CClinicalTrials.gg
Active, not recruitingNCT03210714Updated Sep 22, 2026Results posted

Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)

A Phase 2 interventional study of Biospecimen Collection and Bone Marrow Aspiration and Biopsy in Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma and Recurrent Childhood Malignant Germ Cell Tumor, sponsored by National Cancer Institute (NCI). Active, not recruiting at 116 sites in 2 countries. Open to participants aged 12 Months to 21 Years. Per ClinicalTrials.gov, last updated 2026-09-22.

Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
20
Allocation
Not applicable
Ages
12 Months to 21 Years
Sex
All
01

Study summary

This phase II Pediatric MATCH trial studies how well erdafitinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with FGFR mutations that have spread to other places in the body and have come back or do not respond to treatment. Erdafitinib may stop the growth of cancer cells with FGFR mutations by blocking some of the enzymes needed for cell growth.

Read the detailed description

PRIMARY OBJECTIVE:

I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with erdafitinib with advanced solid tumors (including central nervous system [CNS] tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor genetic alterations in the FGFR1/2/3/4 pathway.

SECONDARY OBJECTIVES:

I. To estimate the progression free survival in pediatric patients treated with erdafitinib with advanced solid tumors (including CNS tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor genetic alterations in the FGFR1/2/3/4.

II. To obtain information about the tolerability of erdafitinib in children with relapsed or refractory cancer.

III. To provide preliminary estimates of the pharmacokinetics of erdafitinib in children with relapsed or refractory cancer.

EXPLORATORY OBJECTIVE:

I. To explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid (DNA).

OUTLINE:

Patients receive erdafitinib orally (PO) once daily (QD) on days 1-28 of each cycle. Treatment repeats every 28 days for up to 26 cycles (2 years) in the absence of disease progression or unacceptable toxicity. Patients undergo an x-ray, computed tomography (CT) scan, magnetic resonance imaging (MRI), positron emission tomography (PET) scan, radionuclide imaging, and/or bone scan, as well as a bone marrow aspiration and/or biopsy during screening and on study. Patients also undergo blood sample collection on study.

After completion of study treatment, patients are followed up periodically.

02

Conditions studied

  • Advanced Malignant Solid Neoplasm
  • Recurrent Childhood Ependymoma
  • Recurrent Childhood Malignant Germ Cell Tumor
  • Recurrent Childhood Medulloblastoma
  • Recurrent Childhood Non-Hodgkin Lymphoma
  • Recurrent Childhood Osteosarcoma
  • Recurrent Childhood Rhabdomyosarcoma
  • Recurrent Childhood Soft Tissue Sarcoma
  • Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
  • Recurrent Hepatoblastoma
  • Recurrent Langerhans Cell Histiocytosis
  • Recurrent Malignant Glioma
  • Recurrent Malignant Solid Neoplasm
  • Recurrent Neuroblastoma
  • Recurrent Primary Central Nervous System Neoplasm
  • Recurrent Rhabdoid Tumor
  • Refractory Childhood Malignant Germ Cell Tumor
  • Refractory Childhood Osteosarcoma
  • Refractory Childhood Rhabdomyosarcoma
  • Refractory Childhood Soft Tissue Sarcoma
  • Refractory Ependymoma
  • Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
  • Refractory Hepatoblastoma
  • Refractory Langerhans Cell Histiocytosis
  • Refractory Malignant Glioma
  • Refractory Malignant Solid Neoplasm
  • Refractory Medulloblastoma
  • Refractory Neuroblastoma
  • Refractory Non-Hodgkin Lymphoma
  • Refractory Primary Central Nervous System Neoplasm
  • Refractory Rhabdoid Tumor
  • Wilms Tumor
03

Who can participate

Ages eligible
12 Months to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to molecular analysis for therapy choice (MATCH) to APEC1621B based on the presence of an actionable mutation as defined in APEC1621SC
  • Patients must be >/= 12 months and =/\< 21 years of age at the time of study enrollment
  • Patients must have a body surface area >/= 0.53 m\^2 at enrollment
  • Patients must have radiographically measurable disease at the time of study enrollment; patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as lesion that is at minimum 10 mm in one dimension on standard MRI or CT

    • Note: The following do not qualify as measurable disease:

      • Malignant fluid collections (e.g., ascites, pleural effusions)
      • Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
      • Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
      • Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
      • Previously radiated lesions that have not demonstrated clear progression post radiation
      • Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
  • Karnofsky >/= 50% for patients > 16 years of age and Lansky >/= 50 for patients =/\< 16 years of age; Note: neurologic deficits in patients with CNS tumors must have been relatively stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
  • Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment; if after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately

    • Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive; >/= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
    • Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil count [ANC] counts): >/= 7 days after the last dose of agent
    • Antibodies: >/= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =/\< 1
    • Corticosteroids: if used to modify immune adverse events related to prior therapy, >/= 14 days must have elapsed since last dose of corticosteroid
    • Hematopoietic growth factors: >/= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor; for growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
    • Interleukins, interferons and cytokines (other than hematopoietic growth factors): >/= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
    • Stem cell infusions (with or without total body irradiation [TBI]):

      • Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >/= 84 days after infusion and no evidence of graft versus host disease (GVHD)
      • Autologous stem cell infusion including boost infusion: >/= 42 days
    • Cellular therapy: >/= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer [NK] cells, dendritic cells, etc.)
    • X-ray therapy (XRT)/external beam irradiation including protons: >/= 14 days after local XRT; >/= 150 days after TBI, craniospinal XRT or if radiation to >/= 50% of the pelvis; >/= 42 days if other substantial bone marrow (BM) radiation

      • Note: radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
    • Radiopharmaceutical therapy (e.g., radiolabeled antibody, iobenguane I-131 [131I-MIBG]): >/= 42 days after systemically administered radiopharmaceutical therapy
    • Patients must not have received prior exposure to erdafitinib or another FGFR inhibitor such as (but not limited to) AZD4547, BGJ398, BAY1163877, LY2874455
  • For patients with solid tumors without known bone marrow involvement:

    • Peripheral absolute neutrophil count (ANC) >/= 1000/mm\^3 (performed within 7 days prior to enrollment)
    • Platelet count >/= 100,000/mm\^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) (performed within 7 days prior to enrollment)
    • Hemoglobin >/= 8.0 g/dL at baseline (may receive red blood cell [RBC] transfusions) (performed within 7 days prior to enrollment)
  • Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive platelet or packed [p]RBC transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) >/= 70 ml/min/1.73 m\^2 or a serum creatinine based on age/gender as follows (performed within 7 days prior to enrollment):

    • Age: 1 to \< 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
    • Age: 2 to \< 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
    • Age: 6 to \< 10 years; maximum serum creatinine (mg/dL): male 1; female 1
    • Age: 10 to \< 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
    • Age: 13 to \< 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
    • Age: >/= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
  • Bilirubin (sum of conjugated + unconjugated) =/\< 1.5 x upper limit of normal (ULN) for age (performed within 7 days prior to enrollment)
  • Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =/\< 135 U/L; (for the purpose of this study, the ULN for SGPT is 45 U/L) (performed within 7 days prior to enrollment)
  • Serum albumin >/= 2 g/dL (performed within 7 days prior to enrollment)
  • Corrected QT (QTc) interval =/\< 480 milliseconds
  • Pulse oximetry > 94% on room air if there is clinical indication for determination (e.g. dyspnea at rest)
  • Patients must be able to swallow intact tablets
  • All patients and/or their parents or legally authorized representatives must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines

Exclusion criteria

Exclusion Criteria:

  • Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal studies; pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method, while receiving study treatment and for 3 months after the last dose of erdafitinib; male subjects (with a partner of child-bearing potential) must use a condom with spermicide when sexually active and must not donate sperm from the first dose of study drug until 3 months after the last dose of study drug
  • Concomitant medications

    • Corticosteroids: patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, >/= 14 days must have elapsed since last dose of corticosteroid
    • Investigational drugs: patients who are currently receiving another investigational drug are not eligible
    • Anti-cancer agents: patients who are currently receiving other anti-cancer agents are not eligible
    • Anti-GVHD agents post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
    • CYP3A4 agents: patients who are currently receiving drugs that are strong inducers or inhibitors of CYP3A4 are not eligible; Note: CYP3A4 inducing anti-epileptic drugs and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed
    • CYP2C9 agents: patients who are currently receiving drugs that are strong inducers or moderate inhibitors of CYP2C9 are not eligible
  • Patients who have an uncontrolled infection are not eligible
  • Patients who have received a prior solid organ transplantation are not eligible
  • Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
  • A history of cardiovascular diseases: unstable angina, myocardial infarction, or known congestive heart failure class II-IV within the preceding 12 months; cerebrovascular accident or transient ischemic attack within the preceding 3 months, pulmonary embolism within the preceding 2 months
  • A history of any of the following: sustained ventricular tachycardia, ventricular fibrillation, torsades de pointes, cardiac arrest, Mobitz II second degree heart block or third degree heart block; known presence of dilated, hypertrophic, or restrictive cardiomyopathy
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  • Patients with significant ophthalmologic conditions (uncontrolled glaucoma, central serous retinopathy, history of retinal vein occlusion or retinal detachment, excluding patients with longstanding findings secondary to existing conditions) are not eligible, to be confirmed with baseline ophthalmologic exam; all patients must have a baseline ophthalmologic exam, including fundoscopy to confirm no significant ophthalmologic conditions are present
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
20 participants (actual)

