Metastatic Breast CancerPhase 1/2HITNCT01373710
Intrathecal Trastuzumab Administration in Metastatic Breast Cancer Patients Developing Carcinomatous Meningitis
A Phase 1/2 study testing Trastuzumab in people with Metastatic Breast Cancer and Carcinomatous Meningitis, run by Institut Curie at 10 sites in France.
Trial summary
The purpose of this study is: Phase I: To determine the Trastuzumab maximum tolerated dose (MTD) when weekly administrated by intrathecal or intraventricular route to reach a intra CSF target concentration (30 µg/mL) near the conventional therapeutic concentration and depending on the dose-limiting toxicity (DLT)… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Metastatic Breast Cancer, Carcinomatous Meningitis
- What is being tested?
- Trastuzumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 10 sitesFrance
- Study dates
- 19 May 2011to 9 Apr 2018
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Metaplastic Infiltrating adenocarcinoma of the breast
- HER2 Overexpression by IHC and / or amplification (FISH and or ICHS)
- Positive diagnosis of neoplastic meningitis: positive CSF cytology (obtained within 28 days before inclusion) AND / OR clinical symptoms of neoplastic meningitis and aspect of tumoral meningitis on MRI
- Brain metastases are allowed without prior treatment, if they are asymptomatics and without engagement. In cases of symptomatic brain metastases, subjects could be included only if surgery and / or radiotherapy (stereotactic or in toto) were performed and if the cerebral metastatic localization allow IT or intra-ventricular treatment. The last radiotherapy session or the surgery must have been done 3 weeks before.
- Aged 18 years old or more
- Male and female
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- Life expectancy more than 2 months
- Satisfactory Cardiac function: left ventricular ejection fraction (LVEF) determined by ultrasound scan or myocardial scintigraphy
- Adequate Biological functions 14 days before inclusion, according to the criteria below: Neutrophils > 1.0 x 109/L, Hemoglobin > 9.0 g/dL (+ transfusion if needed,Platelets > 50 x 109/L,Bilirubin < 3 x N, ALT \& AST < 10 x N, Creatinine < 2.0 mg/dL, Clearance > 25 mL/min (Cockcroft and Gault formula), Prothrombin time > 70 %, Kaolin cephalin coagulation time < 1.5 x N.
- Women of childbearing potential, must take adequate birth control measure during the study period and must have a negative pregnancy test (BetaHCG serum)
- The subjects must perform all evaluations of pre-inclusion, as provided by the protocol
- Signed written inform consent
Who cannot join
- CSF circulation disorders suspected on MRI brain (obstructive hydrocephalus) or medullar (obstacle) with, in case of a focal radiotherapy on obstructive lesion, checking the restoration of transit traffic by isotope CSF
- Anti-coagulant effective dose treatment when trastuzumab administration by lumbar puncture
- Patient on Lapatinib (wash out> 2 weeks from the date of first dose intrathecal trastuzumab)
- Known or suspected trastuzumab allergy
- Contraindications of trastuzumab administration, including cardiac diseases: LVEF <laboratory lower limit of normal or any other heart condition which would expose the subject to an unreasonable risk if he were to participate in the study
- Severe toxicity unresolved or unstable related to another previous study restricted drug and / or a cancer treatment
Show all 15Show fewer
- Ventriculoperitoneal or atrial shunting excepted if the valve could be turn off (on-off switch) and the patient can stand it during 6 h after each injection of trastuzumab
- Dementia, altered mental status or psychiatric condition that would prevent the subject to understand or give informed consent
- Pre-existing severe cerebrovascular disease, such as stroke in a major vessel, vasculitis in the central nervous system or malignant hypertension
