Futibatinib for Cancer

Enrolling by invitation at 9 trial locations

Overseen By Taiho Oncology, Inc

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Taiho Oncology, Inc.

Must be taking: Futibatinib

Disqualifiers: Discontinuation criteria met, 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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the drug futibatinib, either alone or combined with fulvestrant, to evaluate its effectiveness for patients who benefited from it in an earlier study. It targets individuals already taking futibatinib and experiencing positive effects without significant risks. Participants should be able to take medication orally and have participated in a previous futibatinib study, demonstrating benefits without major issues. The trial aims to gather more information on the effectiveness and safety of continuing futibatinib treatment. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It seems focused on patients already receiving futibatinib, so you may need to continue with that treatment.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

A previous study demonstrated that futibatinib was generally safe for 469 patients with various tumor types, with most side effects being mild. Another study found that no patients died while taking futibatinib, which is reassuring.

Early results for the combination of futibatinib and fulvestrant suggest it is safe and tolerable, with side effects remaining consistent over time. These findings indicate that both treatment options are generally well-tolerated. However, consulting a doctor is essential to determine if this treatment is appropriate. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about futibatinib because it targets cancer cells in a unique way. Unlike most standard treatments that focus on inhibiting multiple pathways, futibatinib specifically targets the FGFR (fibroblast growth factor receptor) pathway, which is often altered in certain cancers. This precision allows it to potentially stop cancer growth more effectively while minimizing damage to healthy cells. Additionally, the combination of futibatinib with fulvestrant offers a promising approach by not only targeting the FGFR pathway but also disrupting estrogen receptor signaling, which is crucial in some breast cancers. These features give futibatinib the potential to be more effective and have fewer side effects than current treatments.

What evidence suggests that this trial's treatments could be effective for cancer?

This trial will evaluate futibatinib as a monotherapy and in combination with fulvestrant for treating cancers with changes in the FGFR gene. Studies have shown that futibatinib alone is effective, with research indicating that patients' quality of life remains stable for about 9 months. The main benefits observed were the cancer's response to the treatment and the duration of these positive effects.

In this trial, some participants will receive futibatinib with fulvestrant. Studies have shown that this combination prevented cancer from worsening for an average of 7.2 months, with about 50% of patients experiencing a clinical benefit, meaning their cancer either shrank or stopped growing for some time. These findings suggest that futibatinib, whether used alone or with fulvestrant, may be promising for certain cancer patients. ^{2 3 4 5 6}

Are You a Good Fit for This Trial?

This trial is for patients who have previously been treated with futibatinib, either alone or in combination with other drugs, in a prior Taiho-sponsored study. They must be benefiting from the treatment without significant risks and able to take oral medication. Women of childbearing age need a negative pregnancy test and agree to use birth control.

Inclusion Criteria

I have signed a consent form to participate.

I am not pregnant and agree to use birth control during and after the study.

I can take medications by mouth.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants continue to receive futibatinib as monotherapy or in combination with fulvestrant, as per antecedent study design

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 years

Open-label extension

Participants may continue receiving treatment long-term if they are deriving clinical benefit with no undue risk

What Are the Treatments Tested in This Trial?

Interventions

• Futibatinib

Trial Overview The trial is testing futibatinib as both a single agent and in combination with fulvestrant for cancer treatment. It's an open-label, nonrandomized study which means everyone knows what treatment they're getting and there's no chance element to assignment.

How Is the Trial Designed?

Group I: TAS-120 (futibatinib) monotherapy Experimental Treatment 1 Intervention

TAS-120 tablets, oral, cycle length is defined as per antecedent study design

Group II: TAS-120 (futibatinib) combination therapy with fulvestrant Experimental Treatment 1 Intervention

TAS-120 tablets in combination with fulvestrant, oral, cycle length is defined as per antecedent study design

Who Is Running the Clinical Trial?

Taiho Oncology, Inc.

Lead Sponsor

Trials

79

Recruited

12,700+

Tim Whitten

Taiho Oncology, Inc.

Chief Executive Officer since 2018

MBA and Pharmacy degree

Harold Keer

Taiho Oncology, Inc.

Chief Medical Officer

MD, PhD

Published Research Related to This Trial

Futibatinib is an oral medication that irreversibly inhibits fibroblast growth factor receptors (FGFR1-4) and has been approved for treating adult patients with unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma with FGFR2 gene fusions or rearrangements.

The drug was officially approved in the USA on September 30, 2022, marking a significant milestone in its development for various cancers, including cholangiocarcinoma, breast cancer, and others.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Futibatinib: First Approval. Syed, YY.[2022]

In a phase 2 study involving 103 patients with FGFR2 fusion-positive intrahepatic cholangiocarcinoma, futibatinib demonstrated a 42% objective response rate, with a median response duration of 9.7 months, indicating significant antitumor activity.

