Erdafitinib for Brain Cancer
Recruiting in Palo Alto (17 mi)
+26 other locations
Overseen byMacarena I De La Fuente
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: National Cancer Institute (NCI)
Must not be taking: CYP2C9 inducers, CYP3A4 inducers
Disqualifiers: Pregnancy, Uncontrolled illness, FGFR inhibitors, others
No Placebo Group
Prior Safety Data
Breakthrough Therapy
Approved in 2 Jurisdictions
Trial Summary
What is the purpose of this trial?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.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop your current medications, but you cannot take medications that are moderate CYP2C9 inducers or strong CYP3A4 inducers. It's important to discuss your current medications with the trial team to ensure there are no interactions with erdafitinib.
What data supports the effectiveness of the drug Erdafitinib (Balversa) for brain cancer?
Erdafitinib is approved for treating certain types of bladder cancer and is being studied for other cancers, but there is no direct evidence of its effectiveness for brain cancer. However, similar drugs like gefitinib have shown some benefits in treating brain metastases in lung cancer patients, suggesting potential for further research.
12345Is erdafitinib safe for humans?
Erdafitinib, also known as Balversa, has been approved for treating certain types of bladder cancer and is being studied for other cancers. Some safety concerns include the potential for eye problems, as a case of secondary maculopathy (eye disease) was reported in a patient using erdafitinib.
12678What makes the drug erdafitinib unique for treating brain cancer?
Erdafitinib is unique because it is a pan-fibroblast growth factor receptor (FGFR) inhibitor, which is different from other treatments like erlotinib and gefitinib that target the epidermal growth factor receptor (EGFR). This distinct mechanism of action may offer a new approach for treating brain cancer, especially if FGFR is involved in the cancer's growth.
124910Eligibility Criteria
Adults with specific brain cancers called IDH-wild type gliomas that have returned or worsened, despite previous treatments. Participants must be over 18 and have tumors with a particular gene fusion (FGFR-TACC). The cancer should show growth on scans and not be due to other conditions like radiation effects or infections.Inclusion Criteria
I can understand and am willing to sign the consent form myself or have someone legally authorized to do so on my behalf.
I am HIV positive, on treatment, and my viral load is undetectable.
My high-grade brain tumor has worsened 12 weeks after finishing treatment.
+15 more
Exclusion Criteria
Pregnant women are excluded from this study
Patients with uncontrolled intercurrent illness
History of allergic reactions attributed to compounds of similar chemical or biologic composition to erdafitinib
+5 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive erdafitinib orally once daily on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Up to 2 years
Monthly visits for MRI, OCT, and blood sample collection
Follow-up
Participants are monitored for safety and effectiveness after treatment completion
2 years
Every 3 months
Participant Groups
The trial is testing Erdafitinib, a drug designed to block abnormal proteins in tumor cells which may slow down or shrink the growth of recurrent or progressive brain tumors. It includes imaging tests like MRI and collecting tissue samples to study the drug's effect.
1Treatment groups
Experimental Treatment
Group I: Treatment (erdafitinib)Experimental Treatment4 Interventions
Patients 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.
Erdafitinib is already approved in United States, European Union for the following indications:
🇺🇸 Approved in United States as Balversa for:
- Locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations
🇪🇺 Approved in European Union as Balversa for:
- Locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Memorial Sloan Kettering CommackCommack, NY
Memorial Sloan Kettering Cancer CenterNew York, NY
Memorial Sloan Kettering NassauUniondale, NY
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer CenterNew York, NY
More Trial Locations
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Who Is Running the Clinical Trial?
National Cancer Institute (NCI)Lead Sponsor
References
A translational model-based approach to inform the choice of the dose in phase 1 oncology trials: the case study of erdafitinib. [2022]Erdafitinib (JNJ-42756493, BALVERSA) is a tyrosine kinase inhibitor indicated for the treatment of advanced urothelial carcinoma. In this work, a translational model-based approach to inform the choice of the doses in phase 1 trials is illustrated.
Erdafitinib: First Global Approval. [2020]Erdafitinib (Balversa™, Janssen Pharmaceutical Companies) is a pan-fibroblast growth factor receptor (FGFR) inhibitor that was recently approved in the USA for the treatment of locally advanced or metastatic FGFR3 or FGFR2 urothelial carcinoma. The drug is also being investigated as a treatment for other cancers including cholangiocarcinoma, liver cancer, non-small cell lung cancer, prostate cancer, lymphoma and oesophageal cancer. This article summarizes the milestones in the development of erdafitinib leading to this first approval for the treatment of urothelial carcinoma.
