Deferoxamine for Meningeal Carcinomatosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores whether deferoxamine (DFO), administered directly into the cerebrospinal fluid (CSF), is a safe treatment for individuals with leptomeningeal metastasis, where cancer spreads to the membranes surrounding the brain and spinal cord. Researchers will test various doses to identify the safest one with minimal side effects. The trial targets those with leptomeningeal metastasis from solid tumors, whether newly diagnosed or persistent, who may benefit from additional treatment. Participants should have a functioning Ommaya reservoir (a device for delivering medication to the CSF) or be willing to have one placed. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop all current medications, but you cannot use systemic iron chelators within 4 weeks of the first dose or ascorbic acid or prochlorperazine within 2 weeks of the first dose. If you are on a systemic treatment controlling your extracranial disease, you may continue it during the study.
Is there any evidence suggesting that deferoxamine (DFO) is likely to be safe for humans?
Research has shown that deferoxamine (DFO) has been studied for safety when administered directly into the cerebrospinal fluid (CSF), the fluid surrounding the brain and spinal cord. This method is crucial for treating leptomeningeal metastasis, cancer that has spread to these areas. Since the 1970s, many studies have explored administering treatments like DFO directly into the CSF to find effective ways to treat cancers that spread to the brain and spinal cord.
Although specific data on DFO's safety in these studies is limited, its testing in human trials suggests researchers see potential in it. Early trials aim to find a safe dose and observe the body's response. Testing in humans typically indicates that earlier research has shown it might be safe enough to proceed. This trial is in its early stages, focusing on ensuring the treatment's safety and determining the right dose.12345Why do researchers think this study treatment might be promising?
Deferoxamine (DFO) is unique because it targets leptomeningeal metastases (LM) with a new delivery method: intrathecal administration, which means it is delivered directly into the cerebrospinal fluid. This approach allows the drug to reach cancer cells more directly and effectively than traditional systemic treatments like chemotherapy, which can struggle to penetrate the central nervous system. Researchers are excited about DFO because its mechanism involves chelating iron, which is crucial for tumor growth, potentially slowing down or stopping the progression of cancer cells in the meninges.
What evidence suggests that deferoxamine might be an effective treatment for leptomeningeal metastasis?
Research suggests that deferoxamine (DFO), the treatment under study in this trial, might help treat leptomeningeal metastasis, a condition where cancer spreads to the protective layers around the brain and spinal cord. DFO removes extra iron from the body, potentially slowing cancer growth since cancer cells need iron to spread. Early findings indicate that reducing iron might help control tumors, but specific results for DFO in this condition remain under investigation. Although limited data exists on DFO's effectiveness, its mechanism shows promise for affecting tumor growth in the brain and spine area. Further studies will determine if DFO is truly effective for this purpose.12567
Who Is on the Research Team?
Jessica Wilcox, MD
Principal Investigator
Memorial Sloan Kettering Cancer Center
Are You a Good Fit for This Trial?
This trial is for adults over 18 with leptomeningeal metastasis from solid tumors or NSCLC, who have a life expectancy of at least 8 weeks and can use effective contraception. They must be stable enough not to need immediate brain metastases treatment, able to handle an Ommaya reservoir (a device placed in the brain for drug delivery), and have normal CSF flow.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Phase 1a Treatment
Participants receive intrathecal deferoxamine (IT-DFO) via Ommaya reservoir in an accelerated dose escalation fashion, with conversion to a traditional 3 + 3 dose escalation scheme. Dosing is twice per week during cycle 1, once per week during cycle 2, and once every 2 weeks for subsequent cycles until LM progression, intolerable toxicity, or death.
Phase 1b Treatment
Participants receive IT-DFO at the recommended phase 2 dose (RP2D) determined from phase 1a. Dosing is twice per week during cycle 1, once per week during cycle 2, and once every 2 weeks for subsequent cycles until LM progression, intolerable toxicity, or death. Early efficacy endpoints are assessed.
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Deferoxamine (DFO)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Memorial Sloan Kettering Cancer Center
Lead Sponsor
Center for Experimental Therapeutics
Collaborator
F.M. KIRBY FOUNDATION
Collaborator