Diffuse intrinsic pontine glioma

This study tests a personalized DNA vaccine for children and young adults (ages 12-25) with central nervous system tumors that have come back or not responded to treatment. The vaccine is made from each patient's unique tumor DNA to train the immune system to fight the cancer. Th…

This study tests an experimental drug called ACT001 in children and young adults (ages 1 to 39) with two aggressive brain tumors: DIPG and H3K27-altered high-grade gliomas. The goal is to see if ACT001 can improve survival or shrink tumors after radiation therapy. About 60 partic…

This trial tests a drug called selinexor combined with standard radiation in children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG) or high-grade glioma (HGG) that has a specific genetic change (H3 K27M mutation). The study has two parts: first, fi…

This study tests a new drug called CBL0137 in children and young adults (ages 1 to 21) whose solid tumors, brain tumors, or lymphoma have returned or stopped responding to treatment. The goal is to find the best dose, check side effects, and see if the drug can shrink tumors. The…

This early-stage trial tests whether using focused ultrasound with microbubbles can temporarily open the blood-brain barrier, allowing the immunotherapy drug nivolumab to reach brain tumors more effectively. It includes 30 children and young adults with relapsed diffuse midline g…

This early-phase study tests the safety of an engineered virus called M032 in 20 children and adults with diffuse midline glioma, a rare and aggressive brain tumor. Participants receive the virus directly into the tumor after completing standard radiation therapy. The goal is to …

This study tests two oral drugs, eflornithine (DFMO) and AMXT 1501, in children and young adults up to age 26 with certain cancers that have come back or not responded to treatment. The cancers include neuroblastoma, brain tumors, and sarcomas. The goal is to find a safe dose and…

This early-phase trial tests the safety and best dose of the drug triapine when given together with radiation therapy for people whose glioblastoma or astrocytoma has come back. Triapine works by blocking enzymes that cancer cells need to grow, while radiation kills cancer cells …

This study tests whether adding the drug olutasidenib to standard chemotherapy (temozolomide) can help children and young adults with a specific type of brain cancer (IDH1-mutant high-grade glioma) live longer. About 60 participants will receive the combination as maintenance the…

This early-phase study tests the drug TURALIO (pexidartinib) in people aged 3 to 35 whose cancer has not responded to standard treatments. The goal is to find a safe dose and see if it can shrink tumors or control leukemia. Participants take a daily capsule for up to 2 years, wit…

This Phase 2 trial is testing a vaccine called PEP-CMV in 120 children and young adults with high-grade glioma, diffuse intrinsic pontine glioma (DIPG), or recurrent medulloblastoma. The vaccine targets a common virus (CMV) found in many brain tumors, aiming to boost the immune s…

This phase 3 trial tests whether the drug ONC201 works better than everolimus for children and adults with DIPG, a rare and aggressive brain tumor. About 433 participants will receive one of the two drugs, and can switch if their disease gets worse. The goal is to see which drug …

This study tests a new treatment called BCB-276 for children and young adults with diffuse intrinsic pontine glioma (DIPG), a rare and aggressive brain tumor. BCB-276 is made from a patient's own immune cells, which are modified in a lab to recognize and attack the cancer. The tr…

This early-phase trial is testing whether adding the drug atovaquone to standard radiation therapy is safe and tolerable for children with certain malignant brain tumors, including high-grade glioma, diffuse midline glioma, and medulloblastoma. The study includes two groups: chil…

This early-phase trial tests a new type of immune therapy for people with certain aggressive brain tumors, including DIPG and high-grade glioma. The treatment uses the patient's own T cells, which are modified in the lab to recognize a protein called GD2 on cancer cells and to pr…

This study is testing a drug called Lutathera in children and young adults (ages 4 to 39) whose brain tumors have come back or are growing despite treatment. The drug delivers radiation directly to tumor cells that show up on a special PET scan. The goal is to find the safest dos…

This early-stage trial tests a personalized cell therapy for children with recurrent neuroblastoma or newly diagnosed DIPG, two aggressive cancers. The treatment uses the child's own immune cells (dendritic cells and T cells) and stem cells, trained to attack their specific tumor…