- Uncontrolled infection
- Participation in a clinical study with an experimental molecule
- No affiliation to a Social insurance (beneficiary or assignee)
- Pregnant women, breastfeeding or of childbearing age not taking contraceptive
- Subject unable to make follow up schedule
- Persons deprived of liberty or under guardianship (including curators)
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
France10 sites
Institut Bergonié
Bordeaux, 33076
François Baclesse Center
Caen, Calvados, 14076
CHU Grenoble
Grenoble, 38700
Oscar Lambret Center
Lille, Nord, 59020
Léon Bérard Center
Lyon, Rhone, 69373
Institut du Cancer de Montpellier
Montpellier, 34298
Institut Curie - Claudius Regaud Hospital
Paris, Île-de-France Region, 75248
Pitie Salpetriere Hospital
Paris, Île-de-France Region, 75651
Rene Huguenin Hospital
Saint-Cloud, Haut de Seine, 92210
Institut Univesitaire du Cancer de Toulouse
Toulouse, 31100
About this study
Official title: Phase 1-2 Study of Safety and Efficacy of Intrathecal Trastuzumab Administration in Metastatic HER2 Positive Breast Cancer Patients Developing Carcinomatous Meningitis
The purpose of this study is:
Phase I: To determine the Trastuzumab maximum tolerated dose (MTD) when weekly administrated by intrathecal or intraventricular route to reach a intra CSF target concentration (30 µg/mL) near the conventional therapeutic concentration and depending on the dose-limiting toxicity (DLT)
Phase II: Determination of antitumor activity trastuzumab when administrated by IT or intra-ventricular in terms of neurological progression-free survival at 2 months
Read the detailed description
Phase I: Secondary Outcome Measures:
Recommended dose (RD will be used in Phase II) Toxicity during treatment Clinical response to specific neurologic symptoms Time to neurologic progression Biological response: CSF cellularity and protein concentration Radiological response: cerebrospinal meningitis and neuraxis RMI Impact on quality of life Impact on survival (overall survival, survival without neurological progression, progression-free survival) Pharmacokinetics: dose of trastuzumab in CSF and plasma FCGR3A Genetic status influence on efficacy trastuzumab in metastatic breast cancer
Phase II: Secondary Outcome Measures :
Toxicity during treatment Clinical response to specific neurologic symptoms Time to neurologic progression Biological response: CSF cellularity and protein concentration Radiological response: cerebrospinal meningitis and neuraxis MRI Impact on quality of life Impact on survival (overall survival, survival without neurological progression, progression-free survival) Pharmacokinetics: dose of trastuzumab in CSF and plasma (confirmation of phase I data with 5 patients) FCGR3A Genetic status influence on efficacy trastuzumab in metastatic breast cancer
- Conditions
- Metastatic Breast Cancer, Carcinomatous Meningitis
- Being tested
- Trastuzumab
- Keywords
- Adverse Reaction to Trastuzumab Administered by intrathecal
- Medical subject headings
- Breast Neoplasms, Meningeal Carcinomatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TrastuzumabDrug
Also called Herceptin
One injection per week during 8 weeks by lumbar puncture or Ommaya Reservoir. 4 levels of doses are expected from 30 mg to 150 mg
| Study group | Role | What they receive |
|---|---|---|
| Trastuzumab intrathecal | Experimental | Not described Receives: Drug: Trastuzumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase I : To determine the Trastuzumab maximum tolerated dose (MTD) when weekly administrated by intrathecal or intraventricular route.
Phase I : To determine the Trastuzumab maximimum tolerated dose (MTD) when weekly administrated by intrathecal or intraventricular route to reach a intra CSF target concentration (30 µg/mL) near the conventional therapeutic concentration and depending on the dose-limiting toxicity (DLT).
