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L'erdafitinib dans le traitement des patients atteints de tumeurs solides avancées récidivantes ou réfractaires, de lymphome non hodgkinien ou de troubles histiocytaires avec mutations FGFR (un essai de traitement MATCH pédiatrique)

15 octobre 2026 mis à jour par: National Cancer Institute (NCI)

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Sous-protocole de phase 2 de l'erdafitinib chez les patients présentant des altérations du FGFR1/2/3/4

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.

Aperçu de l'étude

Statut

Actif, ne recrute pas

Les conditions

Intervention / Traitement

Description détaillée

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.

Type d'étude

Interventionnel

Inscription (Réel)

20

Phase

  • Phase 2

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

  • Porto Rico
      • San Juan, Porto Rico, 00926
        • University Pediatric Hospital
      • San Juan, Porto Rico, 00912
        • San Jorge Children's Hospital
  • États-Unis
    • Alabama
      • Birmingham, Alabama, États-Unis, 35233
        • Children's Hospital of Alabama
    • Alaska
      • Anchorage, Alaska, États-Unis, 99508
        • Providence Alaska Medical Center
    • Arizona
      • Mesa, Arizona, États-Unis, 85202
        • Banner Children's at Desert
      • Tucson, Arizona, États-Unis, 85719
        • Banner University Medical Center - Tucson
    • Arkansas
      • Little Rock, Arkansas, États-Unis, 72202-3591
        • Arkansas Children's Hospital
    • California
      • Downey, California, États-Unis, 90242
        • Kaiser Permanente Downey Medical Center
      • Loma Linda, California, États-Unis, 92354
        • Loma Linda University Medical Center
      • Long Beach, California, États-Unis, 90806
        • Miller Children's and Women's Hospital Long Beach
      • Los Angeles, California, États-Unis, 90048
        • Cedars-Sinai Medical Center
      • Los Angeles, California, États-Unis, 90027
        • Children's Hospital Los Angeles
      • Madera, California, États-Unis, 93636
        • Valley Children's Hospital
      • Oakland, California, États-Unis, 94609
        • UCSF Benioff Children's Hospital Oakland
      • Oakland, California, États-Unis, 94611
        • Kaiser Permanente-Oakland
      • Sacramento, California, États-Unis, 95817
        • University of California Davis Comprehensive Cancer Center
      • San Francisco, California, États-Unis, 94158
        • UCSF Medical Center-Mission Bay
    • Colorado
      • Aurora, Colorado, États-Unis, 80045
        • Children's Hospital Colorado
      • Denver, Colorado, États-Unis, 80218
        • Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
    • Connecticut
      • New Haven, Connecticut, États-Unis, 06520
        • Yale University
    • Delaware
      • Wilmington, Delaware, États-Unis, 19803
        • Alfred I duPont Hospital for Children
    • District of Columbia
      • Washington D.C., District of Columbia, États-Unis, 20010
        • Children's National Medical Center
    • Florida
      • Gainesville, Florida, États-Unis, 32610
        • UF Health Cancer Institute - Gainesville
      • Hollywood, Florida, États-Unis, 33021
        • Memorial Regional Hospital/Joe DiMaggio Children's Hospital
      • Jacksonville, Florida, États-Unis, 32207
        • Nemours Children's Clinic-Jacksonville
      • Miami, Florida, États-Unis, 33155
        • Nicklaus Children's Hospital
      • Miami, Florida, États-Unis, 33136
        • University of Miami Miller School of Medicine-Sylvester Cancer Center
      • Orlando, Florida, États-Unis, 32827
        • Nemours Children's Hospital
      • Orlando, Florida, États-Unis, 32803
        • AdventHealth Orlando
      • Orlando, Florida, États-Unis, 32806
        • Arnold Palmer Hospital for Children
      • St. Petersburg, Florida, États-Unis, 33701
        • Johns Hopkins All Children's Hospital
      • Tampa, Florida, États-Unis, 33607
        • Saint Joseph's Hospital/Children's Hospital-Tampa
      • West Palm Beach, Florida, États-Unis, 33407
        • Saint Mary's Medical Center
    • Georgia
      • Atlanta, Georgia, États-Unis, 30329
