Clinical Trials
Clinical trials are research studies that test how diseases can be detected, prevented or treated. They can also study quality of life by looking at how patients feel from a disease and how they feel from the treatment.
For leptomeningeal cancer, clinical trials test new and existing treatments. These treatments may be medications, surgery, radiation, or other methods.
Scientists design trials to answer questions about:
- Safe doses
- Ways to deliver treatment
- Side effects
- Effectiveness
- Length of life
Clinical trials take place in phases:
- Phase 1 trials test a treatment for safety and side effects in a small group of people.
- Phase 2 trials further study safety and determine how effective treatments are in a larger group of people.
- Phase 3 trials compare the new treatment with standard or similar treatments to confirm its effectiveness and monitor side effects. The trials also collect other important information about the new treatment. These trials test treatments in a large group of people.
Items to note:
- Many trials have a set number of people they can admit into a trial.
- Each trial has a list of inclusion and exclusion criteria. These are factors required for someone to be included or factors that prevent someone from being in the trial. This could be your physical condition, the status of your disease, or prior treatments.
- Clinical trials may only be available in certain places. You may need to travel to be in a trial.
- Most trials studying leptomeningeal cancer are Phase 1, some are Phase 2. There are no current Phase 3 trials.
All trials are listed at ClinicalTrials.gov
For trials specific to leptomeningeal cancer, visit LeptoTrials.com
For brief descriptions of current trials, see below.
Please read before proceeding:
- The trials below are updated as of September 2026.
- The information here is adapted from ClinicalTrials.gov for easy reviewing.
- These trials are subject to change. We will do our best to update regularly to reflect any changes.
- Inclusion and exclusion criteria are not included. We have just provided a general idea of who the trial is for. Please read the trial description at the link for more details.
- If any of these trials are of interest, please visit the links for more information about the trials and reach out to the contacts listed.
- Leptomeningeal Cancer Foundation does not recommend or take a position on any of the clinical trials listed on this page.
- Leptomeningeal Cancer Foundation is not responsible for any errors, changes to trials or any misinformation.
- Please consult with your doctor regarding clinical trials and which trial(s) may be appropriate for your individual case.
CLINICAL TRIALS in the UNITED STATES
Trial #: NCT07814352
Locations:
- New Jersey: Basking Ridge, Middletown, Montvale
- New York: Commack, Harrison, Uniondale
A Study of SX-682 in Combination With Proton Craniospinal Irradiation (pCSI) in People With Leptomeningeal Disease
Goal of study:
The goal to find out whether SX-682 in combination with proton craniospinal irradiation is safe and effective for people with melanoma or non-small cell lung cancer with leptomeningeal disease.
Who this is for:
- Adults 18 years and older
- Patients with melanoma or NSCLC and leptomeningeal disease
Contacts:
Monica Chen, MD
Phone: 646-888-5108
Email: chenm9@mskcc.org
Intrathecal Therapy with dendritic cell vaccine in combination with Trastuzumab (for HER2+ breast cancer patients) and Nivolumab (for HER2- breast cancer patients)
Goal of study:
To learn about the effects of the study treatment, Dendritic Cell Vaccine (DCV), in combination with trastuzumab or nivolumab to confirm the highest dose of the study treatment that can be given safely to participants with Breast Cancer (BC) with Leptomeningeal Disease (LMD).
Who this is for:
- People 18 years and older
- Patients with breast cancer and leptomeningeal disease
- Must have an Ommaya port
Study Contacts:
Moffitt Cancer Center
Julianne Hardesty
Phone: 813-745-4098
Email: Julianne.Hardesty@moffitt.org
Optimizing Contrast Dose and Scanning Parameters for Detection of Leptomeningeal Disease
Goal of study:
To learn if gadopiclenol (a contrast agent) used during MRI scanning can help in the detection of early LMD.
