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Promising drug combo targets Hard-to-Treat brain cancers

NCT ID NCT07468136

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests two drugs—retifanlimab (an immunotherapy) and DFMO (a growth blocker)—alone or together in 33 adults with progressive high-grade glioma, an aggressive brain cancer. The goal is to find the safest, most effective dose and see if the combination boosts the immune system's attack on tumors. Participants will undergo surgery, MRI scans, and blood/CSF collection as part of the trial.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
retifanlimab and difluoromethylornithine (DFMO)
What this could lead to
If successful, this could provide a new, more effective treatment option for people with progressive high-grade glioma, potentially slowing tumor growth.
What could go wrong
This is an early-phase trial with only 33 participants, so results may not apply to all patients. The combination therapy may cause side effects or fail to improve outcomes.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 33 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Oct 2030

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Age ≥ 18 years * Diagnosis of high-grade glioma, including any of the following: * Glioblastoma, IDH-wild type (WT) * Grade 3 or 4 IDH1/2 mutant astrocytoma or * Grade 3 oligodendroglioma * Any prior grade 2 astrocytoma or oligodendroglioma that is suspected to have recurred at a higher grade * Other high-grade glioma * Plan for surgical resection as part of routine clinical care * Radiographic disease progression, with or without tissue confirmation * Measurable disease * Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1 or 2 and Karnofsky Performance Status (KPS) ≥ 60 * NOTE: PS must be assessed (again) within 7 days prior to first dose of study drug * Hemoglobin ≥ 9.0 g/dL (obtained ≤ 15 days prior to registration) * Absolute neutrophil count (ANC) ≥ 1500/mm\^3 (obtained ≤ 15 days prior to registration) * Platelet count ≥ 100,000/mm\^3 (obtained ≤ 15 days prior to registration) * Total bilirubin ≤ 1.5 x upper limit of normal (ULN) (obtained ≤ 15 days prior to registration) * Alanine aminotransferase (ALT) and aspartate transaminase (AST) ≤ 3 x ULN (≤ 5 x ULN for patients with liver involvement) (obtained ≤ 15 days prior to registration) * Calculated creatinine clearance ≥ 45 ml/min using the Cockcroft-Gault formula (obtained ≤ 15 days prior to registration) * Negative pregnancy test done ≤ 7 days prior to registration, for persons of childbearing potential only * Provide written informed consent for the current study * Willing to provide consent for the Neuro-oncology biorepository (IRB 12-003458) for archiving of tissue, cerebrospinal fluid (CSF), and/or blood samples * Ability to complete forms by themselves or with assistance * Willing to return to enrolling institution for follow-up (during the Active Monitoring Phase of the study) Exclusion Criteria: * Any of the following because this study involves an investigational agent, the genotoxic, mutagenic, and teratogenic effects of which on the developing fetus and newborn are unknown: * Pregnant persons * Nursing persons * Persons of childbearing potential or able to father a child who are unwilling to employ adequate contraception * Uncontrolled intercurrent illness that by the judgement of the investigator would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the regimens including, but not limited to: * ongoing or active infection (e.g., pneumonia, sepsis, etc.) requiring systemic therapy * current diagnosis or previous history of immune-related (non-infectious) pneumonitis or interstitial lung disease that requires or required steroids * active autoimmune disease that required systemic treatment other than replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroids) ≤ 2 years prior to registration * symptomatic congestive heart failure * unstable angina pectoris * psychiatric illness/social situations that would limit compliance with study requirements (e.g., drug addiction) * concurrent active Hepatitis B (defined as hepatitis B surface antigen \[HBsAg\] positive and/or detectable hepatitis B virus \[HBV\] deoxyribonucleic acid \[DNA\]) and Hepatitis C virus (defined as anti-hepatitis C virus \[HCV\] antibody \[Ab\] positive and detectable HCV ribonucleic acid \[RNA\]) infection EXCEPTIONS: * Patients with evidence of hepatitis B virus (HBV) infection (HBsAg positive) must have completed at least 4 weeks of HBV antiviral therapy, and the HBV viral load must be undetectable at the time of registration * Patients with a history of hepatitis C virus (HCV) are eligible if they have an undetectable HCV viral load. Patients must have completed curative anti-viral treatment ≥ 4 weeks prior to registration. * NOTE: Patients without symptoms or prior history do not require testing prior to registration * Co-morbid systemic illnesses or other severe concurrent disease that would make the patient inappropriate for entry into the study or interfere with proper assessment of safety and toxicity * History of myocardial infarction ≤ 6 months prior to registration or congestive heart failure requiring use of ongoing maintenance therapy for life-threatening ventricular arrhythmias * Active autoimmune disease that has required systemic treatment (other than replacement therapy) ≤ 1 year prior to registration * History of allogeneic stem cell transplant * Receiving any other investigational agent with therapeutic intent * Participants who are unable to swallow the DFMO solution or who are at risk for impaired absorption of oral medication. * NOTE: This restriction includes, but is not limited to, refractory vomiting, gastric resection/bypass, and duodenal/jejunal resection * Patients with known hypersensitivity or allergy to DFMO or retifanlimab * Contraindication to MRI or administration of gadolinium

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    1 site. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Mayo Clinic in Rochester

    RECRUITING

    Rochester, Minnesota, 55905, United States

    Contact Email: •••••@•••••

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