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New drug targets rare gene mutations in advanced cancers

NCT ID NCT04233567

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase II trial tested the drug infigratinib in 17 adults with advanced or metastatic solid tumors that have specific FGFR gene mutations. The drug works by blocking overactive FGFR proteins that help cancer grow. The study measured how many patients' tumors shrank or stabilized, and how long that lasted.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Infigratinib (a drug that blocks FGFR proteins to slow tumor growth)
What this could lead to
If successful, this could point toward a targeted treatment option for people with certain FGFR-mutated cancers that are hard to treat.
What could go wrong
This is a small, early-phase trial (17 people) with no control group, so results may not apply broadly. The drug may cause side effects or fail to shrink tumors in many patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

17 people

The number who actually took part.

Started

Jan 2020

Finished

Dec 2024

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients with histologically or cytologically confirmed advanced or metastatic solid tumors of any histologic classification at the time of diagnosis * Written documentation of local or central Clinical Laboratory Improvement Act (CLIA)-certified laboratory determination of FGFR gene fusions/translocations or activating mutations * The study is open to solid tumors in the following cohorts: * Cohort 1: Solid tumor patients with FGFR1-3 fusion/translocation (n=10) who have progressed on or are intolerant to standard of care (SOC) therapies and received treatment with a different FGFR inhibitor. Cholangiocarcinoma patients are permitted in this cohort * Cohort 2: Solid tumor patients with FGFR1-3 fusion/translocation (n, up to 30) who have progressed on or are intolerant to SOC therapies. Prior therapy with a different FGFR inhibitor is not permitted. Cholangiocarcinoma patients are excluded from this cohort (there are multiple competing studies and opportunities for patients to get treatment in other trials) * Cohort 3: Solid tumor patients with genetic alterations such as point mutations, insertions/deletions, or amplifications in any FGFR gene family member (n=10). Prior therapy with a different FGFR inhibitor is not permitted. Cholangiocarcinoma patients are permitted in this cohort * Evidence of measurable or evaluable disease according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 * Patients must have received at least one prior SOC regimen for advanced/metastatic cancer. Patient should have had evidence of progressive disease following their prior regimen, or if prior treatment was discontinued due to toxicity must have continued evidence of measurable or evaluable disease. Patients who have received prior treatment with an alternate FGFR inhibitor are still eligible for the study * Patients with primary central nervous system (CNS) cancer or CNS metastases are excluded (because it is unclear how much CNS penetration the drug has). However, asymptomatic patients with history of successfully treated CNS metastases with surgery or radiation and follow up imaging showing stability, can be eligible * Eastern Cooperative Oncology Group (ECOG) performance status =\< 1 (patients with ECOG performance status of 2 may be considered on a case-by-case basis after discussion with QED Therapeutics) * Able to read and/or understand the details of the study and provide written evidence of informed consent as approved by Institutional Review Board (IRB)/Ethics Committee (EC) * Recovery from adverse events of previous systemic anti-cancer therapies to baseline or grade 1, except for: * Alopecia * Stable neuropathy of =\< grade 2 due to prior cancer therapy * Able to swallow and retain oral medication * Willing and able to comply with scheduled visits, treatment plan and laboratory tests Exclusion Criteria: * Patients who have therapies available that are known to confer a clinical benefit will be excluded from the study * Neurological symptoms related to underlying disease requiring increasing doses of corticosteroids. Note: Steroid use for management of CNS tumors is allowed but must be at a stable dose for at least 2 weeks preceding study entry * History of another primary malignancy except adequately treated in situ carcinoma of the cervix or non-melanoma carcinoma of the skin or any other curatively treated malignancy that is not expected to require treatment for recurrence during the course of the study or affect survival * Any other medical condition that would, in the investigator's judgment, prevent the patient's participation in the clinical study due to safety concerns or compliance with clinical study procedures * Current evidence of corneal or retinal disorder/keratopathy including, but not limited to, bullous/band keratopathy, corneal abrasion, inflammation/ulceration, keratoconjunctivitis, confirmed by ophthalmologic examination * History and/or current evidence of extensive tissue calcification including, but not limited to, the soft tissue, kidneys, intestine, myocardium and lung with the exception of calcified lymph nodes, minor pulmonary parenchymal calcifications, and