Study arms

  • Experimental
    Treatment (erdafitinib)

    Patients receive erdafitinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 26 cycles (2 years) in the absence of disease progression or unacceptable toxicity. Patients undergo an x-ray, CT scan, MRI, PET scan, radionuclide imaging, and/or bone scan, as well as a bone marrow aspiration and/or biopsy during screening and on study. Patients also undergo blood sample collection on study.

    Procedure: Biospecimen Collection · Procedure: Bone Marrow Aspiration and Biopsy · Procedure: Bone Scan · Procedure: Computed Tomography · Drug: Erdafitinib · Other: Laboratory Biomarker Analysis · Procedure: Magnetic Resonance Imaging · Other: Pharmacological Study · Procedure: Positron Emission Tomography · Procedure: Radionuclide Imaging · Procedure: X-Ray Imaging

Interventions

  • ProcedureBiospecimen Collection

    Undergo blood sample collection

    Also known as: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

  • ProcedureBone Marrow Aspiration and Biopsy

    Undergo a bone marrow aspiration and/or biopsy

  • ProcedureBone Scan

    Undergo a bone scan

    Also known as: Bone Scintigraphy

  • ProcedureComputed Tomography

    Undergo a CT scan

    Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography

  • DrugErdafitinib

    Given PO

    Also known as: Balversa, JNJ 42756493, JNJ-42756493, JNJ42756493

  • OtherLaboratory Biomarker Analysis

    Correlative studies

  • ProcedureMagnetic Resonance Imaging

    Undergo MRI

    Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI

  • OtherPharmacological Study

    Correlative studies

  • ProcedurePositron Emission Tomography

    Undergo a PET scan

    Also known as: Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure), Positron Emission Tomography Scan, Positron-Emission Tomography, PT

  • ProcedureRadionuclide Imaging

    Undergo radionuclide imaging

    Also known as: Gamma Scan, NM, Nuclear Medicine, nuclear medicine scan, radioimaging, Radionuclide Scanning, Scan, Scintigraphy

  • ProcedureX-Ray Imaging

    Undergo an x-ray

    Also known as: Conventional X-Ray, Diagnostic Radiology, Medical Imaging, X-Ray, Plain film radiographs, Radiographic Imaging, Radiographic imaging procedure (procedure), Radiography, RG, Static X-Ray, X-Ray

05

What researchers measure

Primary outcomes

  1. Objective Response Rate

    A responder is defined as a patient who achieves a best response of partial response or complete response on the study. Response rates will be calculated as the percent of evaluable patients who are responders. The revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to determine response and progression in this study, with specific criteria outlined for the different subtypes of tumors (e.g., 2-dimensional measurements for central nervous system (CNS) tumors).

    Time frame: Up to 2 years from study entry

Secondary outcomes

  1. Percentage of Patients Experiencing Grade 3 or Higher Adverse Events

    Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0

    Time frame: Up to 2 years from study entry

  2. Progression Free Survival (PFS)

    The Kaplan-Meier method will be used to estimate the 6 month PFS. PFS is defined as time from initiation of protocol treatment to disease progression, recurrence, death from any cause, or date of last contact

    Time frame: Up to 6 months from study entry

  3. Pharmacokinetic (PK) Parameters

    A descriptive analysis of PK parameters will be performed to define systemic exposure, drug clearance, and other pharmacokinetic parameters. The PK parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit).

