A Phase II Nationwide, Telemedicine Study Of Pemigatinib In Adult Patients With Advanced Or Metastatic Pancreas Cancer With FGFR Genetic Alterations

Study Overview

A Phase II Nationwide, Telemedicine Study of Pemigatinib in Adult Patients With Advanced or Metastatic Pancreas Cancer With FGFR Genetic Alterations

This phase II study evaluates how well pemigatinib works for the treatment of adult patients with pancreatic cancer that has spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or has spread from where it first started to other places in the body (metastatic) and that have abnormal changes (alterations) in the fibroblast growth factor receptor (FGFR) gene. FGFR genes are genes that, when altered, can lead to and promote the growth of cancer in patients. Researchers want to test if using pemigatinib can block the function of these abnormal FGFR genes and prevent the tumor from growing and whether treatment can help improve overall quality of life.

PRIMARY OBJECTIVES: I. To evaluate the efficacy of single agent pemigatinib in patients with advanced or metastatic pancreas cancer of any histologic classification with FGFR2 gene fusions/translocations. II. To understand response rate and potential for pemigatinib to benefit patients who have other FGFR alterations including point mutations, extracellular small indels and kinase domain duplications in pancreas cancer. SECONDARY OBJECTIVES: I. To further evaluate the efficacy of single agent pemigatinib in each above cohort separately. II. To characterize the safety and tolerability of single agent pemigatinib. EXPLORATORY OBJECTIVE: I. To evaluate dynamics of cell-free deoxyribonucleic acid (DNA) (cfDNA) optimized for monitoring response to pemigatinib and detecting emerging resistance mutations to pemigatinib. OUTLINE: Patients receive pemigatinib orally (PO) once daily (QD) on days 1-14 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection, computed tomography (CT) and/or magnetic resonance imaging (MRI), and optical coherence tomography (OCT) throughout the study. Patients may also undergo whole body bone scans and dilated fundoscopy as clinically indicated. After completion of study treatment, patients are followed up at 30 days, then every 4 months for one year.

Advanced Pancreatic Carcinoma
Metastatic Pancreatic Carcinoma
Stage II Pancreatic Cancer AJCC v8
Stage III Pancreatic Cancer AJCC v8
Stage IV Pancreatic Cancer AJCC v8

DRUG: Pemigatinib
PROCEDURE: Computed Tomography (CT)
PROCEDURE: Magnetic Resonance Imaging
PROCEDURE: Optical Coherence Tomography
PROCEDURE: Bone Scan
PROCEDURE: Ophthalmoscopy

OSU-23382
NCI-2025-03043 (REGISTRY Identifier) (REGISTRY: CTRP (Clinical Trial Reporting Program))

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates	Results Reporting Dates	Study Record Updates
First Submitted 2025-03-26	Results First Submitted N/A	Last Update Submitted that met QC Criteria 2025-06-26
First Submitted that Met QC Criteria 2025-03-26	Results First Submitted that Met QC Criteria N/A	Last Update Posted (ACTUAL) 2025-06-29
First Posted (ACTUAL) 2025-04-02	Results First Posted N/A	Last Verified 2025-06

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

Primary Purpose:
Treatment

Allocation:
Na

Interventional Model:
Single Group

Masking:
None

Arms and Interventions

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Pemigatinib Treament Patients receive pemigatinib PO QD on days 1-14 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection, CT and/or MRI, and OCT throughout the study. Patients	DRUG: Pemigatinib Pemigatinib will be taken orally with a targeted starting does of 13.5 mg PROCEDURE: Computed Tomography (CT) Undergo CT scan PROCEDURE: Magnetic Resonance Imaging Undergo MRI PROCEDURE: Optical Coherence Tomography Undergo OCT PROCEDURE: Bone Scan Undergo whole body bone scan PROCEDURE: Ophthalmoscopy Undergo dilated ophthalmoscopy

Participant Group/Arm

Intervention/Treatment

EXPERIMENTAL: Pemigatinib Treament

Patients receive pemigatinib PO QD on days 1-14 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection, CT and/or MRI, and OCT throughout the study. Patients

