Condition

Medulloblastoma clinical trials and research

• 18Clinical trials
• 82NIH research projects
• 6Countries

Newest Medulloblastoma clinical trials

All recruiting trials (18)

• Becotatug Vedotin for EGFR-positive relapsed, refractory, or metastatic pediatric solid tumors

  This trial will test Becotatug Vedotin, an EGFR-targeted antibody-drug conjugate, to see if it is safe and helps children aged 2–18 with EGFR-positive relapsed, refractory, or metastatic solid tumors.

  Recruiting Phase 1 Sun Yat-sen University · Guangzhou, Guangdong · NCT07584499

• B7-H3 CAR T cells for recurrent brain tumors in kids and young adults

  This early-phase trial tests a personalized CAR T-cell therapy called B7-H3 CAR T for children and young adults with recurrent or progressive brain tumors.

  Recruiting at 2 sites Phase 1 Dana-Farber Cancer Institute · Boston, Massachusetts and 1 other locations · NCT07390539

• Liothyronine with BIT chemotherapy for relapsed medulloblastoma in kids and young adults

  This study tests Liothyronine (L-T3) with a BIT chemotherapy regimen in children and young adults whose medulloblastoma has come back or progressed after standard treatment.

  Recruiting at 2 sites Phase 1/2 University of California, San Francisco · San Francisco, California and 2 other locations · NCT07346157

• GPC2-targeted CAR T cell therapy for relapsed or refractory medulloblastoma in children and young adults

  This trial will test whether autologous GPC2-targeted CAR T cells delivered into the brain ventricle are safe and active for children and young adults with relapsed or refractory medulloblastoma or similar CNS embryonal tumors.

  Recruiting Phase 1 Stanford University · Palo Alto, California · NCT07087002

• Long-term follow-up for medulloblastoma survivors treated with craniospinal irradiation

  This project will see if people treated for medulloblastoma with craniospinal irradiation have lasting health effects years after treatment.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT07085325

• Pharmacoscopy-guided treatment for refractory brain tumors

  This trial tests whether personalized drug testing on a patient’s tumor cells can guide treatment choices for adults with hard-to-treat brain cancers like glioblastoma.

  Recruiting Phase 2 University of Zurich · Basel, Basel-Landschaft and 2 other locations · NCT06804655

• Identifying biomarkers to predict cognitive decline after radiation therapy for brain tumors

  This study is trying to find specific markers in blood and spinal fluid from young patients with brain tumors to see if they can predict problems with thinking and memory after radiation treatment.

  Recruiting Tata Memorial Centre · Mumbai, Maharashtra · NCT06779487

• Using digoxin to treat relapsed medulloblastoma in young patients

  This study is testing if digoxin can help young patients with relapsed medulloblastoma feel better when other treatments have not worked.

  Recruiting at 17 sites Phase 2 H. Lee Moffitt Cancer Center and Research Institute · Birmingham, Alabama and 17 other locations · NCT06701812

• Combining Atovaquone with Radiation for Treating Children's Brain Tumors

  This study is testing if adding atovaquone to radiation therapy can help children with aggressive brain tumors feel better and improve their treatment outcomes.

  Recruiting at 2 sites Phase 1 Emory University · Atlanta, Georgia and 1 other locations · NCT06624371

• Combining Lutathera® and Olaparib for treating recurrent solid tumors in children and adolescents

  This study is testing if combining Lutathera® with olaparib can help children and teenagers with hard-to-treat solid tumors that have certain receptors.

  Recruiting Phase 1/2 Fundación de investigación HM · Boadilla del Monte, Madrid · NCT06607692

• Testing PLX038 for brain and spinal cord tumors with MYC or MYCN amplifications

  This study is testing a new treatment called PLX038 for adults with brain and spinal cord tumors that have specific gene changes to see if it helps improve their condition.

  Recruiting Phase 1/2 National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT06161519

• Investigating genetic and molecular profiles of pediatric brain tumors in China

  This study is trying to understand the genetic makeup of brain tumors in children to see how it affects their treatment and recovery.

  Recruiting Beijing Tiantan Hospital · Beijing, Beijing Municipality · NCT05672043

• Targeted therapy for young children with newly diagnosed medulloblastoma

  This study is testing a new targeted treatment for young children with medulloblastoma to see if it helps them live longer without the disease and how it affects their thinking skills.