    Time frame: Pre-dose Cycle 2 Day 1; 1-hour post-dose Cycle 2 Day 1; 2-hour post dose Cycle 2, Day 1; 4-hour post-dose Cycle 2, Day 1; 6-8-hour post-dose, Cycle 2 Day 1; 24-hour post dose, Cycle 2, Day 2

Other outcomes

  1. Changes in Tumor Genomic Profile

    A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

    Time frame: Up to 3 years

06

Results

Posted Sep 19, 2024

Participant flow

Participant flow — Overall Study
MilestoneTreatment (Erdafitinib)
Started20
Completed0
Not completed20
Withdrew: Adverse event6
Withdrew: Physician decision1
Withdrew: Progressive disease11
Withdrew: Refusal by patient/parent/guardian2

Outcome measures

PrimaryObjective Response Rate

A responder is defined as a patient who achieves a best response of partial response or complete response on the study. Response rates will be calculated as the percent of evaluable patients who are responders. The revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to determine response and progression in this study, with specific criteria outlined for the different subtypes of tumors (e.g., 2-dimensional measurements for central nervous system (CNS) tumors).

Time frame:
Up to 2 years from study entry
Reported as:
Number · percentage of patients
Objective Response Rate
percentage of patientsTreatment (Erdafitinib)
Objective Response Rate10 (3.3 to 26.2)
SecondaryPercentage of Patients Experiencing Grade 3 or Higher Adverse Events

Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0

Time frame:
Up to 2 years from study entry

Results for this outcome have not been posted.

SecondaryProgression Free Survival (PFS)

The Kaplan-Meier method will be used to estimate the 6 month PFS. PFS is defined as time from initiation of protocol treatment to disease progression, recurrence, death from any cause, or date of last contact

Time frame:
Up to 6 months from study entry

Results for this outcome have not been posted.

SecondaryPharmacokinetic (PK) Parameters

A descriptive analysis of PK parameters will be performed to define systemic exposure, drug clearance, and other pharmacokinetic parameters. The PK parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit).

Time frame:
Pre-dose Cycle 2 Day 1; 1-hour post-dose Cycle 2 Day 1; 2-hour post dose Cycle 2, Day 1; 4-hour post-dose Cycle 2, Day 1; 6-8-hour post-dose, Cycle 2 Day 1; 24-hour post dose, Cycle 2, Day 2

Results for this outcome have not been posted.

Other pre-specifiedChanges in Tumor Genomic Profile

A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

Time frame:
Up to 3 years

Results for this outcome have not been posted.

Adverse events

Collected over Up to 2 years from study entry.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment (Erdafitinib)8/20 (40%)10/20 (50%)19/20 (95%)
Most frequent serious events
Showing 10 of 25
Most frequent serious events
EventTreatment (Erdafitinib)
Infections and infestations - Other, specifyInfections and infestations2/20
Vascular disorders - Other, specifyVascular disorders2/20
EnterocolitisGastrointestinal disorders1/20
Disease progressionGeneral disorders and administration site conditions1/20
General disorders and administration site conditions - Other, specifyGeneral disorders and administration site conditions1/20
PainGeneral disorders and administration site conditions1/20
Urinary tract infectionInfections and infestations1/20
Alkaline phosphatase increasedInvestigations1/20
AnorexiaMetabolism and nutrition disorders1/20
HyperphosphatemiaMetabolism and nutrition disorders1/20
Most frequent other events
Showing 10 of 107
Most frequent other events
EventTreatment (Erdafitinib)
HyperphosphatemiaMetabolism and nutrition disorders14/20
DiarrheaGastrointestinal disorders12/20
Aspartate aminotransferase increasedInvestigations11/20
AnemiaBlood and lymphatic system disorders10/20
Alanine aminotransferase increasedInvestigations10/20
AlopeciaSkin and subcutaneous tissue disorders8/20
ConstipationGastrointestinal disorders7/20
HyperglycemiaMetabolism and nutrition disorders7/20
HypophosphatemiaMetabolism and nutrition disorders7/20
EpistaxisRespiratory, thoracic and mediastinal disorders6/20

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Treatment (Erdafitinib)
<=18 years17
Between 18 and 65 years3
>=65 years0
Age, Continuous
Age, Continuous(years)Treatment (Erdafitinib)
Mean13.8 ± 7.9
Sex: Female, Male
Sex: Female, Male(Participants)Treatment (Erdafitinib)
Female8
Male12
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Treatment (Erdafitinib)
Hispanic or Latino5
Not Hispanic or Latino14
Unknown or Not Reported1
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Treatment (Erdafitinib)
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American3
White13
More than one race0
Unknown or Not Reported4
Region of Enrollment
Region of Enrollment(participants)Treatment (Erdafitinib)
United States20
07

