Neoplastic syndrome

This study tested a vaccine called VBI-1901 in 70 people with recurrent glioblastoma, an aggressive brain cancer. The goal was to find a safe dose and see if the vaccine could boost the immune system against the tumor. The trial was terminated early, so results are limited.

This early-phase trial is testing a new treatment called CHM-1101 for people with a type of brain cancer (glioblastoma) that has come back or is getting worse. The treatment uses the patient's own immune cells, which are modified in a lab to recognize and attack tumor cells. The …

This trial tested a vaccine called PD1-Vaxx given before surgery to people with a specific type of colorectal cancer (MSI-high). The goal was to see if the vaccine could shrink tumors, measured by how many cancer cells remained after treatment. Only 3 people took part, and the st…

This pilot study tested whether propranolol, a beta-blocker, could shrink or stabilize kidney angiomyolipomas in people with tuberous sclerosis. Only 2 participants were enrolled before the trial was terminated, so the results are very limited. The goal was to find a less invasiv…

This study tests a new treatment called Temferon for people with glioblastoma, a fast-growing brain cancer. The therapy uses a patient's own blood stem cells, modified to deliver interferon directly to the tumor. Up to 27 patients with a specific genetic marker (unmethylated MGMT…

This pilot study tested a new drug called sEphB4-HSA combined with standard chemotherapy (azacitidine or decitabine) in 7 patients with myelodysplastic syndrome, chronic myelomonocytic leukemia, or acute myeloid leukemia that had returned or not responded to prior treatment. The …

This study tested a combination of two drugs, venetoclax and azacitidine, in 34 adults with high-risk blood cancers (MDS or CMML) that returned or didn't respond to earlier treatment. The goal was to find the safest dose and see if the combo could shrink or control the cancer. Th…

This early-stage study tested a new drug called PF-07284892, alone or with other medicines, in people with advanced solid tumors that had specific genetic changes. The goal was to find the safest dose and check for side effects. The study was stopped early, so results are limited…

This early-phase trial tested whether adding the targeted drug ivosidenib to standard FLAG chemotherapy could help people with a specific genetic form of acute myeloid leukemia (IDH1-mutant AML). The study aimed to find the best dose and check for side effects in patients whose c…

This study tested a reduced-intensity bone marrow transplant, or 'mini transplant,' for people with blood cancers like lymphoma and leukemia. The transplant used marrow from a relative and was combined with chemotherapy drugs to help the donor cells take hold. Only 15 people took…

This study tested a drug called vibecotamab in adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) whose cancer did not respond to or came back after standard treatments. The drug is a special antibody designed to help the immune system attack cancer cells.…

This study looked at using umbilical cord blood transplants after strong chemotherapy and radiation to treat various blood cancers like leukemia and lymphoma. It involved 200 participants and aimed to see how many were alive one year after transplant. The trial was stopped early,…

This study tested a new drug called Vodobatinib (K0706) in people with chronic myeloid leukemia or a related leukemia who had not responded to at least three other treatments. The trial was split into parts: first in healthy volunteers to check safety, then in patients to find th…

This phase 2 trial tested two different drug combinations to prevent graft-versus-host disease (GVHD) in 150 people with blood cancers receiving a stem cell transplant from an unrelated donor. Both combinations used cyclosporine and sirolimus, plus either mycophenolate mofetil or…

This study tested whether obeticholic acid, a drug already used for liver disease, can safely reduce the number of polyps in the small bowel and colon in people with familial adenomatous polyposis (FAP). FAP is a rare genetic condition that greatly raises the risk of intestinal c…

This phase II trial tested the drug niraparib (Zejula) in 11 men with high-risk prostate cancer that had not spread. All men had specific DNA repair gene mutations. They took niraparib before having their prostate removed surgically. The goal was to see if the drug could eliminat…

This early-phase trial tested combinations of the drugs bevacizumab and temsirolimus, sometimes with valproic acid or cetuximab, in 154 people with advanced cancers or certain non-cancerous but progressive diseases. The main goal was to find safe doses and look for any signs that…

This pilot study tested the drug ADCT-301 in 3 adults whose acute myeloid leukemia, myelodysplastic syndrome, or related blood cancer had returned after a stem cell transplant. The goal was to see if the drug could safely bring about remission. However, the study was terminated e…

This early-phase trial tested the drug plinabulin combined with radiation and immunotherapy in 19 people with advanced cancers (like lung, bladder, or melanoma) that had stopped responding to standard immunotherapy. The goal was to see if the combination was safe and could shrink…

This early-phase study tested a new drug called AUR103 Calcium in 27 adults with advanced solid tumors, acute myeloid leukemia, myelodysplastic syndromes, or non-Hodgkin lymphoma who had no other treatment options. The goal was to find the best dose and check safety. The study wa…