Futibatinib was associated with manageable safety profiles, with common grade 3 adverse events including hyperphosphatemia and fatigue, but no treatment-related deaths, and quality of life was maintained throughout the treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Futibatinib for FGFR2-Rearranged Intrahepatic Cholangiocarcinoma. Goyal, L., Meric-Bernstam, F., Hollebecque, A., et al.[2023]

Futibatinib, an FGFR inhibitor, was found to have manageable safety and pharmacodynamic activity in a phase I trial involving 86 patients with advanced solid tumors, with a recommended phase II dose of 20 mg once daily.

The trial showed that 5 patients had partial responses to futibatinib, particularly in those with FGFR aberrations, and 41 patients (48%) experienced stable disease, indicating potential efficacy in this patient population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase I, first-in-human study of futibatinib, a highly selective, irreversible FGFR1-4 inhibitor in patients with advanced solid tumors. Bahleda, R., Meric-Bernstam, F., Goyal, L., et al.[2022]

Futibatinib received accelerated FDA approval for treating unresectable intrahepatic cholangiocarcinoma (iCCA) with FGFR2 fusions, showing an overall response rate of 42% and a median duration of response of 9.7 months in a multicenter trial with previously treated patients.

Common side effects included nail toxicity, musculoskeletal pain, and ocular toxicity, highlighting the importance of monitoring for these adverse reactions during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

FDA Approval Summary: Futibatinib for Unresectable Advanced or Metastatic, Chemotherapy Refractory Intrahepatic Cholangiocarcinoma with FGFR2 Fusions or Other Rearrangements. Gandhy, SU., Casak, SJ., Mushti, SL., et al.[2023]

Savolitinib demonstrated a median overall survival of 12.5 months in patients with MET exon 14 positive non-small cell lung cancer (NSCLC), with longer survival in pretreated patients (19.4 months) compared to treatment-naive patients (10.9 months).

The safety profile of savolitinib remained favorable over the long term, with no new safety concerns identified during the extended follow-up period of 28.4 months in a study involving 70 patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-Term Efficacy, Safety, and Subgroup Analysis of Savolitinib in Chinese Patients With NSCLCs Harboring MET Exon 14 Skipping Alterations. Lu, S., Fang, J., Li, X., et al.[2022]

Futibatinib, an irreversible FGFR1-4 inhibitor, showed an objective response rate of 13.7% in a phase I trial with 197 patients across various advanced solid tumors, particularly effective in FGFR2 fusion-positive intrahepatic cholangiocarcinoma with a response rate of 25.4%.

The treatment had a manageable safety profile, with common side effects including hyperphosphatemia (81.2%), diarrhea (33.5%), and nausea (30.4%), supporting its further evaluation in ongoing phase II/III trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Futibatinib, an Irreversible FGFR1-4 Inhibitor, in Patients with Advanced Solid Tumors Harboring FGF/FGFR Aberrations: A Phase I Dose-Expansion Study. Meric-Bernstam, F., Bahleda, R., Hierro, C., et al.[2023]

Citations

1. cancernetwork.com cancernetwork.com/view/futibatinib-fulvestrant-aes-remains-consistent-in-advanced-fgfr-breast-cancer

Futibatinib/Fulvestrant AEs Remains Consistent in ... While futibatinib/fulvestrant resulted in a median progression-free survival of 7.2 months (95% CI, 2.1-7.6) and a clinical benefit rate of 50% ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04024436

A Study of TAS-120 in Patients With Metastatic Breast Cancer This is a Phase 2, open-label, non-randomized, multicenter study designed to evaluate the efficacy and safety of futibatinib (TAS-120) and futibatinib + ...

3. aacrjournals.org aacrjournals.org/cancerres/article/84/9_Supplement/RF01-04/744435/Abstract-RF01-04-Final-results-from-the-phase-2

Final results from the phase 2, open-label FOENIX-MBC2 ... Conclusions: Futibatinib plus fulvestrant showed antitumor activity in patients with advanced HR+/HER2− breast cancer with FGFR1 amplification ...

4. onclive.com onclive.com/view/dr-damodaran-on-futibatinib-plus-fulvestrant-in-fgfr1-amplified-hr-her2-breast-cancer

Dr Damodaran on Futibatinib Plus Fulvestrant in FGFR1- ... The agent also produced a partial response rate of 18.2% (95% CI, 5.2%-40.3%), stable disease in 50% of patients, and disease progression in ...

5. cancernetwork.com cancernetwork.com/view/final-results-show-pfs-benefit-with-futibatinib-for-hr-her2-breast-cancer

Final Results Show PFS Benefit With Futibatinib for HR+ ... We combined futibatinib with fulvestrant. The primary end point of the study was 6-month PFS rate. The observed rate was about 52%, and the ...

6. aacrjournals.org aacrjournals.org/cancerres/article/82/4_Supplement/P1-18-35/680533/Abstract-P1-18-35-Futibatinib-in-combination-with

Abstract P1-18-35: Futibatinib in combination with fulvestrant ... Conclusions: Based on these preliminary safety results, the combination of futibatinib and fulvestrant appears to be safe and tolerable in ...