Who this is for:
- People 18 years and older
- Patients with any type of solid cancer and brain MRI with questionable results for LMD and negative LP CSF sampling
- Patients with high risk clinical LMD and negative brain MR and LP CSF sampling
Study Contacts:
MD Anderson
Rami Eldaya, MD
Phone: (713) 745-2945
Email:reldaya@mdanderson.org
B7-H3.CD28Z.CART in CNS Neoplasms
Goal of study:
Who this is for:
- Children and young adults 2 years to 21 years
- Please see the ClinicalTrials.gov page for specific criteria
Contacts:
Boston Children’s Hospital / Dana-Farber Cancer Institute
Susan Chi, MD
Phone: 617-632-3270
Email:Susan_Chi@dfci.harvard.edu
Trial #: NCT07361562
Locations:
- Grand Rapids, MI
- New York, NY
- Nashville, TN
- Austin, TX
- West Valley City, UT
- Fairfax, VA
A Study of a Selective ERBB2 Inhibitor (CGT4255), in Patients With Advanced Solid Tumors
Goal of study:
To evaluate the safety, tolerability, pharmacokinetic (what the body does to the drug), pharmacodynamic (what the drug does to the body), and antitumor activity of CGT4255 in adult participants with advanced solid tumors with ERBB2 alterations or HER2 overexpression.
Who this is for:
- People 18 years and older
- Locally advanced, metastatic, and/or unresectable solid tumor with ERBB2-activating alteration or NRG1 gene fusion in blood and/or tumor or HER2 overexpression in tumor
- Locally advanced, metastatic, and/or unresectable NSCLC with ERBB2 mutation in blood and/or tumor
- Locally advanced, metastatic and/or unresectable breast cancer with documented ERBB2 mutation in blood and/or tumor or HER overexpression in tumor
Study Contact:
Cogent Biosciences, Inc
Phone: 617-945-5576
Email: trialinfo@cogentbio.com
Multiple Dose Intraventricular Administration of Rhenium-186 NanoLiposome for Leptomeningeal Metastases (CA2024-LM-001)
Goal of study:
To determine the safety and efficacy of multiple doses at defined intervals of rhenium (186Re) obisbemeda (rhenium-186 nanoliposome, 186RNL) given through an Ommaya port to identify a safe dose amount, dosing schedule, and number of doses. This is a Phase 1 study.
Who this is for:
- People 18 years and older
- Patients with any primary solid tumor cancer with leptomeningeal metastases
Study Contacts:
The Cancer Therapy and Research Center at UTHSCA
- Rachael Hershey
Phone: 210.791.8723
Email: patients@respect-trials.com - Leti Velten, RN
Phone: 210.450.1921
Email: velten@uthsca.edu
Photon Craniospinal Irradiation for the Treatment of Leptomeningeal Disease Secondary to Breast Cancer or Non-small Cell Lung Cancer
Goal of study:
To test how well photon craniospinal radiation works in patients with leptomeningeal metastasis from breast or non-small cell lung cancers
Who this is for:
- People 18 years and older
- Patients with breast cancer or non-small cell lung cancer (NSCLC) with leptomeningeal metastasis
Contacts:
City of Hope, California
- Stephanie Yoon
Phone: 626.873.5241
Email: styoon@coh.org
Study in Patients With Breast Cancer Leptomeningeal Metastasis (BioLept)
Goal of study:
To collect tissue, blood, and CNS samples; utilize the Johns Hopkins Molecular Tumor Board to provide multi-disciplinary treatment recommendations; and collect outcome data that can be correlated with biomarkers.
Who this is for:
- People 18 years and older
- Breast cancer with confirmed or suspected leptomeningeal metastasis
Study Contact:
Hopkins Breast Trials
Phone: 410-614-1361
Email: HopkinsBreastTrials@jhmi.edu
Trial #: NCT06518057
Locations: Seattle, Washington’ San Francisco (Not yet recruiting)
Hippocampal Avoidance in Craniospinal Irradiation for the Treatment of Leptomeningeal Metastases from Breast Cancer or Non-small Cell Lung Cancer
Goal of study:
To see how well radiation using proton therapy or volumetric modulated arc therapy (VMAT) with hippocampal avoidance works in patients with breast cancer or non-small cell lung cancer (NSCLC) with leptomeningeal metastases. Hippocampal avoidance reduces the amount of radiation to the hippocampus, which can reduce neurologic side effects from treatment.