  Recruiting at 10 sites Phase 2 St. Jude Children's Research Hospital · Palo Alto, California and 9 other locations · NCT05535166

• Lutathera treatment for recurrent high-grade brain tumors

  This study is testing if Lutathera can safely help children and young adults with recurring high-grade brain tumors that show certain markers on scans.

  Recruiting at 2 sites Phase 1/2 Nationwide Children's Hospital · Aurora, Colorado and 3 other locations · NCT05278208

• Combining Ibrutinib and Indoximod for Pediatric Brain Cancer Treatment

  This study is testing a new combination of two drugs, ibrutinib and indoximod, to see if they can safely help children and young adults with hard-to-treat brain tumors feel better.

  Recruiting Phase 1 Augusta University · Augusta, Georgia · NCT05106296

• Personalized treatment for children and young adults with relapsed brain tumors

  This study is testing personalized treatment plans for children and young adults with relapsed brain tumors to see if using real-time drug screening and genetic information can help them get the best and safest therapies.

  Recruiting at 9 sites University of California, San Francisco · Los Angeles, California and 8 other locations · NCT05057702

• Using DFMO for Maintenance Therapy in High Risk Medulloblastoma

  This study is testing if a new oral medication called DFMO can help children and young adults with high-risk medulloblastoma stay cancer-free after their initial treatment.

  Recruiting at 26 sites Phase 2 Milton S. Hershey Medical Center · Little Rock, Arkansas and 25 other locations · NCT04696029

• Using 68-Ga DOTATATE PET/MRI to Diagnose and Manage Certain Brain Tumors

  This study is testing if a special type of imaging called 68Ga-DOTATATE PET/MRI can help doctors better diagnose and manage certain brain tumors, especially meningiomas, by telling the difference between tumor recurrence and changes after treatment.

  Recruiting Phase 4 Weill Medical College of Cornell University · New York, New York · NCT04081701

NIH-funded research (newest 40 of 82)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Glial origins and signaling in childhood brain cancer (medulloblastoma)

  A new study looks at how glial cells may drive relapse and growth in childhood medulloblastoma, with the goal of finding targeted treatments.

  NIH research University of Alabama at Birmingham · Birmingham, United States · NIH-11535384

• Next generation GPC2 CARs to treat pediatric medulloblastoma

  A new CAR T-cell therapy approach targets a protein common on child brain tumor cells to improve outcomes for kids with medulloblastoma.

  NIH research Stanford University · Stanford, United States · NIH-11457465

• Targeting medulloblastoma with multispecific T-cell engagers

  A pediatric brain cancer treatment study tests a new multi-targeted T-cell engager designed to reach brain tumors and help the immune system kill cancer cells.

  NIH research University of Minnesota · Minneapolis, United States · NIH-11446519

• Efflux-aware brain barrier model to guide pediatric medulloblastoma drug discovery

  A study using a pediatric brain cancer model to test how radiation-activated digoxin-like compounds could safely reach brain tumors in children.

  NIH research Mayo Clinic Rochester · Rochester, United States · NIH-11443917

• Overcoming medulloblastoma resistance in kids using in vivo perturb-seq and preclinical therapies

  A patient-focused study aiming to find new, less toxic ways to treat pediatric medulloblastoma by using advanced genetic screening to identify effective drug targets in preclinical models.

  NIH research University of California, San Francisco · San Francisco, United States · NIH-11428652

• Dysregulation of a key gene complex and its role in Group 4 medulloblastoma

  A study explores how disruption of a core gene complex in brain development may drive Group 4 medulloblastoma and seeks to understand new ways to help affected children.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11421475

• Targeting MBD2 for medulloblastoma therapy

  Aimed at developing brain-penetrant small molecules to treat medulloblastoma in children by reactivating a tumor-suppressing gene.

  NIH research University of Alabama at Birmingham · Birmingham, United States · NIH-11412727

• Exploit immune changes from U1 mutations in Sonic Hedgehog medulloblastoma

  Aimed at children and adults with a type of brain cancer called Sonic Hedgehog medulloblastoma, this project looks at a specific genetic mutation to see if immune-based therapies could work better.

  NIH research University of Tx Md Anderson Can Ctr · Houston, United States · NIH-11404909

• ecDNA: a possible driver of tumor diversity and treatment resistance in high-risk medulloblastoma

  A pediatric brain cancer research project is looking at circular DNA pieces inside tumor cells to see if they help the cancer become diverse and resistant to treatment.

