Leptomeningeal DiseasePhase 1NCT05809752
A First in Human Dose Escalation of Dendritic Cell Vaccine (DCV)
A Phase 1 study testing Dendritic Cell Vaccine in people with Leptomeningeal Disease and Triple Negative Breast Cancer, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
Trial summary
The purpose of this study is to learn about the effects of the study treatment, Dendritic Cell Vaccine (DCV), to find the highest dose of the study treatment that can be given safely to Breast Cancer patients with Leptomeningeal Disease Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leptomeningeal Disease, Triple Negative Breast Cancer, HER2-positive Breast Cancer
- What is being tested?
- Dendritic Cell Vaccine
- Type of treatment
- Vaccines
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Aug 2023to Aug 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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
- Histologically or cytologically confirmed diagnosis of TNBC or HER2+BC per ASCO/CAP guidelines . A tumor can be considered a TNBC if the ER or PR is <10%.
- Trial participants must have a diagnosis of LMD. They must have the presence of malignant cells in the CSF (CSF+; note now cytology is considered diagnostic of LMD if the cytology is read as positive or suspicious; OR characteristic radiographic abnormalities (see below) of LMD). Signs and symptoms of LMD in and of themselves are not sufficient for inclusion.
- Patients must have an Eastern Cooperative Oncology Group performance scale of ≤ 3.
- Coincident Brain or Spinal cord metastases are allowed if these are stable and do not require local therapy at the time of enrollment. Individuals with previously treated stable Brain metastases are eligible to participate.
- Stereotactic Radiosurgery (SRS) and/or prior radiotherapy is permitted > 2 weeks prior to initial Dendritic Cell (DC) vaccine dose. A follow up brain MRI should be obtained prior to DC vaccine to determine stability of the lesions. An interval of at least 4 weeks after the end of whole brain radiation or for any surgical resection of brain lesions is permitted.
- Life expectancy of ≥ 8 weeks.
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- Demonstrate adequate organ function as defined in protocol. All screening labs should be performed with 14 days of treatment initiation.
- Provision of signed and dated informed consent form.
- Corticosteroids at doses equivalent to 8 mg dexamethasone daily for symptom control are acceptable. This should be minimized wherever possible.
- If the disease has progressed on current treatment in the CNS prior to consent, patients may continue current systemic cancer therapies as per PI discretion (Systemic Therapies Allowed) and Exclusion Criteria. Patients should not start a new anti-cancer agent until the 28 day safety period is completed.
- Patients with systemic disease are eligible and will be managed as detailed in Section 6.3.1.
- Pregnancy test: negative serum or urine pregnancy test at screening for women of childbearing potential. Must be repeated once-monthly during treatment. Contraception: Highly effective contraception for both male and female subjects throughout the study and for at least 90 days after last treatment administration, if the risk of conception exists.
- The patient has an Ommaya reservoir or equivalent device which allows routine access to CSF and administration of DC1s.
Who cannot join
- Receiving other treatments specifically administered to treat LMD within the last 2 weeks or 5 half-lives of the agent, whichever is less. However, all other treatments to control systemic disease or bulk CNS disease will be eligible, provided the therapy is not a phase I agent, an agent which significantly and unequivocally penetrates the CSF (e.g., high-dose methotrexate, thiotepa, high-dose ara-C) per PI discretion.
- The use of any immunotherapy within the last four weeks.
- Patients with a ventriculoperitoneal or ventriculoatrial shunt must have an on/off device in their shunt systems to be eligible for the study. Patients must be able to tolerate shunt closure for ~4 hours without development of clinical signs of increased intracranial pressure. Patients unable to tolerate shunt closure for ~4 hours will not be eligible for the study.
- Unable or unwilling to have a contrast-enhanced brain MRI.
- Known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin,squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy.
- Has an active infection requiring systemic therapy which in the opinion of the investigator will increase the risk to the patient.
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- Had major surgical procedure, or significant traumatic injury within two weeks. Ommaya placement is allowed.
- Has a history of current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator.
- Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
- Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 90 days after the last dose of trial treatment.
- Has a known history of Human Immunodeficiency Virus (HIV) (HIV 1, 2 antibodies). Testing is not mandatory.
