Gilteritinib for Lung Cancer

Recruiting at 2 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University of Michigan Rogel Cancer Center

Must not be taking: FLT3 inhibitors, CYP3A inducers

Disqualifiers: Active CNS metastases, Child-Pugh C cirrhosis, others

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 21 days per cycle

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests gilteritinib, a drug already approved for leukemia, to determine its effectiveness for stage IV non-small cell lung cancer with an ALK-positive mutation. The goal is to assess the safety of gilteritinib and identify any side effects when used for lung cancer, particularly in patients whose cancer has resisted other treatments. This study targets individuals diagnosed with stage IV ALK-positive lung cancer who have tried other treatments and seek new options. As a Phase 1 trial, the research focuses on understanding how the treatment works in people.

Will I have to stop taking my current medications?

The trial requires that you stop taking any anti-cancer medications at least 7 days before starting gilteritinib. Additionally, you cannot take certain drugs that affect liver enzymes or specific receptors unless they are absolutely essential for your care.

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

Research has shown that gilteritinib is already approved by the FDA for treating a specific type of leukemia, indicating it has passed safety checks for that illness. In studies with leukemia patients, gilteritinib was generally well-tolerated, though some experienced side effects. Common issues included blood problems, such as low white blood cell counts and anemia (low red blood cell counts). Other side effects occurred but were less common.

For lung cancer, lab studies have demonstrated that gilteritinib can act against certain resistant cancer cells, suggesting it might help treat ALK-positive non-small cell lung cancer (NSCLC). However, using gilteritinib for lung cancer is new, so its safety for lung cancer patients remains under study. As this trial is in its early phase, it primarily focuses on determining the safety of gilteritinib for these patients and identifying the optimal dose. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Gilteritinib is unique because it targets specific genetic mutations that contribute to lung cancer progression, unlike traditional chemotherapy, which attacks all rapidly dividing cells. This precision makes it potentially more effective and less harmful to healthy cells. Researchers are excited about gilteritinib because it represents a new approach with the potential for fewer side effects and improved outcomes compared to standard treatments like surgery, radiation, and broad-spectrum chemotherapy.

What evidence suggests that gilteritinib might be an effective treatment for ALK positive non-small cell lung cancer?

Research has shown that gilteritinib, which participants in this trial will receive, has promising effects against ALK-positive non-small cell lung cancer (NSCLC). One study found that gilteritinib was more effective at fighting tumors than alectinib, a drug already used for this type of lung cancer. This suggests that gilteritinib might help treat cancer that no longer responds to other treatments. Although gilteritinib is currently approved for some types of leukemia, its ability to target lung cancer cells makes it a potential new option for patients with drug-resistant ALK-positive NSCLC. ^{2 5 6 7 8}

Who Is on the Research Team?

Angel Qin

Principal Investigator

University of Michigan Rogel Cancer Center

Are You a Good Fit for This Trial?

This trial is for adults with stage IV ALK positive non-small cell lung cancer (NSCLC) who have not responded to other treatments. Participants must be able to undergo procedures like biopsies and scans, and complete questionnaires.

Inclusion Criteria

AST (SGOT) ≤ 2.5 X ULN OR ≤ 5 X ULN for subjects with liver metastases

Ability to understand and sign a written informed consent

Measurable disease per RECIST v1.1 criteria

Exclusion Criteria

I have been treated with a FLT3 inhibitor before.

Mean triplicate screening EKG QTc > 480 ms

I received comfort-focused radiation therapy within the last week.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Patients receive gilteritinib PO QD on days 1-21 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.

21 days per cycle

Echocardiography, blood sample collection, CT scan, MRI, and potential tumor biopsy

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 2 years after last dose

Regular assessments for adverse events and tumor response

Long-term follow-up

Participants are monitored for progression-free survival and overall survival

Up to 2 years after last dose

What Are the Treatments Tested in This Trial?

Interventions

• Gilteritinib

Trial Overview The trial is testing the safety, side effects, and best dose of a drug called gilteritinib. This medication is approved for leukemia but will now be tested in patients with a specific type of resistant lung cancer.

How Is the Trial Designed?

Group I: Treatment (gilteritinib) Experimental Treatment 7 Interventions

Patients receive gilteritinib PO QD on days 1-21 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients also undergo echocardiography at screening and blood sample collection, CT scan and MRI at screening and on study. Additionally, patients may undergo a tumor biopsy pre-treatment and at end of treatment.

Gilteritinib is already approved in United States, European Union, Japan for the following indications:

Approved in United States as Xospata for:

• Acute myeloid leukemia (AML) with FLT3 mutation

Approved in European Union as Xospata for:

• Acute myeloid leukemia (AML) with FLT3 mutation

Approved in Japan as Xospata for:

• Acute myeloid leukemia (AML) with FLT3 mutation

Who Is Running the Clinical Trial?

University of Michigan Rogel Cancer Center

Lead Sponsor

Trials

303

Recruited

20,700+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10637052/

Efficacy of gilteritinib vs alectinib in ALK+ NSCLC In conclusion, gilteritinib demonstrated significantly improved antitumor efficacy compared with alectinib against ALK‐rearranged NSCLC cells, ...

2. xospatahcp.com xospatahcp.com/efficacy

ADMIRAL Trial: XOSPATA® (gilteritinib) Overall Survival Graph of overall survival in the ADMIRAL trial. 36% reduced risk of death with. Median overall survival was based on Kaplan-Meier estimates2 ...

3. alkpositive.org alkpositive.org/blog/gilteritinib-for-the-treatment-of-alk-positive-non-small-cell-lung-cancer-nsclc/

Gilteritinib Trial for ALK+ NSCLC After TKI & Lorlatinib This phase I trial will study the safety, side effects, and best dose of gilteritinib in treating patients with stage IV ALK NSCLC.

4. nature.com nature.com/articles/s41419-024-07082-x

Cholesterol inhibition enhances antitumor response of gilteritinib The results showed that 1 mg/kg gilteritinib did not hinder the growth of lung cancer xenografts, and 5 mg/kg gilteritinib significantly ...

5. oncpracticemanagement.com oncpracticemanagement.com/special-issues/2019-fourth-annual-oncology-guide-to-new-fda-approvals/xospata-gilteritinib-first-drug-approved-as-monotherapy-for-adults-with-relapsed-or-refractory-acute-myeloid-leukemia-with-flt3-mutation

Xospata (Gilteritinib) First Drug Approved as Monotherapy ... Among the 292 patients who received gilteritinib, 41% were aged ≥65 years. The efficacy and safety were similar in older and younger patients.

6. xospatahcp.com xospatahcp.com/safety

XOSPATA® (gilteritinib) Safety & Adverse Reactions Review the safety profile of XOSPATA in the treatment of relapsed or refractory FLT3m+ AML. See Important Safety Information including BOXED WARNING.

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2019/211349s001lbl.pdf

Xospata - accessdata.fda.gov The safety profile of XOSPATA is based on 319 patients with relapsed or refractory AML treated with gilteritinib. 120 mg daily in three clinical trials. The ...

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8506653/

FDA Approval Summary: Gilteritinib for relapsed or refractory ... Safety analysis showed higher risk of both all grades and grade 3 or higher hematological (neutropenia, anemia, and thrombocytopenia) and non-hematological ( ...