  NIH research Sanford Burnham Prebys Medical Discovery Institute · La Jolla, United States · NIH-11394423

• Deciphering unusual protein drivers in high-risk medulloblastoma

  A physician-scientist at the University of Michigan is studying non-canonical proteins that may drive a deadly form of pediatric brain cancer so future therapies can target them.

  NIH research University of Michigan at Ann Arbor · Ann Arbor, United States · NIH-11393708

• Overcoming therapy resistance in Hedgehog-driven medulloblastoma in kids

  A pediatric brain cancer study tests new ways to keep Hedgehog pathway drugs working longer in children with a specific type of medulloblastoma.

  NIH research Utah State Higher Education System--University of Utah · Salt Lake City, United States · NIH-11384882

• Understanding Why Some Pediatric Medulloblastoma Treatments Fail

  Researchers are studying why medulloblastoma, a pediatric brain tumor, stops responding to treatment and how to stop those relapse cells from growing.

  NIH research Medical University of South Carolina · Charleston, United States · NIH-11381785

• Immunotherapy for high-risk pediatric brain cancer using systems immunology

  A program to understand why a dangerous form of childhood brain cancer resists immunotherapy and to develop new immune-based treatments for kids with Group 3 medulloblastoma.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11380776

• Role of SMARCD3/BAF60C in pediatric brain cancer

  A study in children with medulloblastoma will look at how a protein called SMARCD3 helps tumor cells spread and grow.

  NIH research University of Pittsburgh at Pittsburgh · Pittsburgh, United States · NIH-11371210

• Understanding how KMT2D gene changes drive childhood brain cancer (medulloblastoma)

  This grant explores how changes in a gene called KMT2D contribute to medulloblastoma, a common pediatric brain tumor, with the goal of finding better ways to treat it.

  NIH research University of Tx Md Anderson Can Ctr · Houston, United States · NIH-11364198

• Targeting YBX1 to make radiation work better against high-risk medulloblastoma

  A pediatric brain cancer study tests a new approach to make radiation more effective against aggressive medulloblastoma by blocking a protein called YBX1.

  NIH research Seattle Children's Hospital · Seattle, United States · NIH-11355846

• Lipid-based therapy targeting multiple growth pathways in pediatric brain cancer

  Aimed at exploring a lipid-based medicine to slow or stop growth of medulloblastoma, a common childhood brain tumor.

  NIH research University of Nebraska Medical Center · Omaha, United States · NIH-11333316

• Using thyroid hormone to make medulloblastoma cells mature and stop growing

  Looks at whether thyroid hormone can make medulloblastoma cells mature and slow tumor growth in children with this type of brain cancer.

  NIH research Research Inst of Fox Chase Can Ctr · Philadelphia, United States · NIH-11324247

• PEP-CMV vaccine with nivolumab for pediatric and adult high-grade brain tumors

  A phase 1/2 trial tests a CMV-targeted vaccine plus a PD-1 blocker for newly diagnosed and recurrent high-grade brain tumors in children and adults.

  NIH research Washington University · Saint Louis, United States · NIH-11312693

• How the ABAT enzyme helps medulloblastoma spread in children

  This project looks at whether the enzyme ABAT helps medulloblastoma tumors in children survive and spread through the brain and spinal fluid.

  NIH research University of Southern California · Los Angeles, UNITED STATES · NIH-11310192

• How GLI2-driven medulloblastoma starts and grows in children

  Looking at whether blocking a cancer growth pathway called MAPK can slow or stop aggressive GLI2-driven medulloblastoma in children.

  NIH research Children's Research Institute · Washington, United States · NIH-11300978

• Boosting virus-based immune therapy for childhood medulloblastoma by targeting RNF2

  This project will try blocking a cancer protein called RNF2 to strengthen a virus-based immune treatment for children with medulloblastoma.

  NIH research University of Tx Md Anderson Can Ctr · Houston, United States · NIH-11271139

• Thyroid hormone (T3) as a treatment for childhood medulloblastoma

  This work will see whether the thyroid hormone T3 can slow medulloblastoma growth and be used safely for children with this brain tumor.

  NIH research Research Inst of Fox Chase Can Ctr · Philadelphia, United States · NIH-11266238

• Where different types of medulloblastoma begin in the developing cerebellum

  Researchers are tracing how Group 3 and Group 4 medulloblastoma start in the developing cerebellum to help children with these aggressive brain tumors.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11252813

• Targeting B7-H3 to slow aggressive group 3 childhood medulloblastoma

  This project tests a drug-like compound that blocks the B7-H3 protein to try to slow growth of aggressive group 3 medulloblastoma in children.