Central Nervous System Outcomes of Lazertinib Versus Gefitinib in EGFR-Mutated Advanced NSCLC: A LASER301 Subset Analysis. [2023]Lazertinib, a third-generation mutant-selective EGFR tyrosine kinase inhibitor, improved progression-free survival compared with gefitinib in the phase 3 LASER301 study (ClinicalTrials.gov Identifier: NCT04248829). Here, we report the efficacy of lazertinib and gefitinib in patients with baseline central nervous system (CNS) metastases.
Phase II trial of gefitinib in recurrent glioblastoma. [2022]To evaluate the efficacy and tolerability of gefitinib (ZD1839, Iressa; AstraZeneca, Wilmington, DE), a novel epidermal growth factor receptor tyrosine kinase inhibitor, in patients with recurrent glioblastoma.
Quality-of-life benefits and evidence of antitumour activity for patients with brain metastases treated with gefitinib. [2018]Brain metastases are a common complication of non-small-cell lung cancer (NSCLC). The role of chemotherapy in the treatment of brain metastases has not been clearly defined. Emerging case reports of patients with recurrent NSCLC treated as part of the Expanded Access Programme reveal that gefitinib ("Iressa", ZD1839) has clinical activity in some patients with brain metastases. Here, we describe a number of case studies documenting the response of patients with brain metastases to treatment with gefitinib. Many of these patients had quality-of-life benefits with improvement of neurological and systemic symptoms; some had a partial response of their brain metastases and even complete responses have been seen in a few patients. One case report also describes a durable long-term response with concurrent treatment with gefitinib and radiotherapy. Such results call for larger trials designed to evaluate and define the role of gefitinib in the treatment of brain metastases in NSCLC patients, either as a single agent or in combination with radiation therapy.
Erdafitinib-Induced Secondary Maculopathy. [2023]This work presents a case of secondary maculopathy associated with the use of erdafitinib (Balversa) for the management of bladder urothelial carcinoma with bony metastasis.
Erlotinib for the treatment of brain metastases in non-small cell lung cancer. [2018]Brain metastases (BM) are a common and lethal complication of non-small cell lung cancer (NSCLC) with up to 40% experiencing this complication. The use of erlotinib, a small molecule epidermal growth factor receptor (EGFR) inhibitor, holds promise in this somewhat refractory cohort of patients, and has become the subject of active clinical investigation.
Pharmacokinetics and safety of erlotinib and its metabolite OSI-420 in infants and children with primary brain tumors. [2023]Label="PURPOSE">Erlotinib (Tarceva®), a potent small molecule inhibitor of the epidermal growth factor receptor tyrosine kinase, has been evaluated to treat infants and children with primary brain tumors. The pharmacokinetics of erlotinib and its primary metabolite OSI-420 were characterized and exposure-safety associations were investigated.
The plasma and cerebrospinal fluid pharmacokinetics of erlotinib and its active metabolite (OSI-420) after intravenous administration of erlotinib in non-human primates. [2015]Erlotinib hydrochloride is a small molecule inhibitor of epidermal growth factor receptor (EGFR). EGFR is over-expressed in primary brain tumors and solid tumors that metastasize to the central nervous system. We evaluated the plasma and cerebrospinal fluid (CSF) pharmacokinetics of erlotinib and its active metabolite OSI-420 after an intravenous (IV) dose in a non-human primate model.
Erlotinib accumulation in brain metastases from non-small cell lung cancer: visualization by positron emission tomography in a patient harboring a mutation in the epidermal growth factor receptor. [2022]Drugs directed toward the epidermal growth factor receptor (EGFR), such as erlotinib (Tarceva®) and gefitinib (Iressa®), are used for the treatment of patients with advanced non-small cell lung cancer (NSCLC), including patients with brain metastases. However, whether erlotinib actually enters into brain metastases has not been adequately elucidated. In this study, we investigated the accumulation of [¹¹C]-erlotinib by positron emission tomography (PET) combined with computed tomography (CT) and magnetic resonance imaging (MRI).