Study locations

116 sites
  • Children's Hospital of Alabama
    Birmingham, Alabama 35233, United States
  • Providence Alaska Medical Center
    Anchorage, Alaska 99508, United States
  • Banner Children's at Desert
    Mesa, Arizona 85202, United States
  • Banner University Medical Center - Tucson
    Tucson, Arizona 85719, United States
  • Arkansas Children's Hospital
    Little Rock, Arkansas 72202-3591, United States
  • Kaiser Permanente Downey Medical Center
    Downey, California 90242, United States
  • Loma Linda University Medical Center
    Loma Linda, California 92354, United States
  • Miller Children's and Women's Hospital Long Beach
    Long Beach, California 90806, United States
  • Children's Hospital Los Angeles
    Los Angeles, California 90027, United States
  • Cedars-Sinai Medical Center
    Los Angeles, California 90048, United States
  • Valley Children's Hospital
    Madera, California 93636, United States
  • UCSF Benioff Children's Hospital Oakland
    Oakland, California 94609, United States
  • Kaiser Permanente-Oakland
    Oakland, California 94611, United States
  • University of California Davis Comprehensive Cancer Center
    Sacramento, California 95817, United States
  • UCSF Medical Center-Mission Bay
    San Francisco, California 94158, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
    Denver, Colorado 80218, United States
  • Yale University
    New Haven, Connecticut 06520, United States
  • Alfred I duPont Hospital for Children
    Wilmington, Delaware 19803, United States
  • Children's National Medical Center
    Washington D.C., District of Columbia 20010, United States
  • UF Health Cancer Institute - Gainesville
    Gainesville, Florida 32610, United States
  • Memorial Regional Hospital/Joe DiMaggio Children's Hospital
    Hollywood, Florida 33021, United States
  • Nemours Children's Clinic-Jacksonville
    Jacksonville, Florida 32207, United States
  • University of Miami Miller School of Medicine-Sylvester Cancer Center
    Miami, Florida 33136, United States
  • Nicklaus Children's Hospital
    Miami, Florida 33155, United States
  • AdventHealth Orlando
    Orlando, Florida 32803, United States
  • Arnold Palmer Hospital for Children
    Orlando, Florida 32806, United States
  • Nemours Children's Hospital
    Orlando, Florida 32827, United States
  • Johns Hopkins All Children's Hospital
    St. Petersburg, Florida 33701, United States
  • Saint Joseph's Hospital/Children's Hospital-Tampa
    Tampa, Florida 33607, United States
  • Saint Mary's Medical Center
    West Palm Beach, Florida 33407, United States
  • Children's Healthcare of Atlanta - Arthur M Blank Hospital
    Atlanta, Georgia 30329, United States
  • Saint Luke's Cancer Institute - Boise
    Boise, Idaho 83712, United States
  • Lurie Children's Hospital-Chicago
    Chicago, Illinois 60611, United States
  • University of Chicago Comprehensive Cancer Center
    Chicago, Illinois 60637, United States
  • OSF Children's Hospital of Illinois
    Peoria, Illinois 61637, United States
  • Southern Illinois University School of Medicine
    Springfield, Illinois 62702, United States
  • Riley Hospital for Children
    Indianapolis, Indiana 46202, United States
  • Ascension Saint Vincent Indianapolis Hospital
    Indianapolis, Indiana 46260, United States
  • Blank Children's Hospital
    Des Moines, Iowa 50309, United States
  • University of Iowa/Holden Comprehensive Cancer Center
    Iowa City, Iowa 52242, United States
  • University of Kentucky/Markey Cancer Center
    Lexington, Kentucky 40536, United States
  • Norton Children's Hospital
    Louisville, Kentucky 40202, United States
  • Children's Hospital New Orleans
    New Orleans, Louisiana 70118, United States
  • Ochsner Medical Center Jefferson
    New Orleans, Louisiana 70121, United States
  • Eastern Maine Medical Center
    Bangor, Maine 04401, United States
  • Sinai Hospital of Baltimore
    Baltimore, Maryland 21215, United States
  • Johns Hopkins University/Sidney Kimmel Cancer Center
    Baltimore, Maryland 21287, United States
  • C S Mott Children's Hospital
    Ann Arbor, Michigan 48109, United States
  • Michigan State University
    East Lansing, Michigan 48823, United States
  • Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
    Grand Rapids, Michigan 49503, United States
  • Bronson Methodist Hospital
    Kalamazoo, Michigan 49007, United States
  • Children's Hospitals and Clinics of Minnesota - Minneapolis
    Minneapolis, Minnesota 55404, United States
  • University of Minnesota/Masonic Cancer Center
    Minneapolis, Minnesota 55455, United States