Who this is for:
- People 18 years and older
- Patients with breast cancer or non-small cell lung cancer (NSCLC) and leptomeningeal metastasis
Contacts:
Fred Hutch/University of Washington Cancer Consortium, Seattle, WA
- Lia M. Halasz
Phone: 206.897.2121
Email: lhalasz@uw.edu
Trial #: NCT06500481
Locations:
- Multiple locations across the U.S. See ClinicalTrials.Gov for specific details.
Testing Proton Craniospinal Radiation Therapy Versus the Usual Radiation Therapy for Leptomeningeal Metastasis, RADIATE-LM Trial
Goal of study:
To compare proton craniospinal irradiation (pCSI) to involved-field radiation therapy (IFRT) for the treatment of breast or non-small cell lung cancer with leptomeningeal metastasis
Who this is for:
- People 18 years and older
- Patients with breast cancer or non-small cell lung cancer (NSCLC) and leptomeningeal metastasis
Contacts:
Multiple locations across the U.S. See ClinicalTrials.Gov for specific details.
DATO-BASE: DATOpotamab-deruxtecan for Breast Cancer Brain metAstaSEs
Goal of study:
To test the safety and effectiveness of the study drug datopotamab deruxtecan (an antibody conjugate) in patients with metastatic breast cancer that has spread to the brain.
Who this is for:
- People 18 years and older
- People with estrogen receptor (ER)-positive HER2-negative breast cancer
- People with metastatic triple negative breast cancer
- People with HER2-negative metastatic breast cancer that has spread to the leptomeninges
Study Contacts:
Dana Farber Cancer Center, Boston, MA
Sarah Sammons, MD
617-362-3800
Email:sarahl_sammons@dfci.harvard.edu
Miami Baptist Cancer Institute, Miami, FL
- Manmeet Ahluwalia, MD
Phone: 786-527-8010
Email: manmeeta@baptisthealth.net
Duke University Medical Center, Durham, NC
- Danielle Brander, MD
Email: danielle.brander@dm.duke.edu
- Peggy Alton, RN
Phone: 919-681-4769
Email: peggy.alton@dm.duke.edu
Trastuzumab Deruxtecan (T-DXd) for People With Brain Cancer
Goal of study:
To find out how much tratuzumab deruxtecan (T-DXd) can penetrate the tumor when injected into the body, and whether T-DXd may be an effective treatment for brain cancers that express the HER2 protein.
Who this is for:
- People 18 years and older
- Patients with a brain tumor that needs resection/biopsy and leptomeningeal metastasis may be eligible
Contacts:
Memorial Sloan Kettering Cancer Center, New York, NY
- Nelson Moss
Phone: 212.639.7075
Email: mossn@mskcc.org
Trial #: NCT05967689
Locations:
- 18 Locations in U.S.
- Additional locations around the world
A Study of Zipalertinib in Patients With Advanced Non-Small Cell Lung Cancer With EGFR Exon 20 Insertions or Other Uncommon Mutation. (REZILIENT2)
Goal of study:
This study will evaluate the safety and efficacy of zipalertinib in patients with locally advanced or metastatic NSCLC with EGFR exon 20 mutations or other EGFR mutations
Who this is for:
- Adults 18 years and older
- Patients with NSCLC with ex20ins or other uncommon single or compound EGFR non-ex20ins status,
- Brain metastasis (leptomeningeal disease included)
Contacts:
Taiho Oncology, INC
Phone: +1 844-878-2446
Email: medicalinformation@taihooncology.com
Tumor Treating Fields for the Treatment of Leptomeningeal Metastases of the Spine in Patients with Breast or Lung Cancer
Goal of study
To evaluate the safety and feasibility of tumor treating fields (TTF) in the treatment of spinal leptomeningeal disease in patients with breast or lung cancer.
