Futibatinib for Cancer

Recruiting at 7 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

15 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a nonrandomized, open-label, multicenter rollover study for patients who received futibatinib as monotherapy or as combination therapy in a Taiho-sponsored futibatinib study.

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.

What data supports the effectiveness of the drug Futibatinib for cancer?

Futibatinib has shown effectiveness in treating cancers with FGFR (fibroblast growth factor receptor) alterations, such as intrahepatic cholangiocarcinoma and breast cancer, by inhibiting tumor growth and overcoming resistance to other treatments. It has been approved in the USA for specific cases of cholangiocarcinoma and demonstrated promising results in preclinical and clinical studies for various FGFR-driven tumors. ^{1 2 3 4 5}

Is futibatinib safe for humans?

Futibatinib has been tested in humans and shows a manageable safety profile. Common side effects include nail issues, muscle pain, constipation, diarrhea, fatigue, dry mouth, hair loss, mouth sores, and abdominal pain. Important risks include eye problems and high phosphate levels in the blood. ^{1 2 6 7 8}

How is the drug futibatinib unique in treating cancer?

Futibatinib is unique because it is an oral drug that irreversibly inhibits fibroblast growth factor receptors (FGFR) 1-4, which are often deregulated in various cancers. This makes it particularly effective against tumors with FGFR genetic changes, offering a targeted approach that differs from traditional chemotherapy. ^{1 2 3 5 9}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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 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]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Futibatinib: First Approval. [2022]

2. England 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. [2022]

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

Futibatinib Is a Novel Irreversible FGFR 1-4 Inhibitor That Shows Selective Antitumor Activity against FGFR-Deregulated Tumors. [2021]

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

Futibatinib for FGFR2-Rearranged Intrahepatic Cholangiocarcinoma. [2023]

5. England pubmed.ncbi.nlm.nih.gov

Efficacy of futibatinib, an irreversible fibroblast growth factor receptor inhibitor, in FGFR-altered breast cancer. [2023]

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

FDA Approval Summary: Futibatinib for Unresectable Advanced or Metastatic, Chemotherapy Refractory Intrahepatic Cholangiocarcinoma with FGFR2 Fusions or Other Rearrangements. [2023]

7. United States 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. [2022]

8. United States 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. [2023]

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

Evaluation of the Mass Balance and Metabolic Profile of Futibatinib in Healthy Participants. [2023]