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A Study of Deferoxamine (DFO) in People With Leptomeningeal Metastasis

ConditionLeptomeningeal Metastases
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorMemorial Sloan Kettering Cancer Center

About this trial

The researchers are doing this study to find out whether deferoxamine (DFO) given intrathecally (directly into the CSF) is a safe treatment for people with leptomeningeal metastasis from solid tumor cancer. The researchers will test different doses of DFO to find the highest dose that causes few or mild side effects. When the dose is found, they will test it in future participants to see whether DFO is a safe and effective treatment for people with leptomeningeal metastasis from solid tumor malignancies. They are also doing this study to see how the body absorbs, distributes, gets rid of, and responds to DFO.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age ≥ 18 years on the day of consenting to study

ECOG performance status ≤ 2 or KPS ≥ 60.

Life expectancy ≥ 8 weeks in the opinion of the Investigator

Newly diagnosed: As evidenced by positive CSF cytology, CTC count >3.0/3.0 mL, or unequivocal radiographic evidence of LM on contrast-enhanced MRI, OR

Disqualifiers

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

Patients receiving other therapy (either intrathecal or systemic) designed to treat their LM, with ongoing acceptable control of their LM.

Any contraindication to gadolinium-enhanced MRI

Use of any systemic iron chelators within 4 weeks of first dose

Trial design

Design model

Single group

Treatments tested in this trial

  • Deferoxamine (DFO)

    Drug

    The accelerated single-patient dose escalation will apply to dosing cohorts 1 through 4 (10mg, 30mg, 60mg, 100mg) and will convert to a 3+3 dose escalation for cohorts 5 through 9 (150mg, 210mg, 280mg, 372mg, 495mg).

Treatment groups

32 Participants
are divided into 1 treatment group
Group A: Deferoxamine (DFO)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Patients are considered evaluable for the primary safety endpoint of DLT if they receive at least one full cycle (twice weekly dosing for 4 weeks) without a DLT or if they experience a DLT at any time during the first cycle of IT-DFO. Per CTCAE version 5.0

Time frame
1 year
2

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

Per CTCAE version 5.0

Time frame
1 year

Secondary outcomes

1

objective response rate (ORR)

LM objective response rate (ORR) is defined as the proportion of patients with at least one objective response in LM, using a combined approach taking into account radiographic, neurologic and cytologic assessments based on RANO-LM.

Time frame
1 year

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Memorial Sloan Kettering Cancer Center

Lead sponsor

Center for Experimental Therapeutics

Collaborator

F.M. KIRBY FOUNDATION

Collaborator

Source ClinicalTrials.gov
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