contacts

Site Public Contact

859-257-3379

Principal Investigator:

James T. Badgett

Children's Hospital New Orleans

Recruiting

New Orleans, Louisiana, United States, 70118

Contacts

Site Public Contact

504-894-5377

Principal Investigator:

Maria C. Velez-Yanguas

Ochsner Medical Center Jefferson

Recruiting

New Orleans, Louisiana, United States, 70121

Contacts

Site Public Contact

504-842-8084Elisemarie.curry@ochsner.org

Principal Investigator:

Craig Lotterman

Sinai Hospital of Baltimore

Recruiting

Baltimore, Maryland, United States, 21215

Contacts

Site Public Contact

410-601-9083

Principal Investigator:

Jason M. Fixler

Johns Hopkins University/Sidney Kimmel Cancer Center

Recruiting

Baltimore, Maryland, United States, 21287

Contacts

Site Public Contact

410-955-8804jhcccro@jhmi.edu

Principal Investigator:

Elias Zambidis

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

Recruiting

Grand Rapids, Michigan, United States, 49503

Contacts

Site Public Contact

616-391-1230crcwm-regulatory@crcwm.org

Principal Investigator:

Kathleen Y. Butler

Bronson Methodist Hospital

Recruiting

Kalamazoo, Michigan, United States, 49007

Contacts

Site Public Contact

616-391-1230crcwm-regulatory@crcwm.org

Principal Investigator:

Kathleen Y. Butler

Children's Hospitals and Clinics of Minnesota - Minneapolis

Recruiting

Minneapolis, Minnesota, United States, 55404

Contacts

Site Public Contact

612-813-5913pauline.mitby@childrensmn.org

Principal Investigator:

Michael K. Richards

University of Mississippi Medical Center

Recruiting

Jackson, Mississippi, United States, 39216

Contacts

Site Public Contact

601-815-6700

Principal Investigator:

Amanda Strobel

Cardinal Glennon Children's Medical Center

Recruiting

St Louis, Missouri, United States, 63104

Contacts

Site Public Contact

314-268-4000

Principal Investigator:

William S. Ferguson

Washington University School of Medicine

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Site Public Contact

800-600-3606info@siteman.wustl.edu

Principal Investigator:

Bryan A. Sisk

Mercy Hospital Saint Louis

Recruiting

St Louis, Missouri, United States, 63141

Contacts

Site Public Contact

314-251-7066

Principal Investigator:

Robin D. Hanson

Children's Hospital and Medical Center of Omaha

Recruiting

Omaha, Nebraska, United States, 68114

Contacts

Site Public Contact

402-955-3949

Principal Investigator:

Jill C. Beck

University of Nebraska Medical Center

Recruiting

Omaha, Nebraska, United States, 68198

Contacts

Site Public Contact

402-559-6941unmcrsa@unmc.edu

Principal Investigator:

Jill C. Beck

Alliance for Childhood Diseases/Cure 4 the Kids Foundation

Recruiting

Las Vegas, Nevada, United States, 89135

Contacts

Site Public Contact

702-384-0013research@sncrf.org

Principal Investigator:

Alan K. Ikeda

Summerlin Hospital Medical Center

Recruiting

Las Vegas, Nevada, United States, 89144

Contacts

Site Public Contact

702-384-0013research@sncrf.org

Principal Investigator:

Alan K. Ikeda

Hackensack University Medical Center

Recruiting

Hackensack, New Jersey, United States, 07601

Contacts

Site Public Contact

551-996-2897

Principal Investigator:

Katharine Offer

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

Recruiting

New Brunswick, New Jersey, United States, 08903

Contacts

Site Public Contact

732-235-8675

Principal Investigator:

Marissa Botwinick

Saint Joseph's Regional Medical Center

Recruiting

Paterson, New Jersey, United States, 07503

Contacts

Site Public Contact

973-754-2207HallL@sjhmc.org

Principal Investigator:

Alissa Kahn

Albany Medical Center

Recruiting

Albany, New York, United States, 12208

Contacts

Site Public Contact

518-262-5513

Principal Investigator:

Lauren R. Weintraub

Maimonides Medical Center

Recruiting

Brooklyn, New York, United States, 11219

Contacts

Site Public Contact

718-765-2500

Principal Investigator:

Mahmut Y. Celiker

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

Recruiting

New Hyde Park, New York, United States, 11040

Contacts

Site Public Contact

718-470-3460

Principal Investigator:

Julie I. Krystal

Laura and Isaac Perlmutter Cancer Center at NYU Langone

Recruiting

New York, New York, United States, 10016

Contacts

Site Public Contact

CancerTrials@nyulangone.org

Principal Investigator:

Elizabeth A. Raetz

Mount Sinai Hospital

Recruiting

New York, New York, United States, 10029

Contacts

Site Public Contact

212-824-7309CCTO@mssm.edu

Principal Investigator:

