ClinicalTrials.gg
Active, not recruitingNCT03210714Updated Oct 16, 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 2026-10-16.

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

Updated Oct 15, 2026Posted results revisedGo to Updates ↓
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

In context

Medulloblastoma

317 studies on the registry are indexed under Medulloblastoma; 51 are open to participants now.

This study's enrollment of 20 is below the median of 31 across 255 interventional studies indexed under Medulloblastoma.

Browse Medulloblastoma studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.

Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

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

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 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
      • lesions that do not meet the measurement requirements for (RECIST) 1.1
  • >/= 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 or [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
    • : 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 (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)
    • >/= 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 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 to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
  • or radioisotope (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
  • (sum of conjugated + unconjugated) =/\< 1.5 x (ULN) for age (performed within 7 days prior to enrollment)
  • Serum glutamate pyruvate transaminase () (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)
  • (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 ; 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 may not participate unless they have agreed to use an effective method, while receiving study treatment and for 3 months after the last dose of erdafitinib; male subjects (with a partner of ) 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

    • : 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
    • 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- 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
    • 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 , 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 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
Terms in these criteria, in plain language(24)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Measurable disease

The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.

The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.

Leptomeningeal disease

Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.

Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.

RECIST

The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.

“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.

Karnofsky performance status (KPS)

A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.

Studies often ask for 60 or 70 and above. Your doctor gives the score.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

GVHD (graft-versus-host disease)

After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Metastases

Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

QT and QTc

On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.

The study may name a correction formula and a limit in milliseconds, often around 450–480.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Reproductive potential

Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.

Criteria about it usually ask for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

CYP3A (CYP3A4) inhibitors and inducers

CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.

Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.

TIA (transient ischaemic attack)

A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.

Stroke symptoms need emergency care at once, even if they go away.

05

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

  • 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

06

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

07

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 disorders1/20
General disorders and administration site conditions - Other, specifyGeneral disorders1/20
PainGeneral disorders1/20
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
08

Study locations

116 sites

Showing the first 100 of 116 sites across 2 countries. Choose a country and city above to see the rest.

09

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

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

2 registry updates since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Jun 2018 to primary completion in Sep 2023, 63 months.

Results were first posted in Sep 2024, 11 months after primary completion.

Of 38 non-industry phase 2 interventional studies under Medulloblastoma, half reached primary completion within 68 months of starting (the median), counting those still running; a quarter within 52 and three quarters within 114. This study's 63 months is close to that.

Counting every sponsor, half of 42 studies reached it within 66 months; a quarter within 51 and three quarters within 92.

How these figures are worked out, and which studies are left out of them.

Registered
Jul 6, 2017
First posted
Jul 7, 2017
StartActual
Jun 5, 2018
Primary completionActual
Sep 30, 2023
CompletionEst.
Dec 30, 2026
Results first posted
Sep 19, 2024
Last update
Oct 16, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Results
Posted results revised
revised Oct 15, 2026
Show all 2 updates
  1. Oct 16, 2026
    Minor edits only
    + 1 other change: verification date
  2. Oct 15, 2026
    Posted results revised
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

11

Registry details

Key details

Study ID
NCT03210714
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
Oct 16, 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.

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Discussion

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Studies of the drug tested here: Erdafitinib

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