Efficacy and Safety of Intrathecal Administration of Thiotepa in Combination With Trastuzumab in Breast Cancer With Leptomeningeal Metastasis

NCT07263425 | Recruiting Phase 2

• 1 other identifier: Ommaya 02
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

Brief Summary

Evaluate the efficacy and safety of Intrathecal Administration of Thiotepa in Combination with Trastuzumab via the Ommaya Reservoir in Breast Cancer with Leptomeningeal Metastasis

Conditions

Leptomeningeal Metastasis of HER2-positive Breast Cancer

Keywords

Trastuzumab; Thiotepa; intrathecal chemotherapy

Outcome Measures

Primary Outcomes (1)

• 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.

  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

Secondary Outcomes (4)

• Intracranial Progression Free Survival

  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

  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

  From date of randomization until the date of death from any cause, assessed up to 100 months

• frequency/severity of adverse events, lab abnormalities

  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

Study Arms (1)

Experimental

EXPERIMENTAL

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

Interventions

Intrathecal Administration of Thiotepa in Combination with Trastuzumab via the Ommaya Reservoir DRUG

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.

Experimental

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• The Affiliated Brain Hospital of Nanjing Medical University: collaborator
• The First Affiliated Hospital with Nanjing Medical University: lead

Study Sites (1)

Jiangsu Provincial People's Hospital

Nanjing, Jiangsu, 210000, China

RECRUITING

Central Study Contacts

Wei Li, Ph.D

CONTACT

025-68307102; real.lw@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2025
• First Posted: December 4, 2025
• Study Start: October 27, 2025
• Primary Completion (Estimated): October 27, 2027
• Study Completion (Estimated): October 27, 2027
• Last Updated: December 4, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE

Locations

CN (1)