Active Surveillance for Brain Cancer

(ACTION-Brain Trial)

Overseen By Christopher Anker, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Vermont Medical Center

Must be taking: Immunotherapy, Targeted therapy

Disqualifiers: Pregnancy, Leptomeningeal 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

Compensation: Varies

Number of Visits: 4

45 Participants Needed

Trial Summary

What is the purpose of this trial?

This pilot pragmatic trial evaluates the feasibility of avoiding radiation therapy in patients with brain metastases who demonstrate an intracranial response to systemic therapy-including immunotherapy, targeted therapy, and/or chemotherapy. The study will prospectively enroll 45 patients, divided into two cohorts: 30 with non-small cell lung cancer (NSCLC) receiving immunotherapy, and 15 with brain metastases from other solid tumors. Eligible participants must have at least one brain metastasis not planned for radiation or surgery and must be initiating or planning to initiate a systemic therapy regimen expected to penetrate the blood-brain barrier and achieve intracranial activity. All patients will undergo a re-evaluation brain MRI 4-8 weeks after initiating systemic therapy. If lesions are stable or regressing, patients will continue surveillance without radiation. If progression is noted, standard-of-care radiation may be administered at the discretion of the treating physician. The primary objective is to assess 6-month radiation therapy-free survival (RTFS) in NSCLC patients based on PD-L1 expression status. Secondary endpoints include intracranial progression-free survival, overall survival, radiation necrosis rate, and quality of life. This study seeks to inform future trial design and identify patients who may safely avoid brain radiation.

Research Team

Randall Holcombe, MD, MBA

Principal Investigator

University of Vermont Cancer Center

Eligibility Criteria

This trial is for patients with brain metastases from lung cancer or other solid tumors, who are starting treatments expected to cross into the brain. They must have at least one brain tumor not scheduled for radiation/surgery and be able to undergo MRI scans.

Inclusion Criteria

I was diagnosed with a solid tumor cancer within the last 5 years.

Ability to obtain MRI head scans with contrast

Ability to understand and sign a written informed consent

Exclusion Criteria

Pregnant or nursing individuals

Serious medical comorbidities that would prevent study participation

I haven't started any new cancer treatments in the last 6 months.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Systemic Therapy Initiation

Participants initiate systemic therapy expected to penetrate the blood-brain barrier and achieve intracranial activity

4-8 weeks

1 visit (in-person) for MRI re-evaluation

Surveillance

Participants undergo structured surveillance with MRIs every 3 months to monitor brain metastases

Up to 2 years

MRI every 3 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

• Active Surveillance

Trial Overview The study tests if certain patients can skip radiation therapy after their brain tumors respond well to systemic therapies like immunotherapy, targeted therapy, or chemotherapy. It involves regular MRIs to monitor tumor response.

Participant Groups

Group I: Systemic Therapy With Surveillance for Brain Metastases Experimental Treatment 1 Intervention

Participants with brain metastases from solid tumors will receive standard-of-care systemic therapy expected to have intracranial activity, such as immunotherapy, targeted therapy, or anti-HER2 agents. A re-evaluation brain MRI will be performed 4-8 weeks after starting therapy. If lesions are stable or responding, patients will continue on systemic therapy with MRI surveillance every 3 months. Radiation therapy may be administered only if progression is observed. All participants are managed according to this pathway, regardless of primary tumor type.

Who Is Running the Clinical Trial?

University of Vermont Medical Center

Lead Sponsor

Trials

46

Recruited

25,900+