- Has known active or chronic hepatitis B (HBV) or hepatitis C virus (HCV). Testing is not mandatory.
- ORGAN TRANSPLANTATION: Prior organ transplantation including allogenic stem-cell transplantation.
- Other severe acute or chronic medical conditions or psychiatric conditions including recent (within the past year) or active suicidal ideation or behavior; or laboratory abnormalities that may increase the risk associated with study participation or study treatment administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
- Has received a blood transfusion in the two weeks prior to leukapheresis.
- Has received a live vaccine within 30 days prior to the first dose of study drug. Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guérin (BCG), and typhoid vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (e.g., FluMist®) are live attenuated vaccines and are not allowed. Current COVID vaccines are not live vaccines.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A First in Human Dose Escalation of Dendritic Cell Vaccine (DCV) Administered Intrathecally (IT) Primed Against HER2/HER3 in Patients With Leptomeningeal Disease (LMD) From Triple-Negative Breast Cancer (TNBC) or HER2+ Breast Cancer (HER2+BC)
The purpose of this study is to learn about the effects of the study treatment, Dendritic Cell Vaccine (DCV), to find the highest dose of the study treatment that can be given safely to Breast Cancer patients with Leptomeningeal Disease
- Being tested
- Dendritic Cell Vaccine
- Medical subject headings
- Meningeal Neoplasms, Triple Negative Breast Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dendritic Cell VaccineBiological
Intrathecal (IT) dendritic cell vaccine (DCV) will be administered once every week. As per standard procedures of IT chemotherapy or antibody administration it is administered over 5 -10 minutes or at 1 ml/minute while monitoring the patient under sterile conditions. In general, to assure delivery of the DCVs into the ventricular space and compensate for "dead space" in the Ommaya, the delivery of IT DCV cells is followed by the administration of 2.5 mls of saline. In general there is a maximum volume of 10 mls of DCVs.
| Study group | Role | What they receive |
|---|---|---|
| Dendritic Cell (DC) Vaccine dose escalation | Experimental | HER2/3 peptide-pulsed DC1 will be administered intrathecally (IT) weekly for 6 doses /cycle for a maximum of two cycles, and then re-staged. If there are sufficient DC1s this may be continued weekly thereafter. There are 1 safety cohort (1 million DCV cells) and 3 escalating doses (2 million, 10 million and 50 million DCV cells) possible. Receives: Biological: Dendritic Cell Vaccine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose of Intrathecal Dendritic Cell Vaccine
Maximum tolerated dose of intrathecal dendritic cell vaccine is defined as the highest dose of vaccine that does not cause undesirable side effects/dose limiting toxicity (DLT). To evaluate safety, the study team will monitor toxicities continuously and the study will be halted if an excessive number of toxicities are encountered.
Measured: Up to 12 weeks
Secondary outcomes (2)
Overall Survival
Overall Survival (OS) will be measured from the initial date of treatment to the recorded date of death.
Measured: Up to 12 months
Progression Free Survival
Progression Free Survival defined as the time from start of treatment to the time of progression or death.
Measured: Up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2023
- Study start22 Aug 2023
- Primary completion13 May 2026
- Study completionAug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leptomeningeal DiseaseStudies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- United States Department of Defense
- Responsible party
- Sponsor
- Investigators
- Peter A Forsyth, MD, Principal investigator, Moffitt Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05809752
- Sponsor's protocol ID
- MCC-21262
- Status last verified
- Sep 2026
- First submitted
- 30 Mar 2023
- Last updated
- 15 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A First in Human Dose Escalation of Dendritic Cell Vaccine (DCV) Administered Intrathecally (IT) Primed Against HER2/HER3 in Patients With Leptomeningeal Disease (LMD) From Triple-Negative Breast Cancer (TNBC) or HER2+ Breast Cancer (HER2+BC)
- ClinicalTrials.gov ID
- NCT05809752
- Acronym / sponsor ID
- MCC-21262
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 15 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- United States Department of Defense
- Study start
- 22 Aug 2023 (actual)
- Primary completion
- 13 May 2026 (actual)
- Study completion
- Aug 2027 (estimated)
- First posted
- 12 Apr 2023 (actual)
- 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 to learn about the effects of the study treatment, Dendritic Cell Vaccine (DCV), to find the highest dose of the study treatment that can be given safely to Breast Cancer patients with Leptomeningeal Disease
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dendritic Cell (DC) Vaccine dose escalation | Experimental | Biological: Dendritic Cell VaccineHER2/3 peptide-pulsed DC1 will be administered intrathecally (IT) weekly for 6 doses /cycle for a maximum of two cycles, and then re-staged. If there are sufficient DC1s this may be continued weekly thereafter. There are 1 safety cohort (1 million DCV cells) and 3 escalating doses (2 million, 10 million and 50 million DCV cells) possible. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Histologically or cytologically confirmed diagnosis of TNBC or HER2+BC per ASCO/CAP guidelines . A tumor can be considered a TNBC if the ER or PR is <10%.
