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
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.
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.
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:
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
Stem cell infusions (with or without total body irradiation [TBI]):
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
For patients with solid tumors without known bone marrow involvement:
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):
Exclusion Criteria:
Concomitant medications
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
Undergo blood sample collection
Also known as: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
Undergo a bone marrow aspiration and/or biopsy
Undergo a bone scan
Also known as: Bone Scintigraphy
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
Correlative studies
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
Correlative studies
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
Undergo radionuclide imaging
Also known as: Gamma Scan, NM, Nuclear Medicine, nuclear medicine scan, radioimaging, Radionuclide Scanning, Scan, Scintigraphy
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
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
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
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
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
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
| Milestone | Treatment (Erdafitinib) |
|---|---|
| Started | 20 |
| Completed | 0 |
| Not completed | 20 |
| Withdrew: Adverse event | 6 |
| Withdrew: Physician decision | 1 |
| Withdrew: Progressive disease | 11 |
| Withdrew: Refusal by patient/parent/guardian | 2 |
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).
| percentage of patients | Treatment (Erdafitinib) |
|---|---|
| Objective Response Rate | 10 (3.3 to 26.2) |
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
Results for this outcome have not been posted.
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
Results for this outcome have not been posted.
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).
Results for this outcome have not been posted.
A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.
Results for this outcome have not been posted.
Collected over Up to 2 years from study entry.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment (Erdafitinib) | 8/20 (40%) | 10/20 (50%) | 19/20 (95%) |
| Event | Treatment (Erdafitinib) |
|---|---|
| Infections and infestations - Other, specifyInfections and infestations | 2/20 |
| Vascular disorders - Other, specifyVascular disorders | 2/20 |
| EnterocolitisGastrointestinal disorders | 1/20 |
| Disease progressionGeneral disorders and administration site conditions | 1/20 |
| General disorders and administration site conditions - Other, specifyGeneral disorders and administration site conditions | 1/20 |
| PainGeneral disorders and administration site conditions | 1/20 |
| Urinary tract infectionInfections and infestations | 1/20 |
| Alkaline phosphatase increasedInvestigations | 1/20 |
| AnorexiaMetabolism and nutrition disorders | 1/20 |
| HyperphosphatemiaMetabolism and nutrition disorders | 1/20 |
| Event | Treatment (Erdafitinib) |
|---|---|
| HyperphosphatemiaMetabolism and nutrition disorders | 14/20 |
| DiarrheaGastrointestinal disorders | 12/20 |
| Aspartate aminotransferase increasedInvestigations | 11/20 |
| AnemiaBlood and lymphatic system disorders | 10/20 |
| Alanine aminotransferase increasedInvestigations | 10/20 |
| AlopeciaSkin and subcutaneous tissue disorders | 8/20 |
| ConstipationGastrointestinal disorders | 7/20 |
| HyperglycemiaMetabolism and nutrition disorders | 7/20 |
| HypophosphatemiaMetabolism and nutrition disorders | 7/20 |
| EpistaxisRespiratory, thoracic and mediastinal disorders | 6/20 |
| Age, Categorical(Participants) | Treatment (Erdafitinib) |
|---|---|
| <=18 years | 17 |
| Between 18 and 65 years | 3 |
| >=65 years | 0 |
| Age, Continuous(years) | Treatment (Erdafitinib) |
|---|---|
| Mean | 13.8 ± 7.9 |
| Sex: Female, Male(Participants) | Treatment (Erdafitinib) |
|---|---|
| Female | 8 |
| Male | 12 |
| Ethnicity (NIH/OMB)(Participants) | Treatment (Erdafitinib) |
|---|---|
| Hispanic or Latino | 5 |
| Not Hispanic or Latino | 14 |
| Unknown or Not Reported | 1 |
| Race (NIH/OMB)(Participants) | Treatment (Erdafitinib) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 3 |
| White | 13 |
| More than one race | 0 |
| Unknown or Not Reported | 4 |
| Region of Enrollment(participants) | Treatment (Erdafitinib) |
|---|---|
| United States | 20 |
Showing the first 100 of 116 sites across 2 countries.
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Familial ependymoma
National Cancer Institute (NCI)