Pemigatinib for Pancreatic Cancer

Recruiting at 72 trial locations

Overseen By The Ohio State University Comprehensive Cancer Center and James Cancer Hospital

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sameek Roychowdhury

Must not be taking: CYP3A4 modulators

Disqualifiers: CNS metastases, Cardiac disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 30

Duration: Up to 24 months

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness of pemigatinib, a drug designed to halt pancreatic cancer growth by blocking abnormal genes that drive cancer progression. It targets patients whose pancreatic cancer has spread and involves changes in their FGFR genes, known to promote cancer growth. Patients who have tried at least one standard treatment without success and have FGFR gene changes in their pancreatic cancer may be suitable for this trial. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you must stop taking your current medications, but you cannot take drugs that strongly affect certain liver enzymes (CYP3A4) or medications that increase phosphorus or calcium levels. You should discuss your current medications with the trial team to see if any adjustments are needed.

Is there any evidence suggesting that pemigatinib is likely to be safe for humans?

Research has shown that pemigatinib is generally safe. In previous studies, patients with other cancers, such as bile duct cancer, took pemigatinib and most tolerated it well.

For instance, one study found that only about 22% of patients experienced serious side effects, which were mostly moderate. There were no reports of extremely severe side effects.

This indicates that pemigatinib is usually well-tolerated. However, like any treatment, it can cause side effects. It is important to consult a doctor to understand what to expect and how to manage any side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for pancreatic cancer?

Unlike the standard treatments for pancreatic cancer, which typically include chemotherapy and targeted therapies like gemcitabine or erlotinib, pemigatinib acts differently by specifically targeting fibroblast growth factor receptors (FGFRs). This new mechanism of action may help in attacking cancer cells more precisely and potentially lead to better outcomes with fewer side effects. Researchers are excited about pemigatinib because it offers a novel approach that could provide new hope for patients who may not respond well to existing therapies.

What evidence suggests that pemigatinib might be an effective treatment for pancreatic cancer?

Research has shown that pemigatinib, which participants in this trial will receive, may help treat cancers with changes in the FGFR gene. In studies involving various solid tumors, including pancreatic cancer, patients responded well to pemigatinib. Specifically, some studies found that pemigatinib helped patients go nearly 9 months without their cancer worsening. Additionally, patients lived for about 17 months on average. These results suggest that pemigatinib might help control tumor growth in pancreatic cancer with FGFR changes. ^{3 5 6 7 8}

Who Is on the Research Team?

Sameek Roychowdhury, MD, PhD

Principal Investigator

Ohio State University Comprehensive Cancer Center

Are You a Good Fit for This Trial?

Adults with advanced or metastatic pancreatic cancer that has specific genetic changes called FGFR alterations can join this trial. The study is looking for people whose cancer hasn't responded to standard treatments.

Inclusion Criteria

I can carry out all my self-care but not work activities.

I have recovered from previous cancer treatments, except for hair loss or mild nerve damage.

Willing and able to comply with scheduled visits, treatment plan, and laboratory tests

Exclusion Criteria

I have treatment options available that are known to help my condition.

I need more steroids to manage my neurological symptoms.

I haven't taken specific cancer drugs before starting pemigatinib recently.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive pemigatinib orally once daily on days 1-14 of each 21-day cycle

Up to 24 months

Regular visits for blood sample collection, CT/MRI, and OCT

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Follow-up at 30 days, then every 4 months for one year

What Are the Treatments Tested in This Trial?

Interventions

• Pemigatinib

Trial Overview

The trial tests Pemigatinib, a drug targeting certain genes in cancer cells, to see how well it works on its own in treating pancreatic cancer. Participants will also undergo CT and MRI scans for detailed imaging of the tumor's response.

How Is the Trial Designed?

Group I: Pemigatinib Treament Experimental Treatment 6 Interventions

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.

Who Is Running the Clinical Trial?

Sameek Roychowdhury

Lead Sponsor

Trials

3

Recruited

40+

Incyte Corporation

Industry Sponsor

Trials

408

Recruited

66,800+

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School

Published Research Related to This Trial

Pemigatinib is an effective treatment for patients with advanced cholangiocarcinoma that have FGFR2 genomic alterations, showing a 35.5% objective response rate after a median follow-up of 17.8 months in a phase II study.

