Leptomeningeal MelanomaPhase 1NCT07414979
Phase 1 Study Of Intrathecal Cellular Adoptive Immunotherapy Using Autologous CD8+ Antigen-Specific T Cells For Patients With Leptomeningeal Melanoma
A Phase 1 study testing CD8+T cells in people with Leptomeningeal Melanoma, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The proposed study is a single arm Phase I trial aimed at treating up to 6 HLA-A*0201+ and HLA-A*24 02+ individuals with leptomeningeal disease from metastatic melanoma. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leptomeningeal Melanoma
- What is being tested?
- CD8+T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Aug 2026to 22 Mar 2030
- 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
- Patients must have radiographic and/or CSF cytological evidence of LMD.
- Must have a confirmed diagnosis of primary CNS melanoma, melanocytomas or metastatic melanoma (cutaneous, acral-lentiginous, uveal, or mucosal in origin), based on histological analysis of metastatic tissue and/or cancer cells, archival tissue permitted.
- Male or female subjects ≥18 years of age.
- Expression of HLA-A2 or HLA-A24.
- ECOG/ Zubrod performance status ≤1 at screening visit for this treatment
- Ability to understand and the willingness to sign a written informed consent document.
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- The effects of Endogenous T Cell (ETC) Therapy on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). This includes all female patients, between the onset of menses (as early as 8 years of age) and 55 years unless the patient presents with an applicable exclusionary factor which may be one of the following:
- Postmenopausal (no menses in greater than or equal to 12 consecutive months).
- History of hysterectomy or bilateral salpingo-oophorectomy.
- Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).
- History of bilateral tubal ligation or another surgical sterilization procedure.
- Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
- Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of Endogenous T Cell (ETC) Therapy administration.
- Reproductive Status
- Investigators shall counsel women of childbearing potential participants, on the importance of pregnancy prevention and the implications of an unexpected pregnancy. The investigator shall evaluate the effectiveness of the contraceptive method in relationship to the first dose of study intervention.
- Local laws and regulations may require the use of alternative and/or additional contraception methods.
- Female Participants:
- Female participants must have documented proof that they are not of childbearing potential.
- Note: Women who are not of childbearing potential are exempt from contraceptive requirements.
- WOCBP must have a negative highly sensitive serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotropin) within 24 hours prior to the start of study intervention. An extension up to 72 hours prior to start of study treatment is permissible in situations where results cannot be obtained within the standard 24-hour window.
- If a urine test cannot be confirmed as negative (e.g., an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.
- The investigator is responsible for review of medical history, menstrual history, and recent sexual activity to potentially decrease the risk for inclusion of a woman with an undetected pregnancy.
- WOCBP must agree to follow instructions for method(s) of contraception as described and included in the ICF.
- WOCBP are permitted to use hormonal contraception methods. A female participant is eligible to participate if she is not pregnant or breastfeeding.
- participant is eligible to participate if she is not pregnant or breastfeeding.
- Concurrent Therapy
- Patients may receive steroids to control symptoms related to CNS involvement, but the dose must be ≤ 4 mg per 24 hours of dexamethasone (or the equivalent). Patient's symptoms should experience stability of neurological symptoms for at least 7 days and without increasing needs for steroids. Physiologic replacement doses for adrenal insufficiency are allowed on this protocol but should not exceed 40 mg of hydrocortisone daily (or its equivalent).
- Patients who have been treated with an approved targeted therapy (BRAF inhibitor and/or MEK inhibitor) will be allowed to remain on concurrent approved targeted therapy.
- Patients who have received an approved systemic biologic therapy (e.g., antiPD-1, anti-CTLA4, IL-2, interferon) must have received their last treatment ≥ 2 weeks prior to the start of treatment with IT ECT, but can continue with treatment of anti-PD1 and/or CTLA-4
- Treatment with anti-epileptics is allowed while on protocol.
- No other concomitant intrathecal therapy with another agent will be allowed.
- Washout periods from other therapies Patients who have received radiation to brain and/or spine, including whole brain radiation, stereotactic radiosurgery (SBRT), are eligible, but must have completed radiation treatment at least 7 days prior to the start of treatment with IT ECT.
- Patients that received previous IT therapy must have received their last treatment ≥ 7 days prior to the start of treatment with IT ECT.
- Patients who have received systemic chemotherapy must have received their last treatment ≥ 14 days prior to the start of treatment with IT ECT.