        • Children's Healthcare of Atlanta - Arthur M Blank Hospital
    • Idaho
      • Boise, Idaho, États-Unis, 83712
        • Saint Luke's Cancer Institute - Boise
    • Illinois
      • Chicago, Illinois, États-Unis, 60637
        • University of Chicago Comprehensive Cancer Center
      • Chicago, Illinois, États-Unis, 60611
        • Lurie Children's Hospital-Chicago
      • Peoria, Illinois, États-Unis, 61637
        • OSF Children's Hospital of Illinois
      • Springfield, Illinois, États-Unis, 62702
        • Southern Illinois University School of Medicine
    • Indiana
      • Indianapolis, Indiana, États-Unis, 46202
        • Riley Hospital for Children
      • Indianapolis, Indiana, États-Unis, 46260
        • Ascension Saint Vincent Indianapolis Hospital
    • Iowa
      • Des Moines, Iowa, États-Unis, 50309
        • Blank Children's Hospital
      • Iowa City, Iowa, États-Unis, 52242
        • University of Iowa/Holden Comprehensive Cancer Center
    • Kentucky
      • Lexington, Kentucky, États-Unis, 40536
        • University of Kentucky/Markey Cancer Center
      • Louisville, Kentucky, États-Unis, 40202
        • Norton Children's Hospital
    • Louisiana
      • New Orleans, Louisiana, États-Unis, 70121
        • Ochsner Medical Center Jefferson
      • New Orleans, Louisiana, États-Unis, 70118
        • Children's Hospital New Orleans
    • Maine
      • Bangor, Maine, États-Unis, 04401
        • Eastern Maine Medical Center
    • Maryland
      • Baltimore, Maryland, États-Unis, 21287
        • Johns Hopkins University/Sidney Kimmel Cancer Center
      • Baltimore, Maryland, États-Unis, 21215
        • Sinai Hospital of Baltimore
    • Michigan
      • Ann Arbor, Michigan, États-Unis, 48109
        • C S Mott Children's Hospital
      • East Lansing, Michigan, États-Unis, 48823
        • Michigan State University
      • Grand Rapids, Michigan, États-Unis, 49503
        • Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
      • Kalamazoo, Michigan, États-Unis, 49007
        • Bronson Methodist Hospital
    • Minnesota
      • Minneapolis, Minnesota, États-Unis, 55404
        • Children's Hospitals and Clinics of Minnesota - Minneapolis
      • Minneapolis, Minnesota, États-Unis, 55455
        • University of Minnesota/Masonic Cancer Center
    • Mississippi
      • Jackson, Mississippi, États-Unis, 39216
        • University of Mississippi Medical Center
    • Missouri
      • Kansas City, Missouri, États-Unis, 64108
        • Children's Mercy Hospitals and Clinics
      • St Louis, Missouri, États-Unis, 63110
        • Washington University School of Medicine
      • St Louis, Missouri, États-Unis, 63141
        • Mercy Hospital Saint Louis
      • St Louis, Missouri, États-Unis, 63104
        • Cardinal Glennon Children's Medical Center
    • Nebraska
      • Omaha, Nebraska, États-Unis, 68198
        • University of Nebraska Medical Center
      • Omaha, Nebraska, États-Unis, 68114
        • Children's Hospital and Medical Center of Omaha
    • New Jersey
      • Hackensack, New Jersey, États-Unis, 07601
        • Hackensack University Medical Center
      • Morristown, New Jersey, États-Unis, 07960
        • Morristown Medical Center
      • New Brunswick, New Jersey, États-Unis, 08901
        • Saint Peter's University Hospital
    • New York
      • Albany, New York, États-Unis, 12208
        • Albany Medical Center
      • Buffalo, New York, États-Unis, 14263
        • Roswell Park Cancer Institute
      • New Hyde Park, New York, États-Unis, 11040
        • The Steven and Alexandra Cohen Children's Medical Center of New York
      • New York, New York, États-Unis, 10065
        • Memorial Sloan Kettering Cancer Center
      • New York, New York, États-Unis, 10032
        • NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
      • New York, New York, États-Unis, 10065
        • NYP/Weill Cornell Medical Center
      • Rochester, New York, États-Unis, 14642
        • University of Rochester
      • Syracuse, New York, États-Unis, 13210
        • State University of New York Upstate Medical University
      • The Bronx, New York, États-Unis, 10467