  • University of Mississippi Medical Center
    Jackson, Mississippi 39216, United States
  • Children's Mercy Hospitals and Clinics
    Kansas City, Missouri 64108, United States
  • Cardinal Glennon Children's Medical Center
    St Louis, Missouri 63104, United States
  • Washington University School of Medicine
    St Louis, Missouri 63110, United States
  • Mercy Hospital Saint Louis
    St Louis, Missouri 63141, United States
  • Children's Hospital and Medical Center of Omaha
    Omaha, Nebraska 68114, United States
  • University of Nebraska Medical Center
    Omaha, Nebraska 68198, United States
  • Hackensack University Medical Center
    Hackensack, New Jersey 07601, United States
  • Morristown Medical Center
    Morristown, New Jersey 07960, United States
  • Saint Peter's University Hospital
    New Brunswick, New Jersey 08901, United States
  • Albany Medical Center
    Albany, New York 12208, United States
  • Roswell Park Cancer Institute
    Buffalo, New York 14263, United States
  • The Steven and Alexandra Cohen Children's Medical Center of New York
    New Hyde Park, New York 11040, United States
  • NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
    New York, New York 10032, United States
  • Memorial Sloan Kettering Cancer Center
    New York, New York 10065, United States
  • NYP/Weill Cornell Medical Center
    New York, New York 10065, United States
  • University of Rochester
    Rochester, New York 14642, United States
  • State University of New York Upstate Medical University
    Syracuse, New York 13210, United States
  • Montefiore Medical Center - Moses Campus
    The Bronx, New York 10467, United States
  • Mission Hospital
    Asheville, North Carolina 28801, United States
  • Carolinas Medical Center/Levine Cancer Institute
    Charlotte, North Carolina 28203, United States
  • Duke University Medical Center
    Durham, North Carolina 27710, United States
  • Sanford Broadway Medical Center
    Fargo, North Dakota 58122, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Rainbow Babies and Childrens Hospital
    Cleveland, Ohio 44106, United States
  • Nationwide Children's Hospital
    Columbus, Ohio 43205, United States
  • Dayton Children's Hospital
    Dayton, Ohio 45404, United States
  • ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
    Toledo, Ohio 43606, United States
  • University of Oklahoma Health Sciences Center
    Oklahoma City, Oklahoma 73104, United States
  • Legacy Emanuel Children's Hospital
    Portland, Oregon 97227, United States
  • Oregon Health and Science University
    Portland, Oregon 97239, United States
  • Geisinger Medical Center
    Danville, Pennsylvania 17822, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • Children's Hospital of Pittsburgh of UPMC
    Pittsburgh, Pennsylvania 15224, United States
  • Prisma Health Richland Hospital
    Columbia, South Carolina 29203, United States
  • BI-LO Charities Children's Cancer Center
    Greenville, South Carolina 29605, United States
  • Sanford USD Medical Center - Sioux Falls
    Sioux Falls, South Dakota 57117-5134, United States
  • East Tennessee Childrens Hospital
    Knoxville, Tennessee 37916, United States
  • Saint Jude Children's Research Hospital
    Memphis, Tennessee 38105, United States
  • The Children's Hospital at TriStar Centennial
    Nashville, Tennessee 37203, United States
  • Vanderbilt University/Ingram Cancer Center
    Nashville, Tennessee 37232, United States
  • Dell Children's Medical Center of Central Texas
    Austin, Texas 78723, United States
  • Medical City Dallas Hospital
    Dallas, Texas 75230, United States
  • UT Southwestern/Simmons Cancer Center-Dallas
    Dallas, Texas 75390, United States
  • Cook Children's Medical Center
    Fort Worth, Texas 76104, United States
  • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
    Houston, Texas 77030, United States

Showing the first 100 of 116 sites across 2 countries.

08

References and documents

Publications

  • Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14. PubMed 31401903 ↗

Study documents

  • Protocol and statistical analysis plan · Oct 28, 2022

Documents are hosted by the registry — open the source record to download them.

09

Registry details

Key details

Study ID
NCT03210714
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Jul 7, 2017
Start date
Jun 5, 2018
Primary completion
Sep 30, 2023
Completion
Dec 30, 2026 (estimated)
Results posted
Sep 19, 2024
Last update
Sep 22, 2026

Study contacts

Alice Lee
principal investigator · Children's Oncology Group

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Start the discussion