Testing the Anti-cancer Drug Erdafitinib for Brain Cancers That Have Returned or Progressed Following Treatment

Recruiting now · Phase 2

Conditions studied: Recurrent Glioma, Recurrent WHO Grade 2 Glioma, Recurrent WHO Grade 3 Glioma, Recurrent WHO Grade 4 Glioma

In brief

This phase II trial tests how well erdafitinib works in controlling IDH-wild type (WT), FGFR-TACC gene fusion positive gliomas that have come back after a period of improvement (recurrent) or that are growing, spreading, or getting worse (progressive). Erdafitinib is in a class of medications called kinase inhibitors. It works by blocking the action of an abnormal FGFR protein that signals tumor cells to multiply. This may help keep tumor cells from growing and may kill them. Giving erdafitinib may help to slow the growth of, or to shrink, tumor cells in patients with recurrent or progressive IDH-wild type gliomas with FGFR-TACC gene fusion.

Key facts

Study ID: NCT05859334
Run by: National Cancer Institute (NCI)
People needed: 30
Starts: 2024-01-04
Expected to finish: 2026-10-23
Last updated by the study team: 2026-06-17

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Patient must be >= 18 years of age
Patient must have histologically confirmed IDH-WT gliomas (grade 2-4) as per World Health Organization (WHO) 2016 or 2021 classification
Tumor tissue should be positive for FGFR-TACC gene fusion as per any local next generation sequencing (NGS) (Clinical Laboratory Improvement Act [CLIA]-approved) assay described in background section
The disease should be recurrent or progressive glioma after initial anti-tumor treatment with at least 1 line of treatment including surgical resection, radiation therapy and/or chemotherapy
For patients with WHO grade 3 or 4 glioma and progressive disease < 12 weeks after completion of chemoradiotherapy, progression can be defined by the following set of criteria:
New enhancement outside of the radiation field (beyond the high-dose region or 80% isodose line)
If there is unequivocal evidence of viable tumor on histopathologic sampling (e.g., solid tumor areas. i.e., > 70% tumor cell nuclei in areas), high or progressive increase in Ki-67 proliferation index compared with prior biopsy, or evidence for histologic progression or increased anaplasia in tumor)
For patients with WHO grade 3 or 4 glioma and progressive disease >= 12 weeks after completion of chemoradiotherapy, progression can be defined by the following set of criteria:
New contrast-enhancing lesion outside of radiation field on decreasing, stable, or increasing doses of corticosteroids
Increase by >= 25% in the sum of the products of perpendicular diameters between the first post-radiotherapy scan, or a subsequent scan with smaller tumor size, and the scan at 12 weeks or later on stable or increasing doses of corticosteroids
For patients receiving antiangiogenic therapy, significant increase in T2/fluid attenuated inversion recovery (FLAIR) non-enhancing lesion may also be considered progressive disease. The increased T2/FLAIR must have occurred with the patient on stable or increasing doses of corticosteroids compared with baseline scan or best response after initiation of therapy and not be a result of comorbid events (e.g., effects of radiation therapy, demyelination, ischemic injury, infection, seizures, postoperative changes, or other treatment effects)
For patients with WHO grade 2 glioma progression is defined by any one of the following:
Development of new lesions or increase of enhancement (radiological evidence of malignant transformation)
A 25% increase of the T2 or FLAIR non-enhancing lesion on stable or increasing doses of corticosteroids compared with baseline scan or best response after initiation of therapy, not attributable to radiation effect or to comorbid events
There must be measurable disease (enhancing or non-enhancing as per Response Assessment in Neuro-Oncology [RANO] or RANO-low-grade glioma [LGG] criteria), as evaluated on pre-treatment MRI
Patient understands the procedures and investigational nature of the study drug and agrees to comply with study requirements by providing written informed consent
Patient must have Eastern Cooperative Oncology Group (ECOG) performance status =< 2 (or Karnofsky >= 60%)
Absolute neutrophil count >= 1000/uL
Hemoglobin > 8 g/dL (Patients are allowed to be transfused to this level)
Platelets >= 100 x 10\^9/L
Serum total bilirubin =< 1.5 x upper limit of normal (ULN), unless considered due to Gilbert's disease or disease involvement following approval by the medical monitor
Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase [SGPT]) =< 3 x institutional ULN
Creatinine clearance > 30 mL/min (patients with mild or moderate renal impairment) based on the Cockroft-Gault glomerular filtration rate (GFR) estimation
Patient must have normal serum phosphate level as per local laboratory parameters. (Medical management allowed)
Patient must be recovered from any clinically relevant toxic effects of any prior surgery, radiotherapy, or chemotherapy treatment with temozolomide

