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Recurrent GliomaPhase 2NCT05859334

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

A Phase 2 study testing Biospecimen Collection, Erdafitinib and Magnetic Resonance Imaging in people with Recurrent Glioma and Recurrent WHO Grade 2 Glioma, run by National Cancer Institute (NCI) at 27 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Biospecimen Collection, Erdafitinib and Magnetic Resonance Imaging
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
27 sitesUnited States
Study dates
4 Jan 2024to 23 Oct 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
Show all 31Show fewer
  • 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
  • Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
  • For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
  • Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
  • Patients should be New York Heart Association Functional Classification class 2B or better
  • The effects of erdafitinib on the developing human fetus are unknown. For this reason and because FGFR inhibitors are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and one month after completion of erdafitinib administration. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and one month after completion of erdafitinib administration
  • Ability to understand and the willingness to sign a written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants

Who cannot join

  • 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

Trial locations and contacts

27 sites in 1 country.

27 sites in 1 country

United States27 sites

  • UCHealth University of Colorado Hospital

    Aurora, Colorado, 80045

  • Memorial Sloan Kettering Basking Ridge

    Basking Ridge, New Jersey, 07920

  • Siteman Cancer Center at Saint Peters Hospital

    City of Saint Peters, Missouri, 63376

  • Memorial Sloan Kettering Commack

    Commack, New York, 11725

  • UM Sylvester Comprehensive Cancer Center at Coral Gables

    Coral Gables, Florida, 33146

  • Siteman Cancer Center at West County Hospital

    Creve Coeur, Missouri, 63141

  • UT Southwestern/Simmons Cancer Center-Dallas

    Dallas, Texas, 75390

  • UM Sylvester Comprehensive Cancer Center at Deerfield Beach

    Deerfield Beach, Florida, 33442

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Memorial Sloan Kettering Westchester

    Harrison, New York, 10604

  • UT MD Anderson Cancer Center

    Houston, Texas, 77030

  • University of Wisconsin Carbone Cancer Center - Eastpark Medical Center

    Madison, Wisconsin, 53718

  • University of Wisconsin Carbone Cancer Center - University Hospital

    Madison, Wisconsin, 53792

  • University of Miami Miller School of Medicine-Sylvester Cancer Center

    Miami, Florida, 33136

  • Memorial Sloan Kettering Monmouth

    Middletown, New Jersey, 07748

  • Memorial Sloan Kettering Bergen

    Montvale, New Jersey, 07645

  • Laura and Isaac Perlmutter Cancer Center at NYU Langone

    New York, New York, 10016

  • NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

    New York, New York, 10032

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104

  • Huntsman Cancer Institute/University of Utah

    Salt Lake City, Utah, 84112

  • Memorial Hospital East

    Shiloh, Illinois, 62269

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • Siteman Cancer Center-South County

    St Louis, Missouri, 63129

  • Siteman Cancer Center at Christian Hospital

    St Louis, Missouri, 63136

  • Memorial Sloan Kettering Nassau

    Uniondale, New York, 11553

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157

Other trials in United States

About this study

Official title: A Phase 2 Study of Erdafitinib in Patients With Recurrent or Progressive IDH-Wild Type Glioma With an FGFR-TACC Gene Fusion

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.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To assess the preliminary anti-tumor activity of erdafitinib in patients with recurrent or progressive IDH-WT glioma with FGFR-TACC gene fusion as measured by the best response at any time during treatment in terms of objective response rate (ORR).

SECONDARY OBJECTIVES:

  1. To determine the safety and tolerability of erdafitinib in patients with recurrent or progressive IDH-WT glioma with FGFR-TACC gene fusion.

II. To assess the overall survival (OS) of erdafitinib in patients with recurrent or progressive IDH-WT glioma with FGFR-TACC gene fusion.

III. To assess the progression free survival (PFS) at 6 months of patient with IDH-WT glioma with FGFR-TACC gene fusion treated with erdafitinib.

