Intrathecal thiotepa plus trastuzumab for HER2‑positive breast cancer with leptomeningeal metastasis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a single‑arm, prospective Phase 2 multicenter trial enrolling about 26 patients with HER2‑positive breast cancer and leptomeningeal metastasis to receive intrathecal thiotepa combined with trastuzumab via an Ommaya reservoir. The primary endpoint is intracranial objective radiographic response rate (iORR) using the RANO‑LM radiographic scoring system, with radiographic progression and response defined by percentage change from baseline. Secondary endpoints include intracranial progression‑free survival, overall progression‑free survival, overall survival, and exploratory correlations between molecular markers and response. Patients must have confirmed HER2 positivity, leptomeningeal disease, and either an implanted or implantable Ommaya reservoir and meet hematologic and organ function requirements.

Why it matters

Who can join: the full rules

Inclusion Criteria:The subjects must meet all of the following criteria simultaneously:

* Histologically or cytologically confirmed HER-2 positive breast cancer (immunohistochemistry indicates HER-2 3+ and/or fluorescence in situ hybridization indicates HER-2 gene amplification)
* Diagnosed with breast cancer with leptomeningeal metastasis based on cerebrospinal fluid cytology combined with central nervous system function and brain imaging findings
* The patient has an Ommaya reservoir implanted or is eligible for implantation
* KPS ≥ 30
* Adequate bone marrow and liver and kidney function reserves: absolute neutrophil count (ANC) ≥ 1.5 x 109/L, platelets ≥ 100 x 109/L, hemoglobin ≥ 90 g/L. International normalized ratio (INR) or prothrombin time (PT) ≤ 1.5 x ULN. Activated partial thromboplastin time (aPTT) ≤ 1.5 x ULN. Total serum bilirubin ≤ 1.5 x ULN (patients with Gilbert's syndrome can be enrolled if total bilirubin \< 3 x ULN). Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN; if the patient has liver metastasis, this criterion is AST and ALT ≤ 5 x ULN.
* Female, aged between 18 and 75 years old
* Left ventricular ejection fraction (LVEF) \> 50%
* Voluntary signing of informed consent form

Exclusion Criteria:

Patients meeting any of the following criteria are not eligible to be included in this study:

* Subjects with other malignant tumors, excluding skin basal cell carcinoma and carcinoma in situ
* Presence of severe or uncontrollable systemic diseases, including uncontrollable hypertension or active bleeding tendency
* Patients judged by the investigator to be unsuitable for participation in the trial, or those with factors that may affect the patient's compliance with the protocol
* Toxicity caused by previous treatment has not recovered to normal state or is grade 1 according to NCI-CTCAE 5.0
* Allergic to or with metabolic disorders to the drugs in this protocol
* Pregnant or lactating women, or women with pregnancy plans during the study period and within 6 months after the last administration
* Patients participating in other clinical studies simultaneously

Where it's running

• Jiangsu Provincial People's Hospital
  Nanjing, Jiangsu, China Recruiting

Study contacts

• Study coordinator: Wei Li, Ph.D
• Email: real.lw@163.com
• Phone: 025-68307102

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.