Who this is for:
- People 18 years and older
- Patients with breast cancer and leptomeningeal metastases
- Patients with lung cancer and leptomeningeal metastases
Contacts:
Mayo Clinic, Florida
- Wendy J. Sherman
Phone: 855.776.0015
Email: mayocliniccancerstudies@mayo.edu
Trial #: NCT05184816
Locations:
- New Jersey (Basking Ridge, Middletown, Montvale)
- New York (Commack, Harrison, NYC, Uniondale)
A Study of Deferoxamine (DFO) in People with Leptomeningeal Metastasis
Goal of study:
To find out if deferoxamine given intrathecally (directly into the CSF) is a safe treatment for people with leptomeningeal metastasis from solid tumor cancer.
Who this is for:
- People 18 years and older
- Patients with leptomeningeal metastasis from any solid tumor
Contacts:
Memorial Sloan Kettering Cancer Center, New York, NY
- Jessica Wilcox
Phone: 212.639.7573
Email: wilcoxj@mskcc.org
Compare Encorafenib + Binimetinib + Nivolumab vs. Ipilimumab + Nivolumab in BRAF-V600 Mutant Melanoma with Brain Metastases
Goal of study:
This phase II trial compares the effect of encorafenib, binimetinib, and nivolumab versus ipilimumab and nivolumab in patients with BRAF- V600 mutant melanoma that has spread to the brain (brain metastases). This trial aims to find out which approach is more effective in shrinking and controlling brain metastases from melanoma.
Who this is for:
- Adults 18 years and older
- Participants must have confirmed melanoma that has metastasized to the brain
- Any primary or unknown origin types of melanoma except uveal melanoma
- BRAF-V600 mutant melanoma
- Leptomeningeal disease is acceptable
Contacts:
Catrina Mireles
Phone: 210-614-8808, ext. 1014
Email:Cmireles@swog.org
Palbociclib and Pembrolizumab In Breast Cancer with Central Nervous System Metastases
Goal of study:
To study the efficacy and safety of the combination of palbociclib and pembrolizumab as a possible treatment for recurrent central nervous system (CNS) metastases.
Who this is for:
- People 18 years and older
- Breast cancer with brain or leptomeningeal metastasis
Study Contact:
Priscilla Brastianos, MD
Phone: 617-724-8770
Email:PBRASTIANOS@mgh.harvard.edu
Trial #: NCT02422641
Locations:
- Baltimore, Maryland
- Winston-Salem, North Carolina
- St Louis, Missouri
(active, but not recruiting)
Prospective Evaluation of High-Dose Systemic Methotrexate in Patients with Breast Cancer and Leptomeningeal Metastasis
Goal of study:
To determine if treatment with IV methotrexate will increase survival among patients with triple negative, HER2-positive, and hormone refractory metastatic breast cancer patients with LMD.
Who this is for:
- People 18 years and older
- Patients with invasive breast cancer – triple negative (TNBC), HER2+, and hormone refractory (ER/PR+)
Contacts:
Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD
- Joy Fisher
Email: jfisher@jhmi.edu
Comprehensive Cancer Center at Wake Forest University, Winston-Salem, NC
- Cindy Miller
Email: cytmill@wakehealth.edu
NOT YET RECRUITING as of September 2026
BRain-AVOidant Proton Craniospinal Irradiation for Leptomeningeal Metastases From Solid Tumor Malignancies (BRAVO-LM)
This study is not yet recruiting participants.
Goal of study:
This is a multicenter study evaluating the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), cerebrospinal fluid penetration, and preliminary antitumor activity of orally administered JBI-778 in patients with brain metastases, leptomeningeal disease, or recurrent high-grade glioma.
Who this is for:
- People 18 years and older
Solid tumors with leptomeningeal metastases
- Additional criteria
Contacts:
- Morgan Freret, MD
Phone: (305) 243-5470
Email: mfreret@miami.edu
Study Evaluating the Safety and Clinical Activity of Oral JBI-778 in Patients With Brain Metastases, Leptomeningeal Disease, or Recurrent High Grade Glioma
This study is not yet recruiting participants.
