What side effects are associated with this type of intervention?

Common treatments for metastases include chemotherapy, targeted therapy, and immunotherapy. Chemotherapy can cause side effects such as nausea, fatigue, hair loss, and increased risk of infection. Targeted therapies, like those using monoclonal antibodies, may lead to skin reactions, infusion-related reactions, and gastrointestinal issues. Immunotherapy can result in immune-related adverse effects like colitis, pneumonitis, and thyroid dysfunction. For iron chelation therapy, such as Deferoxamine (DFO), side effects may include injection site reactions, visual and auditory disturbances, and allergic reactions. It is important to discuss these potential side effects with a healthcare provider to manage and mitigate them effectively.

What prior FDA approvals exist?

The FDA has approved various treatments for metastases, including chemotherapy agents like cisplatin and doxorubicin, targeted therapies such as bevacizumab and aflibercept, and immunotherapies like nivolumab and pembrolizumab. These treatments aim to control tumor growth, improve survival rates, and manage symptoms. While Deferoxamine (DFO) is being studied for its potential in treating leptomeningeal metastasis through iron chelation, there are no current FDA-approved iron chelation therapies specifically for metastases.

What other types of research exist?

Promising novel alternatives to Deferoxamine (DFO) for metastases patients include: 1) Selpercatinib and Pralsetinib, which are selective RET inhibitors showing efficacy in RET-altered thyroid cancers and other RET-driven malignancies. 2) Lutetium-177-PSMA-617, a radioligand therapy for metastatic castration-resistant prostate cancer. 3) Futibatinib, an FGFR-selective tyrosine kinase inhibitor for advanced cholangiocarcinoma with FGFR2 fusions/rearrangements. These therapies represent significant advancements in targeted treatment options for metastatic cancer patients.

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Iron Chelator

Deferoxamine for Meningeal Carcinomatosis

Phase 1

Recruiting

Led By Adrienne Boire, MD

Research Sponsored by Memorial Sloan Kettering Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age ≥ 18 years on the day of consenting to study

ECOG performance status ≤ 2 or KPS ≥ 60

Must not have

Any CNS-directed irradiation within 7 days of first dose of IT-DFO

Patients not allowed to receive whole-brain radiation therapy or craniospinal radiation therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether injecting deferoxamine into the fluid around the brain and spinal cord is safe for people with cancer that has spread there. It focuses on patients with non-small cell lung cancer. The drug works by reducing the iron that cancer cells need to grow. Deferoxamine is an iron chelator approved by the FDA, commonly used to treat iron-overload diseases and has shown anticancer activity by depleting cancer cells of iron.

Who is the study for?

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.

What is being tested?

Researchers are testing Deferoxamine (DFO) given directly into the cerebrospinal fluid (CSF) to find a safe dose for treating leptomeningeal metastasis from solid tumor cancers. They aim to determine how DFO affects the body and its safety and effectiveness against non-small cell lung cancer-related metastasis.

What are the potential side effects?

Potential side effects may include reactions related to intrathecal administration like headache, infection risk at the Ommaya reservoir site, possible changes in blood counts due to bone marrow suppression, and other typical drug-related adverse events such as nausea or allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 years old or older.

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I can take care of myself but might not be able to do heavy physical work.

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My cancer, including lung cancer, has spread to the lining of my brain or spinal cord.

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My cancer has spread to my brain but is stable elsewhere.

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My cancer has spread to my brain and other parts of my body.

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I have a working Ommaya reservoir installed for treatment.

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My cancer is a solid tumor and it started in my lungs.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have not had brain radiation within the last week.

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I have not undergone whole-brain or craniospinal radiation therapy.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Frequency of dose-limiting toxicities (DLTs) during Phase Ia (Primary safety endpoint during dose-finding phase)

Frequency of dose-limiting toxicities (DLTs) during Phase Ib (RP2D of IT-DFO in patients with LM from NSCLC)

Secondary study objectives

objective response rate (ORR)

Side effects data

From 2008 Phase 2 trial • 212 Patients • NCT00110617

27%

Headache

16%

Vomiting

13%

Upper respiratory tract infection

13%

Cough

13%

Pyrexia

11%

Abdominal pain

11%

Sickle cell anaemia with crisis

Diarrhoea

Oropharyngeal pain

Pruritus

Chest pain

Nausea

Nasal congestion

Insomnia

Abdominal pain upper

Back pain

Pain in extremity

Dizziness

Rash

Injection site pain

Nasopharyngitis

Acute chest syndrome

Cellulitis

Constipation

Humerus fracture

Hypoxia

Haemoglobin decreased

Cholelithiasis

Implant site reaction

Device malfunction

Asthenia

Clavicle fracture

Pharyngitis streptococcal

Injury

Procedural pain

Neck pain

Cerebral ischaemia

Pneumonia aspiration

Arthralgia

Photophobia

Catheter site pain

Catheter site infection

Viral infection

Fibula fracture

Patella fracture

Chills

100%

80%

60%

40%

20%

Study treatment Arm

Period 1 Deferoxamine (DFO)

Period 2 Deferasirox (ICL670)

Period 1 Deferasirox (ICL670)

Period 2 Deferoxamine (DFO) Then ICL670

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Deferoxamine (DFO)Experimental Treatment1 Intervention

This study is an open-label, non-randomized, single-center, dose escalation phase 1a study of intrathecal deferoxamine (IT-DFO) in patients with leptomeningeal metastases (LM) from solid tumor malignancies, followed by a phase 1b dose expansion cohort at the recommended phase 2 dose (RP2D) in patients with LM from solid tumor malignancies. Study objectives will include safety (1a/1b), pharmacokinetics (PK) and pharmacodynamics (PD) of IT-DFO (1a), and preliminary anti-tumoral efficacy in patients with LM solid tumor malignancies (1b).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Deferoxamine (DFO)

2023

Completed Phase 2

~360

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for metastases include chemotherapy, targeted therapy, and iron chelation. Chemotherapy works by killing rapidly dividing cancer cells, which can reduce tumor burden and alleviate symptoms. Targeted therapies, such as tyrosine kinase inhibitors, specifically target molecular pathways involved in cancer growth and spread, offering a more precise approach with potentially fewer side effects. Iron chelation, like Deferoxamine (DFO), removes excess iron from the body, which is crucial because some cancers rely on iron for growth and proliferation. Understanding these mechanisms helps in selecting appropriate treatments to manage metastases effectively, improving patient outcomes and quality of life.

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