OUTLINE: This is a dose-escalation study of erdafitinib followed by a dose-expansion study.

Patients receive erdafitinib orally (PO) once daily (QD) on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo magnetic resonance imaging (MRI), optical coherence tomography (OCT), and collection of blood samples throughout the trial. Additionally, patients may optionally undergo collection of tissue samples on study.

After study completion, patients are followed up every 3 months for 2 years.

Being tested
Biospecimen Collection, Erdafitinib, Magnetic Resonance Imaging, Optical Coherence Tomography
Medical subject headings
Glioma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

    Undergo collection of blood and/or tissue samples

  • ErdafitinibDrug

    Also called Balversa, JNJ 42756493, JNJ-42756493, JNJ42756493

    Given PO

  • Magnetic Resonance ImagingProcedure or surgery

    Also called Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan

    Undergo MRI

  • Optical Coherence TomographyProcedure or surgery

    Also called OCT, Optical Coherence Tomography (OCT)

    Undergo OCT

Study groups in NCT05859334
Study groupRoleWhat they receive
Treatment (erdafitinib)ExperimentalPatients receive erdafitinib PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI, OCT, and collection of blood samples throughout the trial. Additionally, patients may optionally undergo collection of tissue samples on study.
Receives: Procedure: Biospecimen Collection; Drug: Erdafitinib; Procedure: Magnetic Resonance Imaging; Procedure: Optical Coherence Tomography

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Objective response rate

    Defined as the proportion of patients with either complete response or partial response as per Response Assessment in Neuro-Oncology (RANO) or RANO-low-grade glioma criteria. The response rate will be estimated along with the corresponding 95% confidence interval using the binomial exact method. Univariable and multivariable logistic regression models will be used to estimate the associations between study covariates and response status. Odds ratios and corresponding 95% confidence intervals and p-values will be reported.

    Measured: Up to 2 years

Secondary outcomes (4)

  1. Incidence of dose-limiting toxicities

    Analyzed based on National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

    Measured: Up to 28 days

  2. Incidence of adverse events (AEs) and serious AEs

    Analyzed based on NCI CTCAE version 5.0.

    Measured: Up to 2 years

  3. Progression-free survival

    Kaplan-Meier method will be used to estimate survival rates for pre-specified time points along with 95% confidence intervals. Hazard ratios (HRs) will be estimated using Cox proportional hazards regression model to examine the associations of study covariates with time-to-event outcomes along with corresponding 95% confidence intervals and p-values if these are estimable.

    Measured: Time between date of enrollment and date of progression or date of death from any cause, assessed at 6 months

  4. Overall survival

    Kaplan-Meier method will be used to estimate survival rates for pre-specified time points along with 95% confidence intervals. HRs will be estimated using Cox proportional hazards regression model to examine the associations of study covariates with time-to-event outcomes along with corresponding 95% confidence intervals and p-values if these are estimable.

    Measured: Time between the date of enrollment and date of death from any cause, censored at the last date of follow-up, assessed up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 May 2023
  2. Study start4 Jan 2024
  3. Primary completion23 Oct 2027 (estimated)
  4. Study completion23 Oct 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05859334
Sponsor's protocol ID
NCI-2023-03776
Other IDs
NCI-2023-03776, 10559, 10559, UM1CA186644
Status last verified
Oct 2026
First submitted
13 May 2023
Last updated
15 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.

Publications

  • Alshiehk Nasany R, de la Fuente MI. Molecularly driven therapies in the treatment of primary brain tumors. Adv Cancer Res. 2025;166:183-201. doi: 10.1016/bs.acr.2025.05.005. Epub 2025 May 27.

    PubMed 40675687

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Biospecimen Collection, Erdafitinib and Magnetic Resonance Imaging in people with Recurrent Glioma and Recurrent WHO Grade 2 Glioma, run by National Cancer Institute (NCI) at 27 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 31 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 27 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Objective response rate (time frame: Up to 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05859334, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2026.

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