Goal of study:
This is a multicenter study evaluating the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), cerebrospinal fluid penetration, and preliminary antitumor activity of orally administered JBI-778 in patients with brain metastases, leptomeningeal disease, or recurrent high-grade glioma.
Who this is for:
- People 18 years and older
Solid tumors with stable brain metastases or Recurrent/progressive high-grade glioma, or Brain metastases from NSCLC, breast cancer, or melanoma, or Leptomeningeal disease
- Additional criteria
Contacts:
- Melda Dolan, MD
Phone: 314.498.9959
Email: Melda.Dolan@jubl.com
ctDNA-Guided Intraventricular Therapy for CNS Malignancies (GUIDE-CNS)
This study is not yet recruiting participants.
Goal of study:
Who this is for:
- People 18 years and older
- Patients with HER2+ breast cancer, or triple negative breast cancer, or Stage IV non-small cell lung cancer, or recurrent high-grade glioma
- Additional criteria
Contacts:
Milton S. Hershey Medical Center
- Crystal Sowers
Phone: 717.531.5471
Email: psci-cto@pennstatehealth.psu.edu
Phase 1 Study of Whole Brain Radiation Therapy or Proton Craniospinal Irradiation With REYOBIQ for Leptomeningeal Metastases
This study is not yet recruiting participants.
Goal of study:
Who this is for:
- People 18 years and older
- Any solid tumor with leptomeningeal metastases
- Additional criteria
Contacts:
New York University Langone Health
- Jonathan Yang, MD, PHD
Phone: 212.731.6030
Email: Jonathan.Yang@nyulangone.org
Phase 1 Study Of Intrathecal Cellular Adoptive Immunotherapy Using Autologous CD8+ Antigen-Specific T Cells For Patients With Leptomeningeal Melanoma
This study is not yet recruiting participants.
Goal of study:
Who this is for:
- People 18 years and older
- Patients with confirmed diagnosis of LMD
- Patients with a a confirmed diagnosis of primary CNS melanoma, melanocytomas or metastatic melanoma
- Expression of HLA-A2 or HLA-A24
Contacts:
The University of Texas M.D. Anderson Cancer Center
- Isabella Glitza, MD, PHD
Phone: 713.792.2921
Email: icglitza@mdanderson.org
Gallium Maltolate for the Treatment of Pediatric Patients With Relapsed or Refractory Pediatric High-Grade Glioma and Atypical Teratoid Rhabdoid Tumor (GABRIEL)
This study is not yet recruiting participants.
Goal of study:
In this study, we want to find out more about the side effects and safety of Gallium Maltolate (GaM), an investigational drug, for relapsed or refractory atypical teratoid rhabdoid tumor and high-grade glioma.
Who this is for:
- Children 0 to 17 years
- Patients must have a prior diagnosis of pediatric high-grade glioma or ATRT or molecular features of such tumors
- See study for specific criteria
Contacts:
Sarah Rumler, DO
Phone: 414-266-2000
Email: srumler@mcw.edu
CLINICAL TRIALS in CANADA
Tucatinib, Trastuzumab and Capecitabine With Brain and/or Spinal radiotherapy in Patients With HER2+ Metastatic Breast Cancer and LMD
Goal of study:
To test the safety and efficacy of radiation therapy followed by systemic therapy among patients with HER2+ metastatic breast cancer and LMD
Who this is for:
- People 18 years and older
- Men or women with HER2+ metastatic breast cancer.
