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

Phase 2

Recruiting

• Conditions: Advanced Pancreatic Carcinoma; Metastatic Pancreatic Carcinoma; Stage II Pancreatic Cancer AJCC v8; Stage III Pancreatic Cancer AJCC v8; Stage IV Pancreatic Cancer AJCC v8
• Interventions: Drug: Pemigatinib; Procedure: Computed Tomography (CT); Procedure: Magnetic Resonance Imaging; Procedure: Optical Coherence Tomography; Procedure: Bone Scan; Procedure: Ophthalmoscopy

• Registration Number: NCT06906562
• Lead Sponsor: Sameek Roychowdhury

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study First Submitted: 2025-03-26
• Study First Submitted QC: 2025-03-26
• Study First Posted: 2025-04-02
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-08
• Last Update Posted: 2025-05-11
• Study Start: 2025-06-01
• Primary Completion: 2026-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Pemigatinib Treament	Pemigatinib	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 may also undergo whole body bone scans and dilated fundoscopy as clinically indicated.
Pemigatinib Treament	Computed Tomography (CT)	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 may also undergo whole body bone scans and dilated fundoscopy as clinically indicated.
Pemigatinib Treament	Magnetic Resonance Imaging	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 may also undergo whole body bone scans and dilated fundoscopy as clinically indicated.
Pemigatinib Treament	Optical Coherence Tomography	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 may also undergo whole body bone scans and dilated fundoscopy as clinically indicated.
Pemigatinib Treament	Bone Scan	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 may also undergo whole body bone scans and dilated fundoscopy as clinically indicated.
Pemigatinib Treament	Ophthalmoscopy	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 may also undergo whole body bone scans and dilated fundoscopy as clinically indicated.

Primary Outcome Measures

Name	Time	Method
Overall Response Rate (ORR)	Up to 24 months	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

Secondary Outcome Measures

Name	Time	Method
Progression Free Survival (PFS)	Up to 12 months	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.
Disease Control Rate (DCR)	Up to 12 months	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.
Overall Survival (OS)	Up to 12 months	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.
Best overall response (BOR)	Baseline up to 1 year after completion of study treatment.	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.
Type, frequency and severity of adverse events	Up to 12 months	The severity of AEs will be assessed according to the NCI CTCAE v5.0

Trial Locations

Locations (1)

Ohio State University Comprehensive Cancer Center; 🇺🇸 Columbus, Ohio, United States