Futibatinib for Cancer

No longer recruiting at 91 trial locations

Overseen By Jill Kremer, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Taiho Oncology, Inc.

Must not be taking: FGFR inhibitors

Disqualifiers: Calcium-phosphorus disorders, Ectopic calcification, Retinal/corneal disorders, 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

Duration: Approximately 6 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness and safety of a drug called futibatinib (Lytgobi) for individuals with certain cancers that have specific changes in FGFR genes. The trial includes three groups: one for those with advanced solid tumors with FGFR1-4 changes, another for individuals with stomach or gastro-esophageal cancer with FGFR2 amplification (extra gene copies), and a third for blood cancers like myeloid or lymphoid neoplasms with FGFR1 changes. Participants should have previously tried other treatments without success and have measurable tumors. This trial offers a new treatment option for those meeting these criteria. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

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

Previous studies have shown that futibatinib has a safety profile that most people can tolerate for various tumor types. For instance, research in patients with advanced solid tumors found that side effects were mostly manageable and reversible with proper care, suggesting the treatment is generally well-tolerated.

In patients with stomach or gastroesophageal junction (GEJ) cancer, the treatment showed promising results, and side effects were considered acceptable. Additionally, studies involving patients with certain blood cancers indicated that futibatinib is well-tolerated and effective in other cancer types, such as bile duct cancer with specific genetic changes.

Overall, evidence suggests that futibatinib has a manageable safety profile across different cancer types, making it a potential option for those considering this treatment. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for cancer, which often involve chemotherapy or radiation, Futibatinib offers a targeted therapy option. It works by specifically inhibiting FGFR1-4, which are proteins involved in cancer cell growth and survival. This precision in targeting FGFR proteins means Futibatinib has the potential to effectively treat tumors that have these genetic alterations with potentially fewer side effects. Researchers are excited because this approach could lead to more personalized and effective cancer treatment strategies, particularly for patients with advanced or metastatic solid tumors or specific blood cancers.

What evidence suggests that futibatinib could be an effective treatment for cancer?

Research has shown that futibatinib yields promising results in treating various cancers with changes in a protein called FGFR. In this trial, participants with stomach or GEJ cancer in Cohort B will receive futibatinib. Studies found that 17.9% of these patients experienced tumor shrinkage with the treatment. Participants in Cohort A, who have solid tumors with FGFR changes, will also receive futibatinib, which has demonstrated the ability to reduce tumor size or slow their growth. For blood cancers, participants in Cohort C will receive futibatinib, and recent studies showed a 42% response rate, indicating significant tumor shrinkage in nearly half of the patients. Overall, futibatinib targets FGFR proteins, which play a role in cancer growth, and this targeted action appears effective in various cancer types. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for adults with advanced solid tumors, gastric or gastro-esophageal junction cancer, and certain blood cancers that have specific FGFR genetic changes. Participants must have measurable disease and should have tried standard treatments without success. They can't join if they've had prior FGFR inhibitor treatment or significant eye, mineral balance disorders, or unstable brain metastases.

Inclusion Criteria

I have had at least 2 treatments for my advanced cancer.

I cannot have a stem cell transplant or it didn't work for me, and no other treatments are suitable.

My stomach or GEJ cancer worsened after the last treatment.

Exclusion Criteria

My brain metastases are either untreated or not stable for over a month.

You have a medical condition related to the balance of calcium and phosphorus in your body that the doctor considers important.

I have been treated with an FGFR inhibitor before.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive futibatinib at an oral dose of 20 mg once a day on a continuous 28-day cycle until disease progression or unacceptable toxicity

Approximately 6 months

Follow-up

Participants are monitored for survival every 12 weeks until survival events have been reported for 75% of enrolled patients or the study is terminated

12 months

What Are the Treatments Tested in This Trial?

Interventions

• Futibatinib

Trial Overview The study tests the drug Futibatinib's effectiveness and safety in three groups: those with solid tumors (excluding primary brain tumors) having FGFR1-4 rearrangements; those with gastric cancer with FGFR2 amplification; and blood cancers with FGFR1 rearrangements.

How Is the Trial Designed?

Group I: Futibatinib (Cohort C) Experimental Treatment 1 Intervention

Participants with myeloid or lymphoid neoplasm harboring FGFR1 rearrangement were to receive futibatinib 20 mg, oral tablets, once a day on a continuous 28-day cycle until disease progression, unacceptable toxicity or other treatment discontinuation criteria are met.

Group II: Futibatinib (Cohort B) Experimental Treatment 1 Intervention

Participants with advanced or metastatic solid gastric or GEJ cancer harboring FGFR2 amplification received futibatinib 20 mg, oral tablets, once a day on a continuous 28-day cycle up to a maximum of 297 days.

Group III: Futibatinib (Cohort A) Experimental Treatment 1 Intervention

Participants with advanced or metastatic solid tumors harboring FGFR1-4 rearrangements received futibatinib 20 mg, oral tablets, once a day on a continuous 28-day cycle up to a maximum of 841 days.

Futibatinib is already approved in United States for the following indications:

Approved in United States as Lytgobi for:

• Treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements

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

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2023.41.16_suppl.3102

Efficacy and safety of futibatinib for refractory advanced ... The primary endpoint was met with five (19.2%; 95% CI, 6.6–39.4%) achieving a confirmed partial response (PR) in various cancer types (biliary ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39919334/

Phase 2 study of futibatinib in patients with gastric or ... Results: Among 28 treated patients, the ORR per independent central review was 17.9 %, comprising five patients with a partial response (median ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S0959804925000437

Phase 2 study of futibatinib in patients with gastric or ... In this phase 2 study, futibatinib demonstrated modest antitumor activity in patients with heavily pretreated gastric or GEJ cancer harboring ...

4. ejcancer.com ejcancer.com/article/S0959-8049(25)00043-7/pdf

Phase 2 study of futibatinib in patients with gastric or ... In subsequent phase 1 studies, futibatinib was shown to have a tolerable safety profile and antitumor activity in patients with advanced solid ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT04189445?term=AREA%5BBasicSearch%5D(FUTIBATINIB)&rank=2

Futibatinib in Patients With Specific FGFR Aberrations The purpose of this study is to evaluate the efficacy and safety of futibatinib in patients with FGFR aberrations in 3 distinct cohorts.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9899610/

Phase I study of the irreversible fibroblast growth factor ... This study showed preliminary antitumor activity of futibatinib by FGFR inhibition in patients with advanced solid tumors with FGF/FGFR gene abnormalities, ...

7. annalsofoncology.org annalsofoncology.org/article/S0923-7534(23)00624-5/fulltext

SO-10 Phase 2 study of futibatinib in patients with specific ... We present data for a cohort of patients with gastric or gastroesophageal junction (GEJ) cancer and high-level FGFR2 amplification treated with futibatinib.