- Patients who have received an approved systemic biologic therapy (e.g., antiPD-1, anti-CTLA4, IL-2, interferon) must have received their last treatment ≥ 2 weeks prior to the start of treatment with IT ECT, but can continue with treatment of anti-PD1 and/or CTLA-4 (see exclusion paragraph)
- Patients who have received any other investigational agents must have received their last treatment ≥ 14 days prior to the start of treatment with IT ECT.
Who cannot join
- Any underlying medical or psychiatric condition, which in the opinion of the investigator, will make the administration of study drug hazardous or obscure the interpretation of adverse events, such as a condition associated with frequent diarrhea.
- All associated toxicity from previous or concurrent cancer therapy must be resolved (to ≤ grade 1 or baseline) prior to study treatment administration. This excludes non-serious toxicities. However, stable endocrinopathies requiring replacement therapy will be allowed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
The University of Texas M. D. Anderson Cancer Center
Houston, Texas, 77030
Isabella Glitza, MD, PHD, 713-792-2921, icglitza@mdanderson.org
Isabella Glitza, MD, PHD
About this study
The proposed study is a single arm Phase I trial aimed at treating up to 6 HLA-A*0201+ and HLA-A*24 02+ individuals with leptomeningeal disease from metastatic melanoma.
Read the detailed description
Primary Objective:
Evaluate the safety of adoptively transferred intrathecal ETC targeting melanoma tumors in patients with leptomeningeal disease.
Secondary Objectives:
- Evaluate the duration of in vivo persistence (peripheral blood and CSF) and anti-tumor efficacy achieved following adoptive transfer of antigen specific ETC.
- Overall survival for up to one year after end of patient monitoring.
Primary Endpoint:
Safety and Toxicity Evaluation
Secondary Endpoint:
- Persistence of transferred T cells.
- The numeric and functional persistence of transferred CTL will be performed on CSF samples (where available) and peripheral blood obtained from patients prior to T cell infusion (baseline sample), and as scheduled above. The numeric frequency of transferred T cells will be determined using peptide MHC-tetramer analysis or specific CDR3 TCR quantitative PCR of transferred CTL if necessary. The function of transferred CTL will be determined by intracellular cytokine staining of tetramer+ CD8+ cells following in vitro stimulation.
- Tetramer analysis. Since all patients on study will be HLA-A2+ and HLA-A24+, the use of tetramers to track several well-characterized tumor-associated HLA-A2 and HLA-A24-restricted responses will be feasible. These include responses to several melanosomal antigens: (tyrosinase, MART1/MelanA, gp100, SLC45A2) and CT antigens: (PRAME, MAGE-A1, MAGE-A10). Flow-based tetramer staining permits multiparametric analysis such that surface markers of differentiation (CD62L, CCR7, CD27, CD28) can be used to further characterize the effector, effector memory or central memory phenotype of the induced population of antigen-specific T cells.
- Overall survival
- Conditions
- Leptomeningeal Melanoma
- Being tested
- CD8+T cells
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD8+T cellsDrug
Also called cytotoxic T lymphocytes
Given by infusion
| Study group | Role | What they receive |
|---|---|---|
| IT-ETC: Treatment with CD8+ T-cells (IT) Q4W | Experimental | The proposed study is a single arm Phase I trial aimed at treating up to 6 HLA-A*0201+ and HLA-A*24 02+ individuals with leptomeningeal disease from metastatic melanoma. On Cycle 1 Day 1 and Cycle 1 Day 15, patients will receive an intrathecal infusion of TAA-specific T cells. The patients will receive ~300 (150-300) million cells on Cycle 1 Day 1 and ~1000 (750-1000) million cells on Cycle 1Day 15. Receives: Drug: CD8+T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Adverse Events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2026
- Study start31 Aug 2026 (estimated)
- Primary completion22 Mar 2028 (estimated)
- Study completion22 Mar 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leptomeningeal MelanomaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Isabella Glitza, MD, PHD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07414979
- Sponsor's protocol ID
- 2024-1333
- Other IDs
- NCI-2026-01059
- Status last verified
- Aug 2026
- First submitted
- 13 Feb 2026
- Last updated
- 12 Aug 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 12 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Study Of Intrathecal Cellular Adoptive Immunotherapy Using Autologous CD8+ Antigen-Specific T Cells For Patients With Leptomeningeal Melanoma
- ClinicalTrials.gov ID
- NCT07414979
- Acronym / sponsor ID
- 2024-1333
- Conditions
- Leptomeningeal Melanoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 31 Aug 2026 (estimated)
- Primary completion
- 22 Mar 2028 (estimated)
- Study completion
- 22 Mar 2030 (estimated)
- First posted
- 17 Feb 2026 (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 proposed study is a single arm Phase I trial aimed at treating up to 6 HLA-A*0201+ and HLA-A*24 02+ individuals with leptomeningeal disease from metastatic melanoma.
