A Phase 2 interventional study of Intrathecal Administration of Thiotepa in Combination with Trastuzumab via the Ommaya Reservoir in Leptomeningeal Metastasis of HER2-positive Breast Cancer, sponsored by The First Affiliated Hospital with Nanjing Medical University. Recruiting at 1 site in China. Open to female participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2025-12-04.
Sponsored by The First Affiliated Hospital with Nanjing Medical University · Phase 2, Interventional, and Treatment
Evaluate the efficacy and safety of Intrathecal Administration of Thiotepa in Combination with Trastuzumab via the Ommaya Reservoir in Breast Cancer with Leptomeningeal Metastasis
This was a II, single-arm, prospective, multicenter study designed to estimate the efficacy and safety of intrathecal administration of thiotepa in combination with Trastuzumab via the Ommaya Reservoir in HER2-positive breast cancer with leptomeningeal metastasis.
The primary end point was iORR [complete response (CR) + partial response (PR)] according to RANO-LM. Scoring based on radiographic assessment in leptomeningeal metastases . A composite score (total score) is calculated and compared with the baseline total score. A 25% worsening in the current score relative to baseline defines radiographic progressive disease. A 50% improvement in the current score defines a radiographic partial response. Resolution of all baseline radiographic abnormalities defines a complete response. All other situations define stable disease. The secondary end points were changes in iPFS,PFS, OS, and exploratory analysis of the relationship between molecular markers and therapeutic efficacy.
This study is planned to include 26 patients with leptomeningeal metastasis from breast cancer who meet the entry criteria.
The First Affiliated Hospital with Nanjing Medical University is the lead sponsor of 543 studies on the registry; 301 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria:The subjects must meet all of the following criteria simultaneously:
Exclusion Criteria:
Patients meeting any of the following criteria are not eligible to be included in this study:
Intrathecal chemotherapy group Patients received intrathecal 150mg Trastuzumab combination with 10mg thiotepa once every three weeks until an event that meets the criteria for termination occurs.
Drug: Intrathecal Administration of Thiotepa in Combination with Trastuzumab via the Ommaya Reservoir
Intrathecal chemotherapy group Patients received intrathecal 150mg Trastuzumab combination with 10mg thiotepa once every three weeks until an event that meets the criteria for termination occurs.
Intracranial Overall Response Rate
Intracranial overall response rate (iORR) is defined as the proportion of patients whose best overall response is either complete response (CR) or partial response (PR), as per local review and according to RANO-LM.
Time frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months
Intracranial Progression Free Survival
Intracranial progression-free survival (iPFS) is defined as the time from the date of randomization to the date of the first documented progression, as per local review and according to RANO-LM or death due to any cause.
Time frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months
Progression Free Survival
Progression-free survival (PFS) is defined as the time from the date of randomization to the date of the first documented progression as per local review and according to RECIST 1.1 or death due to any cause.
Time frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months
Overall Survival
Overall survival is defined as the time from the date of randomization to the date of death due to any cause
Time frame: From date of randomization until the date of death from any cause, assessed up to 100 months
frequency/severity of adverse events, lab abnormalities
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
Time frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months
Plan to share: Yes
Supporting information: Study protocol, Sap, Icf, Csr, Analytic code
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The First Affiliated Hospital with Nanjing Medical University