Intrathecal methotrexate-loaded tumor-derived vesicles for leptomeningeal lung cancer

A Phase I/II, Single-arm, Single-center Clinical Study: Exploration of the Application of Methotrexate-loaded Drug Vesicles in Intrathecal Injection for Meningeal Metastasis of Lung Cancer

Phase1; Phase2 Interventional Henan Cancer Hospital · NCT07559097

This treatment will try giving methotrexate-loaded tumor-derived vesicles into the spinal fluid to see if it is safe and helps adults with leptomeningeal metastasis from lung cancer who have not responded to standard treatments.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment14 (estimated)
Ages18 Years and up
SexAll
SponsorHenan Cancer Hospital Government
Drugs / interventionsmethotrexate
Locations1 site (Zhengzhou, Henan)
Trial IDNCT07559097 on ClinicalTrials.gov

What this trial studies

This is a single-center, open-label Phase I/II trial testing intrathecal methotrexate-loaded autologous tumor cell-derived microparticles (MTX-MPs) in patients with cytologically confirmed leptomeningeal metastasis from lung cancer who have failed standard care. Phase I uses an accelerated titration combined with a 3+3 dose-escalation design to define the maximum tolerated dose (MTD) and recommended Phase II dose (RP2D), and Phase II measures objective response rate (ORR) at the RP2D. About 10–20 adults (age ≥18, ECOG PS 0–3) will receive MTX-MPs on days 1, 3, and 5 of the first cycle and then every three weeks until disease progression. Key secondary endpoints include progression-free survival, overall survival, and a detailed safety profile.

Who should consider this trial

Good fit: Adults (≥18 years) with pathologically confirmed lung cancer and cytology-proven leptomeningeal metastasis who have failed standard treatments and have ECOG performance status 0–3 are the intended participants.

Not a fit: Patients with active central nervous system infections, severe immunodeficiency, ECOG ≥4, current participation in other interventional studies, or other conditions judged by investigators to preclude safe participation are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, this approach could improve control of leptomeningeal disease and neurological symptoms by delivering higher methotrexate concentrations into the cerebrospinal fluid with potentially less systemic toxicity.

How similar studies have performed: While intrathecal methotrexate has been used historically for leptomeningeal disease, delivery via methotrexate-loaded autologous tumor cell-derived microparticles is a novel method with limited prior clinical data.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

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Participants can be enrolled in this study only if they meet all of the following inclusion criteria:

1. Age ≥ 18 years old;
2. Diagnosed with lung cancer by pathological biopsy or cytology;
3. Diagnosed with meningeal metastasis by detecting tumor cells in cerebrospinal fluid cytology;
4. Patients with advanced lung cancer and meningeal metastasis who have failed standard treatment;
5. ECOG PS 0 - 3.

Exclusion Criteria:

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The following conditions will disqualify a patient from participating in this study:

1. Concurrent central nervous system infectious diseases;
2. ECOG PS ≥ 4;
3. Patients currently participating in other interventional studies;
4. History of or current severe immunodeficiency diseases;
5. Any other conditions that, in the judgment of the researcher, make the patient unfit to participate in this study.

Where this trial is running

Zhengzhou, Henan

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Leptomeningeal Metastasis From Lung Cancer
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.