This early-phase study tests the drug lorlatinib in children and young adults (ages 1 to 21) with newly diagnosed high-grade glioma, a fast-growing brain tumor, that has a specific genetic change (ALK or ROS1 fusion). The goal is to see if the drug can shrink or control the tumor…

This phase 2 trial is testing whether combining the drug ONC201 with other treatments (like paxalisib or a cancer-killing virus called DNX-2401) can help children and young adults with diffuse midline gliomas, a rare and aggressive brain cancer. The study enrolls 360 participants…

This early-phase trial tests a new drug called ONC206 in children and young adults with diffuse midline glioma or other recurrent brain tumors. The drug aims to kill cancer cells by causing stress in them, without harming healthy cells. Researchers are looking for the safest dose…

This study tests a new cancer drug, repotrectinib, combined with standard chemotherapy in children and young adults with advanced solid tumors that have spread. The goal is to find the safest dose and see if the combination can shrink tumors. Researchers will also study how the b…

This study is testing a combination of two drugs—a targeted radioactive drug (177Lu-DOTATATE) and a PARP inhibitor (olaparib)—in children and teens aged 3 to 18 with certain solid tumors that have come back or not responded to treatment. The goal is to see if the combination is s…

This study tests whether adding two different drug combinations after radiation can help children and young adults (up to age 21) with high-grade gliomas, including DIPG, live longer. One group receives ribociclib plus everolimus; another gets ribociclib plus temozolomide. The go…

This study tests whether adding the drug VRT106 to standard radiation therapy can help control a rare, fast-growing brain tumor called diffuse midline glioma (including DIPG) in adults. About 10 participants aged 18 to 75 will receive the combination treatment. The main goal is t…

This study tests a new drug called Becotatug Vedotin in children aged 2 to 18 with certain solid tumors that have come back or spread. The drug targets a protein called EGFR found on many cancer cells. The main goal is to see if the drug is safe and to find the best dose, while a…

This early-phase study tests whether a new type of immunotherapy, called B7-H3 CAR T cells, can be safely given directly into the brain or spinal fluid of children and young adults with aggressive brain tumors like DIPG, DMG, or other recurrent central nervous system cancers. The…

This early-phase trial is testing a new vaccine (rHSC-DIPGVax) made from pieces of tumor proteins, given together with two immunotherapy drugs (balstilimab and zalifrelimab), in children with DIPG or diffuse midline glioma. The goal is to see if the combination is safe and tolera…

This early-phase trial is testing the drug larotrectinib in 15 children (up to age 21) with newly diagnosed high-grade glioma or DIPG that have a specific genetic change called NTRK fusion. The study aims to see if the drug can control the disease after two cycles, and how safe i…

This early study tests whether focused ultrasound can safely open the blood-brain barrier in children with progressive diffuse midline glioma (DMG), a rare and aggressive brain tumor. Ten children aged 4–21 will receive oral etoposide chemotherapy after ultrasound treatment to al…

This study tests whether giving two drugs (cetuximab and bevacizumab) directly into the brain's blood vessels each month is safe and can shrink tumors or slow their growth. It includes children and young adults under 22 with certain types of brain tumors that have come back or no…

This study tests an oral drug called LAM561 in children under 18 with advanced brain tumors (like high-grade glioma) or other solid tumors that have not responded to standard treatments. The trial has two phases: first, finding a safe dose in up to 18 children, then testing that …

This study tests whether a special PET scan called 18F-Fluciclovine can help doctors tell the difference between actual tumor growth and harmless changes caused by treatment in children with high-grade glioma. About 30 children aged 1 to 21 will receive the scan after radiation t…

This study uses genetic testing of brain tumors from children, adolescents, and young adults (ages 1 to 39) with newly diagnosed high-grade glioma, including DIPG. The goal is to identify specific genetic changes in each tumor and then see if patients can join a treatment trial t…

This study is testing whether an artificial intelligence (AI) model can accurately predict the molecular subtype of midline gliomas (a type of brain tumor) using MRI scans taken before surgery. Researchers will compare the AI's predictions with standard lab tests after surgery to…

This study creates a central registry and tissue bank for people with DIPG or DMG, rare and aggressive brain tumors. Researchers will collect medical information, scans, and tumor samples from up to 1,500 patients of any age. The goal is to understand the biology of these tumors …