Derazantinib in Subjects With FGFR2 Gene Fusion-, Mutation- or Amplification- Positive Inoperable… (NCT03230318) | Clinical Trial Compass
CompletedPhase 2
Derazantinib in Subjects With FGFR2 Gene Fusion-, Mutation- or Amplification- Positive Inoperable or Advanced Intrahepatic Cholangiocarcinoma
United States, Belgium, Canada148 participantsStarted 2017-09-28
Plain-language summary
This Phase II, open-label, single-arm study evaluated the anti-cancer activity of derazantinib in subjects with inoperable or advanced intrahepatic cholangiocarcinoma (iCCA) who received at least one prior regimen of systemic therapy. Patients received an oral once-daily total dose of 300 mg derazantinib capsules.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Signed written informed consent granted prior to initiation of any study-specific procedures
. 18 years of age or older
. Histologically or cytologically confirmed locally advanced, inoperable, or metastatic iCCA or mixed histology tumors
. Substudy 1:
. Received at least one regimen of prior systemic therapy and then experienced documented radiographic progression
. Measurable disease by RECIST version 1.1 criteria
. ECOG performance status ≤ 1
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial specifically enrolled patients with FGFR2 gene fusions, mutations, or amplifications — has my tumor been tested for any of these changes, and which category would I fall into if I did qualify?
2The trial measured two different things depending on the substudy — response rate in one group and progression-free survival at 3 months in the other — so based on those results, how meaningful does the benefit from derazantinib appear to be compared to what we'd expect from standard treatment for my stage of intrahepatic cholangiocarcinoma?
3Since this was a Phase 2 trial and it's now completed, what does the safety data tell us about the side effects patients experienced with derazantinib, and are there any risks I should weigh against other FGFR2-targeted options that might be available?
4Are there now approved FGFR2-targeted therapies — like pemigatinib or futibatinib — that came out of similar research, and would those be a more established path for me to consider before looking at trials like this one?
5Now that this trial is completed, are the full results published and would you be able to review them with me so we can understand whether derazantinib or a related therapy might make sense as part of my treatment plan?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Substudy 1: Objective Response Rate (ORR)
Timeframe: From first dose and up to 54 months
2
Substudy 2: Progression Free Survival at 3 Months (PFS 3)
. Patients who were unable or unwilling to swallow the complete daily dose of derazantinib capsules
. Clinically unstable central nervous system (CNS) metastases (to be eligible, subjects must had stable disease ≥ 3 months, confirmed by magnetic resonance imaging (MRI) or computed tomography (CT) scan, and/or had CNS metastases well controlled by low-dose steroids, anti-epileptics, or other symptom-relieving medications)
. Evidence of clinically significant corneal or retinal disorder which was likely to increase the risk of eye toxicity, including but not limited to bullous/band keratopathy, keratoconjunctivitis (unless keratoconjunctivitis sicca), corneal abrasion, inflammation/ulceration, confirmed by ophthalmologic examination.
. Uncontrolled or active hepatobiliary disorders, untreated or ongoing complications after laparoscopic procedures or stent placement, including but not limited to active cholangitis, biloma or abscess (to be eligible, the subjects had to be treated and disorders/complications should had been resolved within 2 weeks prior to the first dose of derazantinib)