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NCT Number: NCT07559097

Exploration of the Application of Methotrexate-loaded Drug Vesicles in Intrathecal Injection for Meningeal Metastasis of Lung Cancer

This is a phase I/II, single-arm, open-label, single-center clinical trial to evaluate the safety, tolerability, and preliminary efficacy of intrathecal injection of methotrexate-loaded autologous tumor cell-derived microparticles (MTX-MPs) in patients with leptomeningeal metastasis from lung cancer who have failed standard of care.

The study consists of two phases: Phase I employs an accelerated titration combined with a "3+3" dose-escalation design to determine the maximum tolerated dose (MTD) and the recommended phase II dose (RP2D). Phase II further assesses the objective response rate (ORR) at the RP2D. Key secondary endpoints include progression-free survival (PFS), overall survival (OS), and safety profile.

Approximately 10-20 patients with cytologically confirmed leptomeningeal metastasis (age ≥18 years, ECOG PS 0-3) will be enrolled. Participants will receive intrathecal MTX-MPs on days 1, 3, and 5 of the first cycle, followed by once every 3 weeks (Q3W) until disease progression, unacceptable toxicity, or death.

Tumor response will be evaluated using the Response Assessment in Neuro-Oncology (RANO) criteria for leptomeningeal metastasis, and adverse events will be graded according to CTCAE version 5.0. This exploratory study may provide a novel local therapeutic approach for leptomeningeal metastasis from lung cancer.

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Key information

Conditions

Leptomeningeal Metastasis From Lung Cancer

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

-

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

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

Exclusion criteria

-

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

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

Treatment and study plan

Methotrexate-loaded vesicles

Biological

Intrathecal injection of methotrexate-loaded vesicles

Primary outcomes

  1. Phase I: Maximum Tolerated Dose (MTD) and Recommended Phase II Dose (RP2D)

    Time frame: Expected for 15 months

    Phase I: To determine the MTD and RP2D using an accelerated titration plus 3+3 dose-escalation design.

  2. Phase II:Objective Response Rate(ORR)

    Time frame: Expected for 15 months

    Phase II: Objective Response Rate (ORR) per RANO criteria for leptomeningeal metastasis, defined as the proportion of patients achieving complete response (CR) or partial response (PR).

Secondary outcomes

  1. Safety and Tolerability

    Time frame: From first dose up to 30 days after last dose (or study completion)

    Incidence, severity, and causality of adverse events (AEs) graded by CTCAE v5.0, including acute reactions within 3 days after each intrathecal injection, neurological toxicities, and laboratory abnormalities.

  2. Progression-Free Survival (PFS)

    Time frame: From first dose up to approximately 15 months

    Time from first dose of study drug to first documented disease progression per RANO criteria for leptomeningeal metastasis, or death from any cause, whichever occurs first.

  3. Overall Survival (OS)

    Time frame: From first dose up to approximately 27 months.

    Time from first dose of study drug to death from any cause.

Sponsors and collaborators

Lead sponsor

Henan Cancer Hospital

Other Gov

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2026
Study completion
2027
First posted
Apr 30, 2026
Registry last updated
Apr 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.