Pamela R. Merola

NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

Recruiting

New York, New York, United States, 10032

Contacts

Site Public Contact

212-342-5162cancerclinicaltrials@cumc.columbia.edu

Principal Investigator:

Nobuko Hijiya

Memorial Sloan Kettering Cancer Center

Recruiting

New York, New York, United States, 10065

Contacts

Site Public Contact

212-639-7592

Principal Investigator:

Christopher J. Forlenza

NYP/Weill Cornell Medical Center

Recruiting

New York, New York, United States, 10065

Contacts

Site Public Contact

212-746-1848

Principal Investigator:

Nitya Gulati

State University of New York Upstate Medical University

Recruiting

Syracuse, New York, United States, 13210

Contacts

Site Public Contact

315-464-5476

Principal Investigator:

Melanie A. Comito

Mission Hospital

Recruiting

Asheville, North Carolina, United States, 28801

Contacts

Site Public Contact

828-213-7055NCDV.ResearchRegulatory@HCAHealthcare.com

Principal Investigator:

Douglas J. Scothorn

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Site Public Contact

888-275-3853

Principal Investigator:

Jessica M. Sun

East Carolina University

Recruiting

Greenville, North Carolina, United States, 27834

Contacts

Site Public Contact

252-744-1015eubankss@ecu.edu

Principal Investigator:

Andrea R. Whitfield

Wake Forest University Health Sciences

Recruiting

Winston-Salem, North Carolina, United States, 27157

Contacts

Site Public Contact

336-713-6771

Principal Investigator:

Sarah Supples

Rainbow Babies and Childrens Hospital

Recruiting

Cleveland, Ohio, United States, 44106

Contacts

Site Public Contact

216-844-5437

Principal Investigator:

Duncan S. Stearns

Nationwide Children's Hospital

Recruiting

Columbus, Ohio, United States, 43205

Contacts

Site Public Contact

614-722-6039Melinda.Triplet@nationwidechildrens.org

Principal Investigator:

Mark A. Ranalli

Dayton Children's Hospital

Recruiting

Dayton, Ohio, United States, 45404

Contacts

Site Public Contact

800-228-4055

Principal Investigator:

Jordan M. Wright

University of Oklahoma Health Sciences Center

Recruiting

Oklahoma City, Oklahoma, United States, 73104

Contacts

Site Public Contact

405-271-8777ou-clinical-trials@ouhsc.edu

Principal Investigator:

Rene Y. McNall-Knapp

Oregon Health and Science University

Recruiting

Portland, Oregon, United States, 97239

Contacts

Site Public Contact

503-494-1080trials@ohsu.edu

Principal Investigator:

Katrina Winsnes

Geisinger Medical Center

Recruiting

Danville, Pennsylvania, United States, 17822

Contacts

Site Public Contact

570-271-5251cancerresearch@geisinger.edu

Principal Investigator:

Jagadeesh Ramdas

Saint Christopher's Hospital for Children

Recruiting

Philadelphia, Pennsylvania, United States, 19134

Contacts

Site Public Contact

215-427-8991

Principal Investigator:

Gregory E. Halligan

Children's Hospital of Pittsburgh of UPMC

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Contacts

Site Public Contact

412-692-8570jean.tersak@chp.edu

Principal Investigator:

Julia E. Segal

Prisma Health Richland Hospital

Recruiting

Columbia, South Carolina, United States, 29203

Contacts

Site Public Contact

864-522-4317Kim.Williams3@prismahealth.org

Principal Investigator:

Stuart L. Cramer

BI-LO Charities Children's Cancer Center

Recruiting

Greenville, South Carolina, United States, 29605

Contacts

Site Public Contact

864-522-4317Kim.Williams3@prismahealth.org

Principal Investigator:

Aniket Saha

Sanford USD Medical Center - Sioux Falls

Recruiting

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

Contacts

Site Public Contact

605-312-3320OncologyClinicalTrialsSF@SanfordHealth.org

Principal Investigator:

Jordan Fritch-Hanson

East Tennessee Childrens Hospital

Recruiting

Knoxville, Tennessee, United States, 37916

Contacts

Site Public Contact

865-541-8266

Principal Investigator:

Susan E. Spiller

The Children's Hospital at TriStar Centennial

Recruiting

Nashville, Tennessee, United States, 37203

Contacts

Site Public Contact

615-342-1919

Principal Investigator:

Clinton M. Carroll

Vanderbilt University/Ingram Cancer Center

Recruiting

Nashville, Tennessee, United States, 37232

Contacts

Site Public Contact

800-811-8480

Principal Investigator:

Devang J. Pastakia

Dell Children's Medical Center of Central Texas

Recruiting

Austin, Texas, United States, 78723

Contacts

Site Public Contact

512-628-1902TXAUS-DL-SFCHemonc.research@ascension.org

Principal Investigator:

