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Primary Central Nervous System Lymphoma (PCNSL) Clinical Trials

Also called PCNSL, Primary CNS Lymphoma, Brain Lymphoma, CNS Lymphoma, Primary Intraocular Lymphoma, Vitreoretinal Lymphoma, Relapsed Refractory PCNSL

Lymphoma is a cancer of lymphocytes, the white blood cells of the immune system. Most lymphomas start in lymph nodes.

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About Primary Central Nervous System Lymphoma

Lymphoma is a cancer of lymphocytes, the white blood cells of the immune system. Most lymphomas start in lymph nodes. Primary CNS lymphoma starts inside the central nervous system instead, in the brain, the spinal cord, the meninges that wrap them, or the eye, and in almost all cases it stays confined there. Under the microscope nearly every PCNSL is a diffuse large B-cell lymphoma, and they often carry changes in 2 genes, MYD88 and CD79B, that keep a growth signal called the B-cell receptor pathway switched on. That pathway runs through an enzyme called BTK, which is why BTK-blocking drugs are being tested.

Diagnosis usually starts with an MRI, then a needle biopsy of the tumor guided by a frame or computer, because surgery to remove the tumor does not help and is not used. An eye exam with dilated pupils and a spinal tap are part of the workup, since the eyes and spinal fluid can be involved without causing obvious symptoms. There is no formal staging system.

PCNSL is aggressive but treatable. In published trials the median survival ranges from 2 to 5 years, and outcomes are best in people under 60 who are well enough to carry out most daily activities and do not have a weakened immune system. The disease comes back often: roughly 20% to 30% of patients do not respond to the first treatment and up to 60% eventually relapse, which is where most of the new drug research is focused.

Common Symptoms of Primary Central Nervous System Lymphoma

Recognizing the signs of Primary Central Nervous System Lymphoma early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Headaches, nausea or vomiting
  • Confusion
  • Weakness in an arm or leg
  • Seizures
  • Double vision, since the eyes are part of the central nervous system
  • Hearing loss

Who Primary Central Nervous System Lymphoma Affects

PCNSL is mostly a disease of older adults, with more than half of patients diagnosed at 60 or older. A weakened immune system raises the risk sharply: people with HIV, people who have had an organ transplant, and people on long-term immune-suppressing medicines are all more likely to develop it, and in those cases the lymphoma is almost always linked to the Epstein-Barr virus. It also develops in people with no known risk factor at all.

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Side Effect Explorer

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Primary Central Nervous System Lymphoma Resources

Reputable organizations and medical references for learning more about Primary Central Nervous System Lymphoma, including disease registries, foundation resources, and clinical guidelines.

FDA decision ahead
The FDA is due to decide on Tirabrutinib (Deciphera Pharmaceuticals) for Relapsed/refractory primary CNS lymphoma by December 18, 2026. BTK inhibitor already approved in Japan for this rare brain lymphoma.
See all upcoming rare disease FDA decisions →

Active Clinical Trials for Primary Central Nervous System Lymphoma

Use this Primary Central Nervous System Lymphoma clinical trial finder to see the 19 studies recruiting patients and 1 opening soon in the United States and worldwide, with eligibility criteria in plain English. These studies play a critical role in advancing care for rare cancers conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

TrialsSite mapPipeline timeline

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

88 active trials worldwide
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RECRUITINGPHASE1, PHASE2Recently updatedNCT06343311

T-Cell Therapy (EB103) in Adults With Relapsed/Refractory B-Cell Non-Hodgkin's Lymphoma (NHL)

Intervention: EB103

Sponsor: Estrella Immunopharma, Inc. · Eureka Therapeutics Inc.

This is an open-label, multi-center, Phase 1/2 clinical trial evaluating the safety and effectiveness of EB103 in R/R B-cell NHL patients with the highest unmet medical needs, such as Human Immunodeficiency Virus (HIV)-associated lymphoma, primary and secondary central nervous sy...

Ages 18 Years+5 locations
Started Jun 2024Updated 3 days agoEst. Jul 2027 (~9 months)
RECRUITINGPHASE3Recently updatedNCT07104032

IGNITE: Study of Tirabrutinib vs Rituximab/Temozolomide for Relapsed/Refractory Primary Central Nervous System Lymphoma (PCNSL)

Intervention: Tirabrutinib, Rituximab, Temozolomide

Sponsor: Ono Pharmaceutical Co., Ltd. · Deciphera Pharmaceuticals, LLC

The purpose of this clinical trial is to evaluate efficacy and safety of tirabrutinib alone compared with rituximab and temozolomide (R-TMZ) combination therapy in participants with Relapsed/Refractory Primary Central Nervous System Lymphoma (PCNSL).

Ages 18 Years+36 locations
Started Jun 2026Updated 3 days agoEst. Dec 2027 (~1y 2m)
RECRUITINGPHASE2Recently updatedNCT07015242

A Study of the Efficacy and Safety of Lisocabtagene Maraleucel (Liso-cel) as First-Line Therapy in Adults With Transplant-Ineligible Primary Central Nervous System Lymphoma

Intervention: Rituximab, Methotrexate, Procarbazine, Temozolomide, Liso-cel, Fludarabine, Cyclophosphamide, Calcium folinate

Sponsor: Juno Therapeutics, Inc., a Bristol-Myers Squibb Company

The purpose of this study is to evaluate the safety and efficacy of lisocabtagene maraleucel (Breyanzi/liso-cel/BMS-986387) in adults as first-line treatment in transplant-ineligible Primary Central Nervous System Lymphoma (PCNSL).

Ages 18 Years+40 locations
Started Nov 2025Updated 4 days agoEst. Jan 2028 (~1y 3m)
RECRUITINGPHASE2Recently updatedNCT06175000

Maintenance Obinutuzumab in Treating Patients With Central Nervous System Lymphoma Who Have Achieved a Complete or Partial Response

Intervention: Cognitive Assessment, Obinutuzumab, Quality of Life Assessment

Sponsor: Providence Health & Services

This randomized phase II trial studies how well obinutuzumab works as maintenance treatment in patients with central nervous system lymphoma who have achieved the disappearance of all signs of cancer in response to treatment (complete response) or a decrease in the size of a tumo...

Ages 18 Years+7 locations
Started Mar 2024Updated 1 week agoEst. Sep 2028 (~1y 11m)
RECRUITINGPHASE1, PHASE2Recently updatedNCT04870944

CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma

Intervention: Biospecimen Collection, Bone Marrow Aspirate, Bone Marrow Biopsy, Echocardiography Test, FACT Complex-targeting Curaxin CBL0137

Sponsor: Children's Oncology Group

This phase I/II trial evaluates the best dose, side effects and possible benefit of CBL0137 in treating patients with solid tumors, including central nervous system (CNS) tumors or lymphoma that has come back (relapsed) or does not respond to treatment (refractory). Drugs, such a...

Ages 12 Months – 21 Years35 locations
Started Jan 2022Updated 3 weeks agoEst. Dec 2026 (~2 months)
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Active trial locations76 cities in the US
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Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Primary Central Nervous System Lymphoma patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Primary Central Nervous System Lymphoma treatments, clinical trial participation, and day-to-day disease management.

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Related Rare Cancers Conditions

Other rare diseases in the rare cancers category. Patients with Primary Central Nervous System Lymphoma may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Frequently Asked Questions About Primary Central Nervous System Lymphoma