  NIH research University of Nebraska Medical Center · Omaha, United States · NIH-11252537

• Nanoparticle immune treatment for SHH‑subtype medulloblastoma

  This project tests a tiny‑particle version of an immune drug designed to help children with SHH‑subtype medulloblastoma live longer with fewer lifelong side effects.

  NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-11251558

• Blocking SIRT1 to slow childhood medulloblastoma

  This project looks at whether turning down a protein called SIRT1 can stop or slow the growth of a common childhood brain tumor called medulloblastoma.

  NIH research George Washington University · Washington, United States · NIH-11247564

• Understanding cognitive outcomes in young survivors of medulloblastoma.

  This study is looking at what affects thinking and learning in kids and young adults who have survived medulloblastoma, a type of brain tumor, to help understand why some have more trouble than others, and it's for those who have gone through this experience.

  NIH research Emory University · Atlanta, United States · NIH-11246676

• How brain support cells (glia) may cause medulloblastoma to come back

  Researchers are looking at whether a type of brain support cell called glial progenitors helps medulloblastoma return after treatment in children.

  NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11238534

• Helping high-risk medulloblastoma respond again to targeted therapy

  This project explores whether blocking two tumor enzymes can make aggressive, MYC-driven medulloblastoma in children respond again to targeted drugs.

  NIH research University of Colorado Denver · Aurora, UNITED STATES · NIH-11231704

• Immune approaches for U1‑mutated Sonic Hedgehog medulloblastoma

  This project looks at whether immune-based therapies can help people with Sonic Hedgehog medulloblastoma that carry a U1 spliceosome mutation.

  NIH research University of Tx Md Anderson Can Ctr · Houston, United States · NIH-11193455

• New CAR T-cell treatment targeting GPC2 for childhood medulloblastoma

  Engineered immune cells called CAR T cells will be directed at a protein named GPC2 to try to treat children with medulloblastoma.

  NIH research Stanford University · Stanford, United States · NIH-11192835

• Gene-control complex problems behind Group 4 medulloblastoma

  This project looks at how problems with the Core Binding Factor, a gene-controlling complex, keep developing brain cells immature and lead to Group 4 medulloblastoma in children.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11182673

• Cell behavior and treatment response in WNT-type childhood medulloblastoma

  The team is working to understand how the gene DDX3X and related changes affect tumor behavior and to develop immune-based treatments for children with WNT-subgroup medulloblastoma.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11178025

• ELP1 and SHH-type childhood medulloblastoma risk

  This work looks at whether inherited changes in the ELP1 gene increase the chance of SHH-subgroup medulloblastoma in children.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11178023

• How methyl‑adding enzymes like SMYD3 help aggressive childhood medulloblastoma

  This project looks at whether blocking a methyltransferase called SMYD3 can slow aggressive, MYC-driven medulloblastoma in children.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11178022

• Peptide vaccine targeting CMV for children with high-grade glioma, DIPG, and recurrent medulloblastoma

  This vaccine trains the immune system to target a viral protein (CMV pp65) that is often found in certain pediatric brain tumors to help slow or shrink the tumor.

  NIH research Duke University · Durham, United States · NIH-11177623

• Helping medulloblastoma tumor cells turn into mature, non-cancerous brain cells

  Looks at whether blocking a protein called EZH2 can make medulloblastoma cells in children become less harmful, brain-like tissue.

  NIH research New York University School of Medicine · New York, United States · NIH-11164621

• Immune-based treatments for aggressive Group 3 medulloblastoma

  Working to unlock the immune system to create new treatments for children with high-risk Group 3 medulloblastoma.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11162311

• Circular extrachromosomal DNA in high‑risk medulloblastoma

  Researchers are looking at whether tiny circular pieces of DNA outside chromosomes help tumors in children with high‑risk medulloblastoma become different inside and resist treatment.

  NIH research Sanford Burnham Prebys Medical Discovery Institute · La Jolla, United States · NIH-11159744

Related topics

Quick answers

How many Medulloblastoma clinical trials are recruiting right now?

18, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 6 countries. The most sites are in United States (13 trials), China (2 trials), Canada (1 trial), India (1 trial), Switzerland (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1: 5, Phase 2: 4, Phase 1/2: 4, Phase 4: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.

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