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 April 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
Radiation Therapy followed by Intrathecal Trastuzumab/Pertuzumab in HER2+ Breast Leptomeningeal Disease
Goal of study:
To find out if radiation therapy followed by intrathecal trastuzumab and pertuzumab is safe and will improve survival in patients with HER2 positive breast cancer with leptomeningeal metastasis.
Who this is for:
- People 18 years and older
- Patients with HER2+ cancers with leptomeningeal metastasis / LMD
Contacts:
Moffitt Cancer Center, Tampa, FL
- Kamran Ahmed
Phone: 813.745.8424
Email: Kamran.Ahmed@moffitt.org - Michelle DeJesus
Phone: 813.745.6911
Email: Michelle.DeJesus@moffitt.org
Northwestern University, Evanston, IL
- Priya Kumthekar
Phone: 312.695.4360
Email: Priya.kumthekar@nm.org
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
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
A First in Human Dose Escalation of Dendritic Cell Vaccine (DCV)
Goal of study:
To learn about the effects of Dendritic Cell Vaccine (DCV), and to find the highest dose of DCV that can be given safely to breast cancer patients with leptomeningeal disease.
Who this is for:
- People 18 years and older
- Patients with triple negative breast cancer (TNBC) or HER2+ breast cancer with leptomeningeal metastasis
Contacts:
Moffitt Cancer Center, Tampa, FL
- Cecily Piteo
Phone: 813.745.6963
Email: Cecily.Piteo@moffitt.org
Concurrent Azeliragon with Craniospinal Irradiation
Goal of study:
To assess the safety of Azeliragon with radiation of the brain and spine in patients with leptomeningeal metastasis from solid tumor cancers and high-grade gliomas.
Who this is for:
- People 18 years and older
- Patients with solid tumors or high-grade gliomas and leptomeningeal metastasis
Contacts:
NY Langone Health, New York, NY
- Jonathan Yang
Phone: 212.731.6276
Email: Jonathan.Yang@nyulangone.org
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
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 #: 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.
Study of B7-H3-Specific CAR T Cell Immunotherapy for DIPG/DMG and Recurrent or Refractory Pediatric Central Nervous System Tumors
Goal of study:
Primary:
To test if an adequate amount of B7H3-specific CAR T cells can be manufactured to complete two courses of treatment; to test if B7H3-specific CAR T cells can safely be administered through an indwelling CNS catheter to allow the T cells to directly interact with tumor cells.
Secondary:
To evaluate CAR T cell distribution with the cerebrospinal fluid and the extent to which CAR T cells travel to the peripheral circulation or blood stream, and, evaluate disease response to therapy.
Who this is for:
- Children and young adults 1 to 26 years
- Patients with refractory or recurrent CNS disease for which there is no standard therapy, or diagnosis of DIPG or DMG at any time point following completion of standard therapy
Contacts:
Seattle Children’s Hospital
- Nick Vitanza, MD
Phone: 206-987-2106
Email: CBDCIntake@seattlechildrens.org
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
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
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
Intravenous and Intrathecal Nivolumab in Treating Patients With Melanoma or Lung Cancer and Leptomeningeal Disease
Goal of study:
To study the side effects and best dose of intrathecal nivolumab, and how well it works in combination with intravenous nivolumab in treating patients with leptomeningeal disease.
Who this is for:
- People 18 years and older
- Patients with primary central nervous system (CNS) melanoma, melanocytomas or metastatic melanoma (cutaneous, acral-lentiginous, uveal and mucosal in origin)
- Patients with non-small cell lung cancer
Study Contacts:
MD Anderson
- Isabella Glitza, MD
Phone: (713) 792-2921
Email:icglitza@mdanderson.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
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
Screening MRI of the Brain in Patients With Breast Cancer
Goal of study:
To study the usefulness of magnetic resonance imaging (MRI) to screen for brain/leptomeningeal metastases.
Who this is for:
- People 18 years and older
- Breast cancer with specific requirements (see criteria on clinicaltrials.gov)
Study Contact:
Brigham and Women’s Hospital, Boston, MA
NOT YET RECRUITING as of March 2026
A Phase I Pilot Safety and Feasibility Study for Solid Tumor Patients With CNS Metastases
This study is not yet recruiting participants.
Goal of study:
To evaluate the safety and feasibility of giving SV-BR-1-GM with pembrolizumab to patients who have solid tumor cancer with central nervous system metastases (brain metastases or leptomeningeal metastases)
Who this is for:
- People 18 years and older
- Patients with any solid tumor cancer with leptomeningeal metastasis and disease progression after 1 or more treatments
Contacts:
Medical College of Wisconsin Cancer Center Clinical Trials Office
- Phone: 866-680-0505 ext 8900
Email: cccto@mcw.edu
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
B7-H3.CD28Z.CART in CNS Neoplasms
This study is not yet recruiting participants.
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
CLINICAL TRIALS in CANADA
Tucatinib With Brain and/or Spinal XRT 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
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
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
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