This early-stage trial tested a new drug called gentulizumab in 58 people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that had come back or stopped responding to treatment. Gentulizumab is an antibody that helps the immune system attack cancer cells. The m…

This study tested two different drug combinations given before a stem cell transplant for people with myeloid blood cancers like leukemia. One group got fludarabine and busulfan, the other got those drugs plus low-dose total body irradiation. The goal was to see which regimen led…

This study tested a combination of chemotherapy drugs (cladribine, cytarabine, mitoxantrone, and G-CSF) in 53 adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) that had come back or not responded to prior treatment. The goal was to see how many …

This early-stage study tested an experimental drug called INKmune in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The goal was to find a safe dose and check for side effects. However, the study was stopped early after only 2 people enrolled, so no c…

This early-phase trial tested an experimental drug called SL-172154, given alongside standard chemotherapy drugs, for people with higher-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The main goals were to check safety and find the right dose. The study was…

This early-phase trial tested a new treatment for adults with acute myeloid leukemia that had come back or not responded to standard therapy. The treatment involved giving patients natural killer cells grown from donated umbilical cord blood, along with a drug called IL-2 to help…

This phase II trial tested a combination of two drugs, venetoclax and decitabine, in 235 patients with certain blood cancers like acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS) that had come back or not responded to treatment, as well as in older patien…

This early-phase study tested two chemotherapy drugs, pevonedistat and belinostat, in 18 adults with acute myeloid leukemia or myelodysplastic syndrome that returned or didn't respond to prior treatment. The main goal was to find the safest dose and understand side effects. The s…

This early-stage trial tested a new drug called DFF332, which targets a protein (HIF2α) that helps some cancers grow. The study included 40 adults with advanced clear cell renal cell carcinoma or other cancers with specific gene changes. DFF332 was tested alone and in combination…

This study tested a drug called MBG453 in 10 people with a type of bone marrow disorder called lower-risk myelodysplastic syndromes (MDS). The goal was to see if the drug could improve blood cell counts and reduce the need for transfusions. The trial was terminated early, so the …

This phase 2 trial tested the drug guadecitabine (SGI-110) in people aged 12 and older with rare cancers like wild-type GIST, pheochromocytoma, paraganglioma, and HLRCC-related kidney cancer that had not responded to other treatments. Participants received daily injections for 5 …

This study tested a combination of two drugs, venetoclax and azacitidine, for people with a high-risk type of bone marrow cancer called myelodysplastic syndrome (MDS) that either came back or didn't respond to treatment. The goal was to find the best dose and see how well it work…

This early-phase study tested whether a special type of cell (interferon gamma-primed mesenchymal stromal cells) could be safely given to prevent graft-versus-host disease, a common and serious complication after bone marrow transplant. Only 4 patients with acute leukemia or myel…

This early-phase study tested two drugs, AZD1775 and belinostat, together in 20 adults with blood cancers like acute myeloid leukemia that had come back or not responded to treatment. The main goal was to find the safest dose and understand side effects. The study was stopped ear…

This early-stage trial tested a new drug called NC525 in people with advanced myeloid neoplasms, including relapsed or refractory acute myeloid leukemia. The main goal was to check safety and tolerability, with a secondary focus on whether the drug could shrink tumors or improve …

This study tested leflunomide, a drug used for rheumatoid arthritis, in 3 adults with MEN1 syndrome—a genetic condition that raises the risk of multiple tumors. The goal was to see if 6 months of daily leflunomide could shrink or stabilize tumors. However, the trial was terminate…

This study tested a combination of two drugs (regorafenib and pembrolizumab) for people with a specific type of advanced colorectal cancer called MSI-H. The goal was to see if the combination worked better than pembrolizumab alone. The trial was stopped early after enrolling only…

This study tested the safety of giving a second dose of an experimental virus (C134) directly into the brain tumor of patients whose malignant glioma had returned. Only one patient was enrolled before the study was stopped. The goal was to see if repeat dosing was safe and tolera…

This early-phase trial tested an experimental drug called TAK-500, alone or with the immunotherapy pembrolizumab, in 61 adults with advanced solid tumors like pancreatic, liver, and lung cancer. The main goals were to check safety and see if the drug could shrink tumors. The stud…

This study tested whether adding sabatolimab to standard chemotherapy (azacitidine) helps people with high-risk myelodysplastic syndrome (MDS) or a related leukemia (CMML-2). The trial aimed to see if the combination improved survival. However, the study was stopped early, so we …

This study tested adding an experimental drug called MBG453 to standard chemotherapy for people with a serious bone marrow disorder called myelodysplastic syndrome (MDS). The trial included 127 adults with high-risk MDS who could not have a stem cell transplant. The goal was to s…

This early-stage study tested a radioactive drug called 177Lu-FF58 in 24 people with advanced pancreatic, gastroesophageal, or brain cancer. The drug seeks out and attaches to specific proteins on cancer cells to deliver radiation directly. The main goals were to check safety, fi…

This study tested a new drug called SAR443579 in adults and children with certain blood cancers that had come back or not responded to treatment. The goal was to see if the drug was safe and could shrink or control the cancer. The trial was stopped early, so results are limited.

