NCT05184816

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

A Phase 1a/1b Trial of Intrathecal Deferoxamine for Leptomeningeal Metastases

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 35 (estimated)
• Sponsor: Memorial Sloan Kettering Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

Phase 1a During the phase 1a arm, the MTD and PK/PD data will be evaluated in patients with LM from any solid tumor malignancy in an accelerated dose escalation fashion, with conversion to a traditional 3 + 3 dose escalation scheme at either dosing cohort 5 or when alternative criteria is met \[either 1 patient experiences a DLT or 2 patients experience any grade 2 or higher nervous system disorder toxicity (except headache)\]. All patients will receive IT-DFO via Ommaya reservoir 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. DLTs and new grade 2 or higher nervous system toxicities will be assessed for the first 28 days of each cohort. The Principal Investigator will consider the MTD determined by the dose escalation, any cumulative or delayed CNS toxicities (if present), and PK/PD data of phase 1a when determining the RP2D of phase 1b. Phase 1b The phase 1b dose expansion will be determined by the RP2D of the phase 1a arm. All patients will receive IT-DFO via Ommaya reservoir 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. Patients in phase 1b will also be assessed for early efficacy endpoints.

Conditions

• Leptomeningeal Metastases

Interventions

Type	Name	Description
DRUG	Deferoxamine (DFO)	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).

Timeline

• Start date: 2021-12-22
• Primary completion: 2026-12-01
• Completion: 2026-12-01
• First posted: 2022-01-11
• Last updated: 2026-03-27

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study