        • Montefiore Medical Center - Moses Campus
    • North Carolina
      • Asheville, North Carolina, États-Unis, 28801
        • Mission Hospital
      • Charlotte, North Carolina, États-Unis, 28203
        • Carolinas Medical Center/Levine Cancer Institute
      • Durham, North Carolina, États-Unis, 27710
        • Duke University Medical Center
    • North Dakota
      • Fargo, North Dakota, États-Unis, 58122
        • Sanford Broadway Medical Center
    • Ohio
      • Cincinnati, Ohio, États-Unis, 45229
        • Cincinnati Children's Hospital Medical Center
      • Cleveland, Ohio, États-Unis, 44106
        • Rainbow Babies and Childrens Hospital
      • Columbus, Ohio, États-Unis, 43205
        • Nationwide Children's Hospital
      • Dayton, Ohio, États-Unis, 45404
        • Dayton Children's Hospital
      • Toledo, Ohio, États-Unis, 43606
        • ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
    • Oklahoma
      • Oklahoma City, Oklahoma, États-Unis, 73104
        • University of Oklahoma Health Sciences Center
    • Oregon
      • Portland, Oregon, États-Unis, 97239
        • Oregon Health and Science University
      • Portland, Oregon, États-Unis, 97227
        • Legacy Emanuel Children's Hospital
    • Pennsylvania
      • Danville, Pennsylvania, États-Unis, 17822
        • Geisinger Medical Center
      • Philadelphia, Pennsylvania, États-Unis, 19104
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, États-Unis, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • South Carolina
      • Columbia, South Carolina, États-Unis, 29203
        • Prisma Health Richland Hospital
      • Greenville, South Carolina, États-Unis, 29605
        • BI-LO Charities Children's Cancer Center
    • South Dakota
      • Sioux Falls, South Dakota, États-Unis, 57117-5134
        • Sanford USD Medical Center - Sioux Falls
    • Tennessee
      • Knoxville, Tennessee, États-Unis, 37916
        • East Tennessee Childrens Hospital
      • Memphis, Tennessee, États-Unis, 38105
        • Saint Jude Children's Research Hospital
      • Nashville, Tennessee, États-Unis, 37232
        • Vanderbilt University/Ingram Cancer Center
      • Nashville, Tennessee, États-Unis, 37203
        • The Children's Hospital at TriStar Centennial
    • Texas
      • Austin, Texas, États-Unis, 78723
        • Dell Children's Medical Center of Central Texas
      • Dallas, Texas, États-Unis, 75390
        • UT Southwestern/Simmons Cancer Center-Dallas
      • Dallas, Texas, États-Unis, 75230
        • Medical City Dallas Hospital
      • Fort Worth, Texas, États-Unis, 76104
        • Cook Children's Medical Center
      • Houston, Texas, États-Unis, 77030
        • UT MD Anderson Cancer Center
      • Houston, Texas, États-Unis, 77030
        • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
      • San Antonio, Texas, États-Unis, 78207
        • Children's Hospital of San Antonio
      • San Antonio, Texas, États-Unis, 78229
        • Methodist Children's Hospital of South Texas
      • Temple, Texas, États-Unis, 76508
        • Scott and White Memorial Hospital
    • Utah
      • Salt Lake City, Utah, États-Unis, 84113
        • Primary Children's Hospital
    • Vermont
      • Burlington, Vermont, États-Unis, 05405
        • University of Vermont and State Agricultural College
    • Virginia
      • Norfolk, Virginia, États-Unis, 23507
        • Children's Hospital of The King's Daughters
      • Richmond, Virginia, États-Unis, 23298
        • VCU Massey Comprehensive Cancer Center
    • Washington
      • Seattle, Washington, États-Unis, 98105
        • Seattle Children's Hospital
      • Spokane, Washington, États-Unis, 99204
        • Providence Sacred Heart Medical Center and Children's Hospital
      • Tacoma, Washington, États-Unis, 98431
        • Madigan Army Medical Center
    • West Virginia
      • Morgantown, West Virginia, États-Unis, 26506
        • West Virginia University Healthcare
    • Wisconsin
      • Madison, Wisconsin, États-Unis, 53792
        • University of Wisconsin Carbone Cancer Center - University Hospital
      • Milwaukee, Wisconsin, États-Unis, 53226
        • Children's Hospital of Wisconsin