Measured: 2 months
Secondary outcomes (10)
Phase I : Recommended dose (RD will be used in Phase II)
Measured: 2 months
Phase I&II : Toxicity during treatment
Issued the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 National Cancer Institute (NCI)
Measured: 2 months
Time to neurologic progression
Measured: 2 years
Biological response: CSF cellularity and protein concentration
Measured: 2 years
Radiological response: cerebrospinal meningitis and neuraxis MRI
Measured: 2 years
Impact on quality of life
Measured: 2 years
Impact on survival (overall survival, survival without neurological progression, progression-free survival)
Measured: 2 years
Pharmacokinetics: dose of trastuzumab in CSF and plasma
Measured: 2 months
FCGR3A Genetic status influence on efficacy trastuzumab in metastatic breast cancer
Measured: 2 years
Phase II : Determination of antitumor activity trastuzumab when administrated by IT or intra-ventricular in terms of neurological progression free survival at 2 months
Measured: 2 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2011 (estimated)
- Study start19 May 2011
- Primary completionApr 2018
- Study completion9 Apr 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Metastatic Breast CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut Curie
- Sponsor type
- University, hospital or other
- Collaborators
- Hoffmann-La Roche, Centre Leon Berard, Groupe Hospitalier Pitie-Salpetriere, Centre Oscar Lambret, Centre Francois Baclesse, Institut Bergonié, University Hospital, Toulouse, Institut du Cancer de Montpellier - Val d'Aurelle, University Hospital, Grenoble
- Responsible party
- Sponsor
- Investigators
- Maya Gutierrez, MD, Study director, Institut Curie - Hopital Rene Huguenin - Saint-Cloud - France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01373710
- Sponsor's protocol ID
- 09/501/M
- Other IDs
- 2009-017218-63, 2009
- Acronym
- HIT
- Status last verified
- Nov 2025
- First submitted
- 24 May 2011
- Last updated
- 24 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Bonneau C, Paintaud G, Tredan O, Dubot C, Desvignes C, Dieras V, Taillibert S, Tresca P, Turbiez I, Li J, Passot C, Mefti F, Mouret-Fourme E, Le Rhun E, Gutierrez M. Phase I feasibility study for intrathecal administration of trastuzumab in patients with HER2 positive breast carcinomatous meningitis. Eur J Cancer. 2018 May;95:75-84. doi: 10.1016/j.ejca.2018.02.032. Epub 2018 Apr 7.
PubMed 29635147
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1-2 Study of Safety and Efficacy of Intrathecal Trastuzumab Administration in Metastatic HER2 Positive Breast Cancer Patients Developing Carcinomatous Meningitis
- ClinicalTrials.gov ID
- NCT01373710
- Acronym / sponsor ID
- HIT / 09/501/M
- Conditions
- Metastatic Breast Cancer, Carcinomatous Meningitis
- Keywords
- Adverse Reaction to Trastuzumab Administered by intrathecal
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Institut CurieUniversity, hospital or other
- Collaborators
- Hoffmann-La Roche, Centre Leon Berard, Groupe Hospitalier Pitie-Salpetriere, Centre Oscar Lambret, Centre Francois Baclesse, Institut Bergonié, University Hospital, Toulouse, Institut du Cancer de Montpellier - Val d'Aurelle, University Hospital, Grenoble
- Study start
- 19 May 2011 (actual)
- Primary completion
- Apr 2018 (actual)
- Study completion
- 9 Apr 2018 (actual)
- First posted
- 15 Jun 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is:
Phase I: To determine the Trastuzumab maximum tolerated dose (MTD) when weekly administrated by intrathecal or intraventricular route to reach a intra CSF target concentration (30 µg/mL) near the conventional therapeutic concentration and depending on the dose-limiting toxicity (DLT)
Phase II: Determination of antitumor activity trastuzumab when administrated by IT or intra-ventricular in terms of neurological progression-free survival at 2 months
Detailed description
Phase I: Secondary Outcome Measures:
Recommended dose (RD will be used in Phase II) Toxicity during treatment Clinical response to specific neurologic symptoms Time to neurologic progression Biological response: CSF cellularity and protein concentration Radiological response: cerebrospinal meningitis and neuraxis RMI Impact on quality of life Impact on survival (overall survival, survival without neurological progression, progression-free survival) Pharmacokinetics: dose of trastuzumab in CSF and plasma FCGR3A Genetic status influence on efficacy trastuzumab in metastatic breast cancer
Phase II: Secondary Outcome Measures :
Toxicity during treatment Clinical response to specific neurologic symptoms Time to neurologic progression Biological response: CSF cellularity and protein concentration Radiological response: cerebrospinal meningitis and neuraxis MRI Impact on quality of life Impact on survival (overall survival, survival without neurological progression, progression-free survival) Pharmacokinetics: dose of trastuzumab in CSF and plasma (confirmation of phase I data with 5 patients) FCGR3A Genetic status influence on efficacy trastuzumab in metastatic breast cancer
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Trastuzumab intrathecal | Experimental | Drug: Trastuzumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Metaplastic Infiltrating adenocarcinoma of the breast
- HER2 Overexpression by IHC and / or amplification (FISH and or ICHS)
- Positive diagnosis of neoplastic meningitis: positive CSF cytology (obtained within 28 days before inclusion) AND / OR clinical symptoms of neoplastic meningitis and aspect of tumoral meningitis on MRI
- Brain metastases are allowed without prior treatment, if they are asymptomatics and without engagement. In cases of symptomatic brain metastases, subjects could be included only if surgery and / or radiotherapy (stereotactic or in toto) were performed and if the cerebral metastatic localization allow IT or intra-ventricular treatment. The last radiotherapy session or the surgery must have been done 3 weeks before.