asymptomatic coronary calcification * Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral infigratinib (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection) * Current evidence of endocrine alterations of calcium/phosphate homeostasis, e.g., parathyroid disorders, history of parathyroidectomy, tumor lysis, tumoral calcinosis etc * Treatment with any of the following anti-cancer therapies prior to the first dose of infigratinib within the stated timeframes: * Cyclical chemotherapy (intravenous) within a period of time that is shorter than the cycle length used for that treatment (e.g., 6 weeks for nitrosourea, mitomycin-C) * Biological therapy (e.g., antibodies - including bevacizumab) within a period of time that is =\< 5 half-life (t1/2) or =\< 4 weeks, whichever is shorter, prior to starting study drug * Continuous or intermittent small molecule therapeutics within a period of time that is =\< 5 t1/2 or =\< 4 weeks (whichever is shorter) prior to starting study drug * Any other investigational agents within a period of time that is =\< 5 t1/2 or less than the cycle length used for that treatment or =\< 4 weeks (whichever is shortest) prior to starting study drug * Wide field radiotherapy (including therapeutic radioisotopes such as strontium 89) =\< 4 weeks or limited field radiation for palliation =\< 2 weeks prior to starting study drug * Patients who are currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 and medications which increase serum phosphorus and/or calcium concentration are excluded. Patients are not permitted to receive enzyme-inducing anti-epileptic drugs * Consumption of grapefruit, grapefruit juice, grapefruit hybrids, pomegranates, star fruits, pomelos, Seville oranges or products within 7 days prior to first dose * Use of medications that are known to prolong the QT interval and/or are associated with a risk of Torsades de Pointes (TdP) 7 days prior to first dose * Use of amiodarone within 90 days prior to first dose * Current use of therapeutic doses of warfarin sodium or any other coumadin-derivative anticoagulants. Heparin and/or low molecular weight heparins are allowed * Absolute neutrophil count (ANC) =\< 1,000/mm\^3 (1.0 x 10\^9/L) * Platelets =\< 75,000/mm\^3 (75 x 10\^9/L) * Hemoglobin =\< 9.0 g/dL * Total bilirubin \>= 1.5 x upper limit of normal (ULN) unless associated with patient's primary cancer and/or metastases and with principal investigator's approval * Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \>= 3 x ULN unless associated with patient's primary cancer and/or metastases and with principal investigator's approval * Alkaline phosphatase \>= 2.5 x ULN unless associated with patient's primary cancer and/or metastases and with principal investigator's approval * Calculated or measured creatinine clearance of \< 40 mL/min * Calcium-phosphate homeostasis: * Inorganic phosphorus outside of institutional normal limits * Total serum calcium (can be corrected) outside of institutional normal limits * Clinically significant cardiac disease including any of the following: * Congestive heart failure requiring treatment (New York Heart Association \[NYHA\] grade \>= 2), left ventricular ejection fraction (LVEF) \< 50% as determined by multi-gated acquisition (MUGA) scan or echocardiogram (ECHO), or uncontrolled hypertension (refer to World Health Organization \[WHO\] and International Society for Hypertension \[ISH\] guidelines) * History or presence of clinically significant ventricular arrhythmias, atrial fibrillation, resting bradycardia, or conduction abnormality * Unstable angina pectoris or acute myocardial infarction =\< 3 months prior to starting study drug * Corrected QT using Fridericia's formula (QTcF) \> 470 msec (males and females) * History of congenital long QT syndrome * Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive human chorionic gonadotropin (hCG) laboratory test * Women of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 3 months following the discontinuation of study treatment. Highly effective contraception methods include: * Total abstinence (when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception * Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow-up hormone level assessment * Male sterilization (at least 6 months prior to screening). For female patients on the study the vasectomized male partner should be the sole partner for that patient * Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine systems (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate \< 1%), for example hormone vaginal ring or transdermal hormone contraception * In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment. Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least 6 weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow-up hormone level assessment is she considered not of child bearing potential * Sexually active males unless they use a condom during intercourse while taking drug and for 3 months after the last dose of the study drug and should not father a child in this period. A condom is required to be used also by vasectomized men as well as during intercourse with a male partner in order to prevent delivery of the drug via seminal fluid

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.