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

1 an à 21 ans (Enfant, Adulte)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

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:

  • 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

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: N / A
  • Modèle interventionnel: Affectation à un seul groupe
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: 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.
Autre: Laboratory Biomarker Analysis

Correlative studies

Procédure: Biospecimen Collection

Undergo blood sample collection

Autres noms:
  • Collecte d'échantillons biologiques
  • Spécimen biologique collecté
  • Collecte de spécimens
  • Collection d'échantillons
Procédure: Magnetic Resonance Imaging

Undergo MRI

Autres noms:
  • IRM
  • Résonance magnétique
  • Balayage d'imagerie par résonance magnétique
  • Imagerie Médicale, Résonance Magnétique / Résonance Magnétique Nucléaire
  • M
  • Imagerie IRM
  • Imagerie RMN
  • NMRI
  • Imagerie par résonance magnétique nucléaire
  • Imagerie par résonance magnétique (IRM)
  • IRMs
  • Imagerie par résonance magnétique (procédure)
  • IRM structurelle
Autre: Pharmacological Study

Correlative studies

Médicament: Erdafitinib

Given PO

Autres noms:
  • Balversa
  • JNJ-42756493
  • JNJ42756493
Procédure: Bone Marrow Aspiration and Biopsy

Undergo a bone marrow aspiration and/or biopsy

Procédure: Bone Scan

Undergo a bone scan

Autres noms:
  • Scintigraphie osseuse
Procédure: Computed Tomography

Undergo a CT scan

Autres noms:
  • TDM
  • CHAT
  • Scanner
  • Tomographie axiale informatisée
  • Tomographie assistée par ordinateur
  • Tomodensitométrie
  • tomographie
  • Tomographie axiale informatisée (procédure)
  • Tomodensitométrie (TDM)
  • Scan de chat diagnostique
  • Type de service de scan de chat diagnostique
Procédure: Positron Emission Tomography

Undergo a PET scan

Autres noms:
  • Imagerie médicale, Tomographie par émission de positrons
  • ANIMAUX
  • TEP-scan
  • Scan de tomographie par émission de positrons
  • Tomographie par émission de positrons
  • PT
  • Tomographie par émission de positrons (procédure)
Procédure: Radionuclide Imaging

Undergo radionuclide imaging

Autres noms:
  • NM
  • Médecine nucléaire
  • échographie de médecine nucléaire
  • radioimagerie
  • Balayage des radionucléides
  • Analyse
  • Scintigraphie
  • Gamma Scan
Procédure: X-Ray Imaging

Undergo an x-ray

Autres noms:
  • Radiographie conventionnelle
  • Radiologie diagnostique
  • Imagerie médicale, rayons X
  • Imagerie radiographique
  • Radiographie
  • GR
  • Radiographie statique
  • Radiographies sur film simple
  • Procédure d'imagerie radiographique (procédure)

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Objective Response Rate
Délai: Up to 2 years from study entry

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).
Up to 2 years from study entry

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Percentage of Patients Experiencing Grade 3 or Higher Adverse Events
Délai: Up to 2 years from study entry

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
Up to 2 years from study entry
Progression Free Survival (PFS)
Délai: Up to 6 months from study entry

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
Up to 6 months from study entry
Pharmacokinetic (PK) Parameters
Délai: 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

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).
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

Autres mesures de résultats

Mesure des résultats
Description de la mesure
Délai
Changes in Tumor Genomic Profile
Délai: Up to 3 years

A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.
Up to 3 years

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

National Cancer Institute (NCI)

Les enquêteurs

  • Chercheur principal: Alice Lee, Children's Oncology Group

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Publications générales

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

5 juin 2018

Achèvement primaire (Réel)

30 septembre 2023

Achèvement de l'étude (Estimé)

30 décembre 2026

Dates d'inscription aux études

Première soumission

6 juillet 2017

Première soumission répondant aux critères de contrôle qualité

6 juillet 2017

Première publication (Réel)

7 juillet 2017

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

16 octobre 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

15 octobre 2026

Dernière vérification

1 septembre 2026

Plus d'information

Termes liés à cette étude

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • NCI-2017-01159
  • U10CA180886 (Subvention/contrat des NIH des États-Unis)
  • UM1CA081457 (Subvention/contrat des NIH des États-Unis)
  • NCI-2017-01159 (Identificateur de registre: CTRP (Clinical Trial Reporting Program))
  • APEC1621B (Autre identifiant: CTEP)
  • APEC1621B (Autre identifiant: Children's Oncology Group)

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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