Detailed description
Primary Objective:
Evaluate the safety of adoptively transferred intrathecal ETC targeting melanoma tumors in patients with leptomeningeal disease.
Secondary Objectives:
- Evaluate the duration of in vivo persistence (peripheral blood and CSF) and anti-tumor efficacy achieved following adoptive transfer of antigen specific ETC.
- Overall survival for up to one year after end of patient monitoring.
Primary Endpoint:
Safety and Toxicity Evaluation
Secondary Endpoint:
- Persistence of transferred T cells.
- The numeric and functional persistence of transferred CTL will be performed on CSF samples (where available) and peripheral blood obtained from patients prior to T cell infusion (baseline sample), and as scheduled above. The numeric frequency of transferred T cells will be determined using peptide MHC-tetramer analysis or specific CDR3 TCR quantitative PCR of transferred CTL if necessary. The function of transferred CTL will be determined by intracellular cytokine staining of tetramer+ CD8+ cells following in vitro stimulation.
- Tetramer analysis. Since all patients on study will be HLA-A2+ and HLA-A24+, the use of tetramers to track several well-characterized tumor-associated HLA-A2 and HLA-A24-restricted responses will be feasible. These include responses to several melanosomal antigens: (tyrosinase, MART1/MelanA, gp100, SLC45A2) and CT antigens: (PRAME, MAGE-A1, MAGE-A10). Flow-based tetramer staining permits multiparametric analysis such that surface markers of differentiation (CD62L, CCR7, CD27, CD28) can be used to further characterize the effector, effector memory or central memory phenotype of the induced population of antigen-specific T cells.
- Overall survival
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IT-ETC: Treatment with CD8+ T-cells (IT) Q4W | Experimental | Drug: CD8+T cellsThe proposed study is a single arm Phase I trial aimed at treating up to 6 HLA-A*0201+ and HLA-A*24 02+ individuals with leptomeningeal disease from metastatic melanoma. On Cycle 1 Day 1 and Cycle 1 Day 15, patients will receive an intrathecal infusion of TAA-specific T cells. The patients will receive ~300 (150-300) million cells on Cycle 1 Day 1 and ~1000 (750-1000) million cells on Cycle 1Day 15. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (36 listed)
- Patients must have radiographic and/or CSF cytological evidence of LMD.
- Must have a confirmed diagnosis of primary CNS melanoma, melanocytomas or metastatic melanoma (cutaneous, acral-lentiginous, uveal, or mucosal in origin), based on histological analysis of metastatic tissue and/or cancer cells, archival tissue permitted.
- Male or female subjects ≥18 years of age.
- Expression of HLA-A2 or HLA-A24.
- ECOG/ Zubrod performance status ≤1 at screening visit for this treatment
Key exclusion criteria (2 listed)
- Any underlying medical or psychiatric condition, which in the opinion of the investigator, will make the administration of study drug hazardous or obscure the interpretation of adverse events, such as a condition associated with frequent diarrhea.
- All associated toxicity from previous or concurrent cancer therapy must be resolved (to ≤ grade 1 or baseline) prior to study treatment administration. This excludes non-serious toxicities. However, stable endocrinopathies requiring replacement therapy will be allowed.
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 |
|---|---|---|
| CD8+T cellsAlso: cytotoxic T lymphocytesGiven by infusion | Drug | IT-ETC: Treatment with CD8+ T-cells (IT) Q4W |
Primary outcome
Safety and Adverse Events (AEs)
Time frame: Through study completion; an average of 1 year
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
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 12 Aug 2026.
- MD Anderson Cancer Center WebsiteLink provided by the sponsor.
Related trials for Leptomeningeal Melanoma
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Active, not recruitingPhase 2
Lymphodepletion Plus Adoptive Cell Transfer With or Without Dendritic Cell Immunization in Patients With Metastatic Melanoma
Melanoma
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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.
Isabella Glitza, MD, PHD principal investigator
M.D. Anderson 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 CD8+T cells in people with Leptomeningeal Melanoma, run by M.D. Anderson Cancer Center 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 36 inclusion and 2 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: opening soon, as of the sponsor's last update on 12 Aug 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?
Contact Isabella Glitza, MD, PHD ((713) 792-2921, icglitza@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Adverse Events (AEs) (time frame: Through study completion; an average of 1 year). 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 12 Aug 2026.