You may not qualify if…

Patients who are receiving any other investigational agents
History of allergic reactions attributed to compounds of similar chemical or biologic composition to erdafitinib
Patients requiring any medications or substances that are moderate CYP2C9 inducers and strong CYP3A4 inducers are ineligible. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment/informed consent procedures, the patient will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the patient is considering a new over-the-counter medicine or herbal product
Patients with uncontrolled intercurrent illness
Pregnant women are excluded from this study because erdafitinib is an FGFR inhibitor agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with erdafitinib, breastfeeding should be discontinued if the mother is treated with erdafitinib
Current central serous retinopathy (CSR) or retinal pigment epithelial detachment of any grade
Corrected QT interval (QTc) prolongation as confirmed by electrocardiography (ECG) at screening (Fridericia; QTc > 480 milliseconds)
Patients who have previously received FGFR inhibitors

Where it is running

University of Wisconsin Carbone Cancer Center - University Hospital — Madison, Wisconsin, United States (enrolling)
UCHealth University of Colorado Hospital — Aurora, Colorado, United States (enrolling)
UM Sylvester Comprehensive Cancer Center at Coral Gables — Coral Gables, Florida, United States (enrolling)
UM Sylvester Comprehensive Cancer Center at Deerfield Beach — Deerfield Beach, Florida, United States (enrolling)
University of Miami Miller School of Medicine-Sylvester Cancer Center — Miami, Florida, United States (enrolling)
Memorial Hospital East — Shiloh, Illinois, United States (enrolling)
Siteman Cancer Center at Saint Peters Hospital — City of Saint Peters, Missouri, United States (enrolling)
Siteman Cancer Center at West County Hospital — Creve Coeur, Missouri, United States (enrolling)
Washington University School of Medicine — St Louis, Missouri, United States (enrolling)
Siteman Cancer Center-South County — St Louis, Missouri, United States (enrolling)
Siteman Cancer Center at Christian Hospital — St Louis, Missouri, United States (enrolling)
Memorial Sloan Kettering Basking Ridge — Basking Ridge, New Jersey, United States (enrolling)
Hackensack University Medical Center — Hackensack, New Jersey, United States (enrolling)
Memorial Sloan Kettering Monmouth — Middletown, New Jersey, United States (enrolling)
Memorial Sloan Kettering Bergen — Montvale, New Jersey, United States (enrolling)
Memorial Sloan Kettering Commack — Commack, New York, United States (enrolling)
Memorial Sloan Kettering Westchester — Harrison, New York, United States (enrolling)
Laura and Isaac Perlmutter Cancer Center at NYU Langone — New York, New York, United States (enrolling)
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center — New York, New York, United States (enrolling)
Memorial Sloan Kettering Cancer Center — New York, New York, United States (enrolling)
Memorial Sloan Kettering Nassau — Uniondale, New York, United States (enrolling)
Wake Forest University Health Sciences — Winston-Salem, North Carolina, United States (enrolling)
University of Wisconsin Carbone Cancer Center - Eastpark Medical Center — Madison, Wisconsin, United States (enrolling)
UT MD Anderson Cancer Center — Houston, Texas, United States (enrolling)
Huntsman Cancer Institute/University of Utah — Salt Lake City, Utah, United States (enrolling)

Full record on ClinicalTrials.gov

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