This study tested a drug called basmisanil in children aged 2 to 14 with Dup15q syndrome, a rare genetic condition that affects development. The goal was to see if the drug could improve daily living skills like communication and movement. The trial was stopped early after enroll…

This early-phase trial tested the drug AR-42 in 7 adults with vestibular schwannoma or meningioma brain tumors who were already scheduled for surgery. Participants took AR-42 pills for about 3 weeks before their operation. The main goal was to see if the drug could lower a protei…

This study tested two oral drugs, eltanexor and Inqovi, in adults with high-risk myelodysplastic syndromes (MDS) that did not improve with standard treatment. MDS is a bone marrow disorder that can lead to leukemia. The goal was to find a safe dose and see if the combination help…

This study tested a sirolimus cream (0.2% and 0.4%) applied daily for 12 weeks to treat facial angiofibromas in children aged 2-21 with tuberous sclerosis complex. The goal was to see if the cream could safely reduce the size and redness of these skin growths. The trial was termi…

This early-phase study tested an experimental drug called BR108 in 8 adults with advanced blood cancers (lymphoma, leukemia, or myelodysplastic syndrome) that had not responded to prior treatments. The drug was given by IV infusion twice per cycle. The main goals were to check sa…

This early-stage trial tested a new drug called DSP107 combined with standard chemotherapy drugs (azacitidine and venetoclax) in 25 people with relapsed or hard-to-treat acute myeloid leukemia, myelodysplastic syndrome, or chronic myelomonocytic leukemia. The study aimed to check…

This study tested a bone marrow transplant using a partially matched family donor for people with blood cancers like leukemia. The goal was to see if a milder conditioning regimen and a drug called cyclophosphamide could help patients survive at least 180 days after transplant. T…

This study tested a new drug called INCB059872 in people with advanced solid tumors or blood cancers that had not responded to standard treatments. The goal was to find a safe dose and see if it could control the disease. The trial was stopped early, so results are limited.

This early-phase trial tested a combination of two drugs, omacetaxine and venetoclax, in 24 adults with relapsed or refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that had a specific genetic change called RUNX1. The goal was to find the best dose and se…

This early-phase trial tested a new drug called INCB062079 in 25 people with advanced liver cancer and other solid tumors. The main goal was to check safety and find the best dose. The study was terminated early, so results are limited.

This phase 2 trial tested a daily pill called etavopivat in 17 adults with very low, low, or intermediate risk MDS who had anemia. The goal was to see if the drug could improve red blood cell counts and reduce the need for transfusions. The study was terminated early, so the full…

This early-phase trial tested whether adding the drug itacitinib to standard immune-suppressing medications could help prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers. The study enrolled 84 participants with leukemia, lymphoma,…

This study tested a daily pill called INCB000928 for people with anemia caused by myelodysplastic syndromes (MDS) or multiple myeloma. The goal was to see if it was safe and could raise red blood cell levels, reducing the need for transfusions. The trial was terminated early afte…

This phase 2 study tested adding sabatolimab, an experimental immunotherapy, to standard chemotherapy drugs (azacitidine, decitabine, or oral decitabine) in 39 adults with intermediate to very high risk myelodysplastic syndrome (MDS). The main goal was to check safety, and the tr…

This early-stage trial tested two new drug combinations in 52 adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). One group received HDM201 plus MBG453, the other HDM201 plus venetoclax. The main goal was to check safety and find the right dose. …

This phase 2 trial tested a new drug called sabatolimab, combined with two standard chemotherapy drugs (azacitidine and venetoclax), in 20 adults with high or very high risk myelodysplastic syndromes (MDS) who could not have intensive chemotherapy or a stem cell transplant. The g…

This early-phase study tested combinations of three antibody drugs (sabatolimab, NIS793, and canakinumab) in 33 adults with lower-risk myelodysplastic syndrome (MDS), a blood disorder. The main goal was to check safety and find the right doses. The study was terminated early, so …

This study tested a drug called REC-2282 in people aged 12 and older with progressive meningiomas (brain tumors) linked to NF2 gene mutations. Participants took the drug or a placebo three times a week in cycles. The goal was to see if the drug could slow tumor growth. The trial …