Shannon M. Cohn

UT Southwestern/Simmons Cancer Center-Dallas

Recruiting

Dallas, Texas, United States, 75390

Contacts

Site Public Contact

214-648-7097canceranswerline@UTSouthwestern.edu

Principal Investigator:

Erin B. Butler

El Paso Children's Hospital

Recruiting

El Paso, Texas, United States, 79905

Contacts

Site Public Contact

915-298-5444ranjan.bista@ttuhsc.edu

Principal Investigator:

Benjamin Carcamo

Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Site Public Contact

713-798-1354burton@bcm.edu

Principal Investigator:

Olive S. Eckstein

UT MD Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Site Public Contact

866-632-6789askmdanderson@mdanderson.org

Principal Investigator:

Najat C. Daw

Children's Hospital of San Antonio

Recruiting

San Antonio, Texas, United States, 78207

Contacts

Site Public Contact

210-704-2894bridget.medina@christushealth.org

Principal Investigator:

Julie Voeller

Methodist Children's Hospital of South Texas

Recruiting

San Antonio, Texas, United States, 78229

Contacts

Site Public Contact

210-575-6240Vinod.GidvaniDiaz@hcahealthcare.com

Principal Investigator:

Jose M. Esquilin

University of Texas Health Science Center at San Antonio

Recruiting

San Antonio, Texas, United States, 78229

Contacts

Site Public Contact

210-450-3800phoresearchoffice@uthscsa.edu

Principal Investigator:

Allison C. Grimes

Scott and White Memorial Hospital

Recruiting

Temple, Texas, United States, 76508

Contacts

Site Public Contact

254-724-5407

Principal Investigator:

Nicholas W. McGregor

Primary Children's Hospital

Recruiting

Salt Lake City, Utah, United States, 84113

Contacts

Site Public Contact

801-585-5270

Principal Investigator:

Taumoha Ghosh

University of Virginia Cancer Center

Recruiting

Charlottesville, Virginia, United States, 22908

Contacts

Site Public Contact

434-243-6303uvacancertrials@hscmail.mcc.virginia.edu

Principal Investigator:

Brian C. Belyea

Children's Hospital of The King's Daughters

Recruiting

Norfolk, Virginia, United States, 23507

Contacts

Site Public Contact

757-668-7243CCBDCresearch@chkd.org

Principal Investigator:

Melissa S. Mark

VCU Massey Comprehensive Cancer Center

Recruiting

Richmond, Virginia, United States, 23298

Contacts

Site Public Contact

804-628-6430CTOclinops@vcu.edu

Principal Investigator:

Nadirah El-Amin

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105

Contacts

Site Public Contact

866-987-2000

Principal Investigator:

Sarah E. Leary

Providence Sacred Heart Medical Center and Children's Hospital

Recruiting

Spokane, Washington, United States, 99204

Contacts

Site Public Contact

800-228-6618HopeBeginsHere@providence.org

Principal Investigator:

Judy L. Felgenhauer

West Virginia University Charleston Division

Recruiting

Charleston, West Virginia, United States, 25304

Contacts

Site Public Contact

304-388-9944

Principal Investigator:

Mohamad H. Badawi

Saint Vincent Hospital Cancer Center Green Bay

Recruiting

Green Bay, Wisconsin, United States, 54301

Contacts

Site Public Contact

920-433-8889WI_research_admin@hshs.org

Principal Investigator:

Catherine A. Long

Children's Hospital of Wisconsin

Recruiting

Milwaukee, Wisconsin, United States, 53226

Contacts

Site Public Contact

414-955-4727MACCCTO@mcw.edu

Principal Investigator:

Peter H. Shaw

University of Alberta Hospital

Recruiting

Edmonton, Alberta, Canada, T6G 2B7

Contacts

Site Public Contact

780-407-8798pedsoncologyresearch@ahs.ca

Principal Investigator:

Sarah J. McKillop

British Columbia Children's Hospital

Recruiting

Vancouver, British Columbia, Canada, V6H 3V4

Contacts

Site Public Contact

604-875-2345

Principal Investigator:

Rebecca J. Deyell

IWK Health Centre

Recruiting

Halifax, Nova Scotia, Canada, B3K 6R8

Contacts

Site Public Contact

902-470-8520Research@iwk.nshealth.ca

Principal Investigator:

Chelsea Ash

Centre Hospitalier Universitaire Sainte-Justine

Recruiting

Montreal, Quebec, Canada, H3T 1C5

Contacts

Site Public Contact

514-345-4931yvan.samson@umontreal.ca

Principal Investigator:

Monia Marzouki

CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)

Recruiting

Québec, Canada, G1V 4G2

Contacts

Site Public Contact

418-525-4444rechclinique@crchudequebec.ulaval.ca

Principal Investigator:

Bruno Michon

More Information

Sponsor

National Cancer Institute (NCI)

Last update posted

Sep 4, 2026

Last verified

Jul, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by National Cancer Institute (NCI) on 2026-09-04.