- Aged 18 years old or more
Key exclusion criteria (15 listed)
- CSF circulation disorders suspected on MRI brain (obstructive hydrocephalus) or medullar (obstacle) with, in case of a focal radiotherapy on obstructive lesion, checking the restoration of transit traffic by isotope CSF
- Anti-coagulant effective dose treatment when trastuzumab administration by lumbar puncture
- Patient on Lapatinib (wash out> 2 weeks from the date of first dose intrathecal trastuzumab)
- Known or suspected trastuzumab allergy
- Contraindications of trastuzumab administration, including cardiac diseases: LVEF <laboratory lower limit of normal or any other heart condition which would expose the subject to an unreasonable risk if he were to participate in the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TrastuzumabAlso: HerceptinOne injection per week during 8 weeks by lumbar puncture or Ommaya Reservoir. 4 levels of doses are expected from 30 mg to 150 mg | Drug | Trastuzumab intrathecal |
Primary outcome
Phase I : To determine the Trastuzumab maximum tolerated dose (MTD) when weekly administrated by intrathecal or intraventricular route.
Time frame: 2 months
Phase I : To determine the Trastuzumab maximimum tolerated dose (MTD) when weekly administrated by intrathecal or intraventricular route to reach a intra CSF target concentration (30 µg/mL) near the conventional therapeutic concentration and depending on the dose-limiting toxicity (DLT).
10 secondary outcomes
Phase I : Recommended dose (RD will be used in Phase II)
Time frame: 2 months
Phase I&II : Toxicity during treatment
Time frame: 2 months
Time to neurologic progression
Time frame: 2 years
Biological response: CSF cellularity and protein concentration
Time frame: 2 years
Radiological response: cerebrospinal meningitis and neuraxis MRI
Time frame: 2 years
Impact on quality of life
Time frame: 2 years
Impact on survival (overall survival, survival without neurological progression, progression-free survival)
Time frame: 2 years
Pharmacokinetics: dose of trastuzumab in CSF and plasma
Time frame: 2 months
FCGR3A Genetic status influence on efficacy trastuzumab in metastatic breast cancer
Time frame: 2 years
Phase II : Determination of antitumor activity trastuzumab when administrated by IT or intra-ventricular in terms of neurological progression free survival at 2 months
Time frame: 2 month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Nov 2025.
- Results publication: PubMed 29635147Bonneau C, Paintaud G, Tredan O, Dubot C, Desvignes C, Dieras V, Taillibert S, Tresca P, Turbiez I, Li J, Passot C, Mefti F, Mouret-Fourme E, Le Rhun E, Gutierrez M. Phase I feasibility study for intrathecal administration of trastuzumab in patients with HER2 positive breast carcinomatous meningitis. Eur J Cancer. 2018 May;95:75-84. doi: 10.1016/j.ejca.2018.02.032. Epub 2018 Apr 7.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Maya Gutierrez, MD study director
Institut Curie - Hopital Rene Huguenin - Saint-Cloud - France
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Trastuzumab in people with Metastatic Breast Cancer and Carcinomatous Meningitis, run by Institut Curie at 10 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase I : To determine the Trastuzumab maximum tolerated dose (MTD) when weekly administrated by intrathecal or intraventricular route. (time frame: 2 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01373710, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Nov 2025.