Pemigatinib for Pancreatic Cancer

Overseen By The Ohio State University Comprehensive Cancer Center

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

Trial Summary

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.

What is known about the safety of pemigatinib in humans?

Pemigatinib has been associated with some side effects, including nail changes and kidney stones, based on safety studies and reports. These findings come from its use in treating cholangiocarcinoma, a type of cancer, but they provide insight into its general safety profile. ^{1 2 3 4 5}

How does the drug Pemigatinib differ from other treatments for pancreatic cancer?

Pemigatinib is a targeted therapy that works by inhibiting specific pathways involved in cancer cell growth, which is different from the conventional chemotherapy used for pancreatic cancer. While standard treatments like gemcitabine are cytotoxic (kill cancer cells directly), Pemigatinib targets specific molecular changes in cancer cells, potentially offering a more personalized treatment approach. ^{6 7 8 9 10}

What is the purpose of this trial?

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.

Research Team

Sameek Roychowdhury, MD, PhD

Principal Investigator

Ohio State University Comprehensive Cancer Center

Eligibility Criteria

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.

I can swallow pills.

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Pemigatinib-induced nail changes - A case report. [2021]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Safety analysis of pemigatinib leveraging the US Food and Drug administration adverse event reporting system. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

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

4. England pubmed.ncbi.nlm.nih.gov

A drug safety evaluation of pemigatinib for advanced cholangiocarcinoma. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study. [2023]

6. France pubmed.ncbi.nlm.nih.gov

Molecular Targeting of a BRAF Mutation in Pancreatic Ductal Adenocarcinoma: Case Report and Literature Review. [2021]

7. England pubmed.ncbi.nlm.nih.gov

Small molecule inhibitors in pancreatic cancer. [2023]

8. United States pubmed.ncbi.nlm.nih.gov

KRASG12D inhibition reprograms the microenvironment of early and advanced pancreatic cancer to promote FAS-mediated killing by CD8+ T cells. [2023]

9. United States pubmed.ncbi.nlm.nih.gov

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

10. China pubmed.ncbi.nlm.nih.gov

State of the art biological therapies in pancreatic cancer. [2023]