This early-phase trial tested a new drug called BMS-986253, alone or with standard chemotherapy, for people with myelodysplastic syndromes (MDS), a group of bone marrow disorders. The study aimed to find a safe dose and see if the drug could shrink cancer. However, the trial was …

This study tested a treatment called TCB008, made from donated immune cells, for adults with acute myeloid leukemia (AML) that came back or didn't respond to standard therapy. The goal was to see if it could reduce cancer cells in the bone marrow. The trial was stopped early, but…

This study looked at a new way to prevent severe graft-versus-host disease (GVHD) in children who had a bone marrow transplant for blood cancers. The goal was to see if stopping certain immune-suppressing drugs after day 60 could lower the risk of severe GVHD. Only 3 children too…

This phase 3 study tested the drug ganaxolone as an add-on treatment for seizures in children and adults with tuberous sclerosis complex (TSC). The trial was open-label, meaning everyone received the drug, and it included 117 people who had previously been in related studies. The…

This study tested a targeted cancer drug called ponatinib combined with standard chemotherapy in children and young adults up to age 21 with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) that had come back or stopped responding to other treatments. The g…

This early-stage study tested a new drug called CC-95251, alone or with other cancer drugs, in people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that had come back or not responded to treatment. The main goals were to check safety and find the right dose…

This early-stage trial tested an experimental oral drug called SGR-2921 in adults with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The main goals were to check safety and find the right dose. However, the study was terminated e…

This study tested a new drug called VAL-083 in people with glioblastoma, an aggressive brain cancer that had returned after standard treatments. The trial aimed to see if VAL-083 could help patients live longer. However, the study was stopped early after only 2 people enrolled, s…

This early-phase trial tested a radioactive imaging agent called 68Ga-FF58 in 14 adults with certain advanced solid tumors (glioblastoma, brain metastases from breast cancer, gastroesophageal cancer, or pancreatic cancer). The goal was to see if the agent could help detect these …

This pilot study tested the blood pressure drug telmisartan (Micardis) to prevent acute graft-versus-host disease (GVHD) in people receiving a stem cell transplant for blood cancers. The trial aimed to enroll 60 participants but was terminated early with 32. Researchers tracked s…

This study tested a cannabidiol (CBD) oral solution in children under 2 years old with tuberous sclerosis complex, Dravet syndrome, or Lennox-Gastaut syndrome who had uncontrolled seizures. The goal was to see if CBD is safe and can reduce seizures. However, the study was termina…

This study looks at how the drug luspatercept is used in real-world clinics for patients with lower-risk myelodysplastic syndromes (MDS) or beta-thalassemia. Researchers will track patient characteristics, treatments, and outcomes like reduced need for blood transfusions. The goa…

This study aimed to find genetic mutations in people with idiopathic cytopenia of undetermined significance (ICUS), a condition of unexplained low blood cell counts. Researchers wanted to see if these mutations are similar to those found in myelodysplastic syndrome (MDS), a type …

This study reviewed medical records of 42 people with familial adenomatous polyposis (FAP) who had previously taken the drug erlotinib. Researchers wanted to see how quickly the disease progressed after the drug was stopped. The goal was to better understand what happens when tre…

This study looked at whether a liquid biopsy—testing the fluid around the brain and spinal cord—can find tumor DNA in children and young adults with brain tumors. The goal was to see if this safer, non-surgical test could replace risky tissue biopsies for diagnosis and monitoring…

This very early study aimed to compare an oral (pill) version of the chemotherapy drug cladribine with the standard IV (vein) version in people with certain leukemias. Only 3 people took part before the study was stopped early. The main goal was to see how the body handles the or…

This study tested a new lab method (RAD51 assay) to see if it could identify ovarian cancer patients whose tumors are vulnerable to the drug olaparib, even if they don't have BRCA gene mutations. The goal was to find a larger group of patients who might benefit from this treatmen…

This early-phase trial tested whether posaconazole, a drug used for fungal infections, can reach brain tumors in high enough amounts to stop cancer cells from dividing. Seven adults with glioblastoma took the drug before surgery, and researchers measured drug levels in their spin…

This study looked at how moderate or severe liver problems change the amount of the cancer drug oral azacitidine in the blood. It was designed for adults with certain blood cancers. The study was stopped early after only 2 people enrolled, so the results are very limited.

This early-phase trial tested whether ketoconazole, a drug normally used for fungal infections, can enter brain tumors in high enough amounts to stop tumor cells from dividing. Only 1 person was enrolled before the study was terminated. The goal was to measure drug levels in the …

This early-stage study looked at how two common drugs (carbamazepine and itraconazole) change the levels of radiprodil in the blood of 30 healthy adults. The goal was to understand safety and dosing for future use in neurological conditions like tuberous sclerosis. However, the s…