A Phase 1/2 interventional study of Toripalimab, pemetrexed in Leptomeningeal Metastasis, sponsored by Guangzhou Medical University. Active, not recruiting at 5 sites in China. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-09-24.
Sponsored by Guangzhou Medical University · Phase 1/2, Interventional, and Treatment
This is an open-label, single-arm, phase I/II trial of immune checkpoint inhibitor combined with pemetrexed intrathecal injection for leptomeningeal metastasis from solid tumors.
This is an open-label, single-arm, phase I/II trial of immune checkpoint inhibitor combined with pemetrexed intrathecal injection for leptomeningeal metastasis from solid tumors. The primary objective was to assess recommended dose and safety based on adverse events (AEs). All participants were observed to evaluate the clinical response rate (CRR), disease control rate (DCR) and overall survival (OS). Patients underwent cerebrospinal fluid (CSF) and blood specimen collection to evaluate potential clinical, molecular, and/or immune predictors of treatment efficacy and safety.
120 studies on the registry are indexed under Meningeal Carcinomatosis; 42 are open to participants now.
This study's enrollment of 45 is above the median of 30 across 106 interventional studies indexed under Meningeal Carcinomatosis.
Browse Meningeal Carcinomatosis studies →Guangzhou Medical University is the lead sponsor of 38 studies on the registry; 20 are open to participants now.
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Exclusion Criteria:
Immune checkpoint inhibitor combined with pemetrexed
Drug: Toripalimab, pemetrexed
Drug 1: Toripalimab 40 mg; Drug 2: pemetrexed 15mg. intrathecal injection therapy
Recommended dose
The recommended dose of intrathecal PD-1 inhibitor. The dose limiting toxicity was defined as ≥ grade 3 neurological toxicities (e.g., chemical meningitis) or other grade 4 toxicity.
Time frame: The dose limiting toxicity will be evaluated during the first 6 weeks of study treatment (the DLT window).
Incidence of treatment-related adverse events
The incidence of treatment-related adverse events were measured for determining tolerability and safety. Adverse events (AEs) are evaluated according to the Common Terminology Criteria for Adverse Events (CTCAE). Events of grade 3-5 are defined as moderate and severe adverse events.
Time frame: From date of treatment until discontinuation of study treatment.
Clinical response rate
The response assessment in neuro-oncology criteria (RANO) proposal for response criteria of leptomeningeal metastasis was used to assess the clinical response in this study.
Time frame: From date of treatment until the date of first documented progression or date of death from any cause, whichever came first.
Progression-free survival related to leptomeningeal metastasis (LMPFS)
LMPFS was defined as time from the enrollment until leptomeningeal metastasis progression or death, whichever occurred first. The leptomeningeal metastasis progression was determined based on the RANO proposal evaluation criteria which have been established and published on Neuro Oncol.
Time frame: From enrollment to LM progression or death, whichever occurred first, or the last follow-up (at least 7 months).
Overall survival(OS)
Survival time was recorded since the date of patient enrollment. All patients were followed up until death or the end of the study.
Time frame: From the enrollment of this study until date of death from any cause, whichever came first, or the last follow-up (at least 7 months).
Disease control rate
Disease control rate is defined as the proportion who achieve an leptomeningeal metastasis response or stable disease.
Time frame: From date of treatment until the date of first documented progression or date of death from any cause, whichever came first.
Plan to share: Yes — All individual participant data that underlie results in a publication will be available to other researchers.
Supporting information: Study protocol, Sap
This study is active, not recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.
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Guangzhou Medical University