- Evidence of LMD in the brain and/or spine
Contacts:
The Ottawa Hospital, Ottawa, Ontario, Canada
- CLIMB-LMD Project Manager
Phone: 437.247.2617Email: CLIMB-LMD@sunnybrook.ca
Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada
- Dr. Katarzyna Jerzak
Phone: 437.247.2617
Email: katarzyna.jerzak@sunnybrook.ca
CLINICAL TRIALS in EUROPE
Trial #: NCT06933199
Locations:
Czechia (Brno, Hradec Králové, Olomouc); Moravian-Silesian Region (Ostrava)
Neoadjuvant Stereotactic Radiotherapy for Brain Metastasis (NeoSTROBE)
Goal of study:
To establish the value of fractionated neoadjuvant stereotactic radiotherapy as a new treatment paradigm for brain metastases.
Who this is for:
- People 18 years and older
- Target metastases indicated for surgical resection.
- No history of whole brain radiation or stereotactic radiation to a brain metastasis
- No cases of small cell lung cancer
Contacts:
- Jiří Hynčica
Phone: 0042059737 ext 2587
Email: jiri.hyncica@fno.cz
Trial #: NCT05800275
Locations:
France (Bordeaux, Caen, Clermont-Ferrand, Dijon, Lyon, Montpellier, Nice, Reims, Rouen, Strasbourg, Villejuif)
Capecitabine, Tucatinib, and Intrathecal Trastuzumab for Breast Cancer Patients with Leptomeningeal Disease
Goal of study:
To test the efficacy of tucatinib and capecitabine in combination with intrathecal trastuzumab on overall survival rate in HER2-positive metastatic breast cancer patients with leptomeningeal disease.
Who this is for:
- People 18 years and older
- Patients with HER2+ metastatic breast cancer and leptomeningeal cancer
Contacts:
- Telma ROQUE, PhD
Phone: +33 (0) 1 80 50 12 92
Email:t-roque@unicancer.fr - Jérôme LEMONNIER, PhD
Phone: +33 (0) 1 71 93 67 02
Email:j-lemonnier@unicancer.fr
Proton Cranio-spinal Irradiation for Leptomeningeal Metastasis (CSI ProLong)
Goal of study:
To test the effects of proton craniospinal radiotherapy, believed to offer superior survival and CNS control compared to focal photon radiotherapy.
Who this is for:
- People 18 years and older
- Patients with leptomeningeal metastasis from solid or hematological cancers
Contacts:
Aalborg University Hospital
- Weronoka Szejniuk, MD
Aarhus University Hospital
- Kenneth Jensen, PhD
Phone: +45 78 45 64 00
Email: kennjens@rm.dk
- Dorte Winther, M.H.Sc.
Phone: +45 78 45 64 00
Email: dorte.skriver.winther@auh.rm.dk
Trial #: NCT05112549
Locations:
Germany (Bonn, Freiburg, Heidelberg, Heilbronn, Mannheim, München, Stuttgart, Tübingen, Ulm)
Intrathecal Application of PD1 Antibody in Metastatic Solid Tumors With Leptomeningeal Disease
Goal of study:
To determine the safety of intrathecal PD1 antibody (Nivolumab) in metastatic solid tumors with leptomeningeal disease.
Who this is for:
- People 18 years and older
- Patients with LMD from a solid tumor
Contact:
- Ghazaleh Tabatabai, Prof.Dr.
Phone: +49 (0) 7071 – 2985018
Email:ghazaleh.tabatabai@uni-tuebingen.de
NOT YET RECRUITING as of September 2026
Platform Trial Assessing Uptake, Safety and Efficacy of Theranostic Agents in HER2+ Breast Cancer Patients With Active Brain Metastases (THERANOVA)
This study is not yet recruiting participants.
Goal of study:
This trial will evaluate treatment for patients with solid tumors with active brain metastases. Patients will be treated with a theranostic or diagnostic agent. Theranostic agents are targeted radioactive drugs used to identify (diagnose) and to deliver therapy. The main purpose of the study is to analyze the efficacy (to find out how effective a treatment is) of each of the theranostic agents in patients who have solid tumors with active brain metastases.
Who this is for:
- People 18 years and older
- Patients with HER2+ breast cancer and active brain metastases or leptomeningeal metastases
Contacts:
- Medsir
- Phone: +34 932 214 135
- Email: contact.trials@medsir.org