The drug has a manageable safety profile, with common side effects including changes in blood phosphate levels, fatigue, and gastrointestinal issues, leading to its accelerated approval by the FDA for specific cancer patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A drug safety evaluation of pemigatinib for advanced cholangiocarcinoma. Uson, PLS., Bearss, J., Babiker, HM., et al.[2023]

Pemigatinib has shown effectiveness in treating patients with cholangiocarcinomas that have FGFR2 fusions or rearrangements, indicating its potential as a targeted therapy for this specific cancer type.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pemigatinib Is Active in Some FGFR2-Altered Cholangiocarcinomas. [2021]

A patient with advanced pancreatic ductal adenocarcinoma harboring a V600E BRAF mutation showed an objective tumor response to a combination treatment of the BRAF inhibitor vemurafenib and the MEK inhibitor cobimetinib.

This case highlights the potential for targeted therapy in a small subset of pancreatic cancer patients, suggesting that BRAF mutations, although rare, may offer a new avenue for treatment beyond conventional chemotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Molecular Targeting of a BRAF Mutation in Pancreatic Ductal Adenocarcinoma: Case Report and Literature Review. Seghers, AK., Cuyle, PJ., Van Cutsem, E.[2021]

Pancreatic cancer has a very low 5-year survival rate of less than 7%, and current therapies have not significantly improved patient outcomes, with median overall survival (MoS) for standard treatments like gemcitabine and fluorouracil being around 23 months.

Some clinical trials of new small molecule inhibitors show promise, such as a phase I trial with vorinostat and capecitabine achieving a MoS of 13 months, and a phase II trial of pazopanib showing a MoS of 25 months, indicating potential for improved treatment strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Small molecule inhibitors in pancreatic cancer. Sun, J., Russell, CC., Scarlett, CJ., et al.[2023]

Erlotinib, a targeted therapy that inhibits the epidermal growth factor receptor, has been approved for use alongside gemcitabine in treating advanced pancreatic cancer, marking a significant step in personalized cancer treatment.

Despite the exploration of various targeted agents, most have shown limited success, highlighting the need for therapies tailored to the specific molecular characteristics of both the patient and the tumor.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Future strategies for targeted therapies and tailored patient management in pancreatic cancer. Ko, AH.[2018]

Citations

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ascopubs.org

ascopubs.org/doi/10.1200/JCO.2024.42.3_suppl.TPS717

A phase II telemedicine study of pemigatinib in adult ...

Our observations provide preliminary evidence that FGFR targeted therapy results in excellent clinical outcomes. We hypothesize that FGFR ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11186762/

Pemigatinib in previously treated solid tumors with activating ...

Efficacy outcomes are summarized in Table 2 and Extended Data Fig. 1. Fig. 2. Best percent change from baseline by FGFR co-alteration ...

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06906562

Study Details | NCT06906562 | A Phase II Nationwide, ...

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 ...

4.

sciencedirect.com

sciencedirect.com/science/article/pii/S0959804924000637

Pemigatinib for patients with previously treated, locally ...

The median PFS was 8.7 months (95%CI: 7.3–11.8; 41/72 events), and the median OS was 17.1 months (95%CI: 12.7-NA; 27/72 events) (Fig. 1, Fig.

5.

esmoopen.com

esmoopen.com/article/S2059-7029(24)01257-2/fulltext

An open-label study of pemigatinib in cholangiocarcinoma

Pemigatinib demonstrated durable response and prolonged OS with manageable AEs in patients with previously treated, advanced/metastatic CCA with ...

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8461541/

Pemigatinib for previously treated, locally advanced or ...

This study evaluated the safety and antitumour activity of pemigatinib in patients with previously treated, locally advanced or metastatic cholangiocarcinoma

7.

sciencedirect.com

sciencedirect.com/science/article/pii/S0923753422001107

FIGHT-101, a first-in-human study of potent and selective ...

Pemigatinib was associated with a manageable safety profile and pharmacodynamic and clinical activity, with responses seen across tumors and driven by FGFR ...

8.

ejcancer.com

ejcancer.com/article/S0959-8049(24)00063-7/fulltext

Pemigatinib for patients with previously treated, locally ...

Safety profile of pemigatinib and treatment modification. Open table in a new tab. Grade 3 AEs affected 16 (22.2%) patients. No G4 AEs and no ...