DRUG: Pemigatinib

Pemigatinib will be taken orally with a targeted starting does of 13.5 mg

PROCEDURE: Computed Tomography (CT)

Undergo CT scan

PROCEDURE: Magnetic Resonance Imaging

Undergo MRI

PROCEDURE: Optical Coherence Tomography

Undergo OCT

PROCEDURE: Bone Scan

Undergo whole body bone scan

PROCEDURE: Ophthalmoscopy

Undergo dilated ophthalmoscopy

Primary Outcome Measures	Measure Description	Time Frame
Overall Response Rate (ORR)	The proportion of patients with a best overall response of complete response (CR) or partial response (PR). Overall response rate assessed per RECIST v1.1. Will be evaluated in all patients who received at least 80% of the recommended dose of pemigatinib averaged over a 9-week period. ORR will be calculated along with its 95% confidence interval	Up to 24 months

Secondary Outcome Measures	Measure Description	Time Frame
Progression Free Survival (PFS)	PFS will be evaluated in all patients who received at least 80% of the recommended dose of pemigatinib averaged over a 9-week period. Will be analyzed using the Kaplan-Meier method.	Up to 12 months
Disease Control Rate (DCR)	Defined as the proportion of patients with a best overall response of CR or PR or SD. Will be assessed using descriptive stats. The estimated ORR and corresponding 95% confidence intervals based on the binomial distribution will be reported.	Up to 12 months
Overall Survival (OS)	OS will be evaluated in all patients who received at least 80% of the recommended dose of pemigatinib averaged over a 9-week period. Will be analyzed using the Kaplan-Meier method.	Up to 12 months
Type, frequency and severity of adverse events	The severity of AEs will be assessed according to the NCI CTCAE v5.0	Up to 12 months
Best overall response (BOR)	BOR will be summarized for each cohorts using the ORR and the Disease Control Rate which are the proportion of patients having respectively a best overall response of partial response (PR) or complete response (CR), or stable disease (SD), PR or CR. The estimated ORR and corresponding 95% confidence intervals based on the binomial distribution will be reported.	Baseline up to 1 year after completion of study treatment.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Name: The Ohio State University Comprehensive Cancer Center

Phone Number: 614-685-4296

Email: OSUCCCClinicaltrials@osumc.edu

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person’s general health condition or prior treatments.

Ages Eligible for Study:
ALL

Sexes Eligible for Study:
18 Years

Accepts Healthy Volunteers:

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 pancreatic cancer in the following cohorts:

Cohort 1: Pancreatic cancer of any histology with FGFR2 fusion/translocation (n, up to 30) who have progressed on or are intolerant to at least one standard of care (SOC) therapy. Prior therapy with a different FGFR inhibitor is not permitted. Patients with concurrent Kirsten rat sarcoma (KRAS) mutations are excluded from this cohort
Cohort 2: Pancreatic cancer of any histology with activating point mutations, fusion/translocation (FGFR1,3,4) extracellular small indels, or kinase domain duplications (n, up to 10). Patients must have progressed on or are intolerant to at least one SOC therapy. Prior therapy with a different FGFR inhibitor is not permitted. Patients with concurrent KRAS mutations 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 pancreas cancer. Patients 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 not eligible for the study
Patients with symptomatic central nervous system (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
Patients ≥ 18 years of age of either gender
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 Incyte)
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

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 , 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, keratoconjuctivitis, 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 pemigatinib (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 pemigatinib 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 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
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 ≥ 1.5x 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) ≥ 3x ULN unless associated with patient's primary cancer and/or metastases and with principal investigator's approval
Alkaline phosphatase ≥ 2.5x 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
History of clinically significant or uncontrolled cardiac disease including unstable angina, acute myocardial infarction, New York Heart Association Class III or IV congestive heart failure, or arrhythmia requiring therapy. Subjects with a pacemaker and well-controlled rhythm for at least 1 month prior to first dose will be allowed
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Incyte Corporation

Investigators

PRINCIPAL_INVESTIGATOR: Sameek Roychowdhury, MD, PhD, Ohio State University Comprehensive Cancer Center

Publications

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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