- Trial participants must have a diagnosis of LMD. They must have the presence of malignant cells in the CSF (CSF+; note now cytology is considered diagnostic of LMD if the cytology is read as positive or suspicious; OR characteristic radiographic abnormalities (see below) of LMD). Signs and symptoms of LMD in and of themselves are not sufficient for inclusion.
- Patients must have an Eastern Cooperative Oncology Group performance scale of ≤ 3.
- Coincident Brain or Spinal cord metastases are allowed if these are stable and do not require local therapy at the time of enrollment. Individuals with previously treated stable Brain metastases are eligible to participate.
- Stereotactic Radiosurgery (SRS) and/or prior radiotherapy is permitted > 2 weeks prior to initial Dendritic Cell (DC) vaccine dose. A follow up brain MRI should be obtained prior to DC vaccine to determine stability of the lesions. An interval of at least 4 weeks after the end of whole brain radiation or for any surgical resection of brain lesions is permitted.
Key exclusion criteria (16 listed)
- Receiving other treatments specifically administered to treat LMD within the last 2 weeks or 5 half-lives of the agent, whichever is less. However, all other treatments to control systemic disease or bulk CNS disease will be eligible, provided the therapy is not a phase I agent, an agent which significantly and unequivocally penetrates the CSF (e.g., high-dose methotrexate, thiotepa, high-dose ara-C) per PI discretion.
- The use of any immunotherapy within the last four weeks.
- Patients with a ventriculoperitoneal or ventriculoatrial shunt must have an on/off device in their shunt systems to be eligible for the study. Patients must be able to tolerate shunt closure for ~4 hours without development of clinical signs of increased intracranial pressure. Patients unable to tolerate shunt closure for ~4 hours will not be eligible for the study.
- Unable or unwilling to have a contrast-enhanced brain MRI.
- Known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin,squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy.
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 |
|---|---|---|
| Dendritic Cell VaccineIntrathecal (IT) dendritic cell vaccine (DCV) will be administered once every week. As per standard procedures of IT chemotherapy or antibody administration it is administered over 5 -10 minutes or at 1 ml/minute while monitoring the patient under sterile conditions. In general, to assure delivery of the DCVs into the ventricular space and compensate for "dead space" in the Ommaya, the delivery of IT DCV cells is followed by the administration of 2.5 mls of saline. In general there is a maximum volume of 10 mls of DCVs. | Biological | Dendritic Cell (DC) Vaccine dose escalation |
Primary outcome
Maximum Tolerated Dose of Intrathecal Dendritic Cell Vaccine
Time frame: Up to 12 weeks
Maximum tolerated dose of intrathecal dendritic cell vaccine is defined as the highest dose of vaccine that does not cause undesirable side effects/dose limiting toxicity (DLT). To evaluate safety, the study team will monitor toxicities continuously and the study will be halted if an excessive number of toxicities are encountered.
2 secondary outcomes
Overall Survival
Time frame: Up to 12 months
Progression Free Survival
Time frame: Up to 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2026.
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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.
Peter A Forsyth, MD principal investigator
Moffitt Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Dendritic Cell Vaccine in people with Leptomeningeal Disease and Triple Negative Breast Cancer, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
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 13 inclusion and 16 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: under way, not looking for participants, as of the sponsor's last update on 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Maximum Tolerated Dose of Intrathecal Dendritic Cell Vaccine (time frame: Up to 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Sep 2026.