Neoplastic syndrome

This study tests a new drug, AZD9829, in people with blood cancers that have come back or not responded to other treatments. The drug targets a protein called CD123 found on cancer cells. The main goals are to find a safe dose and check for side effects. About 56 adults will take…

This study tests a 5-week community-based program that teaches protective behavioral strategies to reduce alcohol use in men aged 35–44 with hazardous drinking habits in rural Vietnam. Participants learn to set safe drinking limits, choose nonalcoholic drinks, and plan ahead, wit…

This study tests a drug called FT-2102 (olutasidenib) in people with acute myeloid leukemia or myelodysplastic syndrome whose cancers have a specific IDH1 gene mutation. The drug is given alone or combined with standard chemotherapy drugs (azacitidine or cytarabine). The goal is …

This study tested whether adding the drug ZARNESTRA to standard radiation therapy can slow tumor growth in people with glioblastoma, an aggressive brain cancer. Twenty-seven patients took the drug daily starting a week before radiation and continuing through treatment. The main g…

This study tested the safety of combining two drugs, alpelisib and fulvestrant, in 40 men and post-menopausal women with a certain type of advanced breast cancer (HR+, HER2-, with a PIK3CA mutation) that had worsened after hormone therapy. The goal was to track side effects. The …

This large study tested whether evolocumab (Repatha), a cholesterol-lowering injection, could reduce major cardiovascular events like heart attacks, strokes, or death in over 12,000 adults at high risk but without a prior heart attack or stroke. Participants received either evolo…

This phase III trial tests whether the drug tipifarnib can prevent acute myeloid leukemia (AML) from coming back in patients who are already in remission. Participants include adults over 60 in first remission, those in second or later remission, or those who achieved remission a…

This study tested a daily pill called E7820 in 12 adults with relapsed or treatment-resistant acute myeloid leukemia, myelodysplastic syndromes, or chronic myelomonocytic leukemia. All participants had specific mutations in splicing factor genes. The goal was to see if the drug c…

This study tested the drug RAD001 (everolimus) in 29 patients with relapsed or hard-to-treat blood cancers like leukemia, lymphoma, and myelofibrosis. The goal was to find the safest highest dose and see if it helps control the disease. It was a small early-phase trial at a singl…

This study reviewed medical records of 37 adults with Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL) who took asciminib because other treatments stopped working or caused side effects. Researchers wanted to see how many patients achieved complete remission within th…

This study offers ongoing treatment with alectinib or crizotinib to about 200 adults with ALK- or RET-positive cancer who were already benefiting from these drugs in a previous Roche trial. The main goal is to monitor safety and side effects as patients continue therapy after the…

This study tested a drug called fedratinib in 25 adults with rare blood cancers (MDS/MPN and CNL). The goal was to see if the drug could shrink the spleen and improve blood counts. While not a cure, the treatment aims to control the disease and reduce symptoms.

This study tested a new drug called pelabresib in people with certain blood cancers, including myelofibrosis and leukemia. In the first part, the drug was given alone to find the safest dose. In the second part, it was given with or without another drug (ruxolitinib) to see if it…

This study tested whether N-acetylcysteine (NAC), an antioxidant drug, can improve motor skills and learning in children with neurofibromatosis type 1 (NF1). Twenty-five children aged 8 to 16 took NAC or a placebo for 8 weeks. The goal was to see if NAC is safe and helps with mov…

This study tested a new way to treat blood cancers like leukemia in 16 adults who did not have a standard bone marrow donor. Doctors used a single unit of cord blood that was grown in a lab to increase the number of stem cells before transplant. The goal was to see if this method…

This study tested a new surgical technique in 12 patients with newly diagnosed glioblastoma, an aggressive brain cancer. Surgeons placed a tissue flap from the scalp into the brain tumor cavity to bypass the blood-brain barrier, which normally blocks many treatments. The main goa…

This study tested giving chemotherapy before surgery to children with Wilms tumor, a rare kidney cancer. The goal was to shrink tumors so surgeons could remove less kidney tissue, helping preserve kidney function. 249 children took part, and the approach aimed to reduce the need …

This phase 2 trial tested a combination of intravenous busulfan, etoposide, and low-dose total-body irradiation before a donor stem cell or bone marrow transplant in 30 patients with advanced leukemia or myelodysplastic syndromes. The goal was to see how well this regimen control…

This study tested whether removing a specific type of immune cell (naïve T cells) from donor stem cell grafts could prevent graft-versus-host disease (GVHD) after a stem cell transplant for blood cancers. The trial enrolled 84 patients who received high- or medium-intensity chemo…

This early-phase trial tested a drug called ONC-201 in 20 people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who had recently received a stem cell transplant. Participants took the drug by mouth once a week for a year to see if it was safe and co…

This phase 2 trial tested two drugs, selumetinib and sirolimus, in 21 people with advanced malignant peripheral nerve sheath tumors (MPNST), a rare cancer often linked to neurofibromatosis type 1. The goal was to see if the combination could shrink or stabilize tumors. The study …

This study tested whether adding the drug tocilizumab to standard care could reduce the risk of graft-versus-host disease (GVHD) after a cord blood transplant in adults with blood cancers. GVHD is a serious complication where donor cells attack the patient's body. Tocilizumab blo…

This study tested a three-drug combination (navitoclax, venetoclax, and decitabine) in 6 adults with high-risk myelodysplastic syndrome (MDS) that came back or didn't respond to standard therapy. The goal was to find the safest dose and see if the combo could shrink or control th…

This study looked at 88 adults in China with lower-risk myelodysplastic syndromes (MDS) who had not received prior treatment with erythropoiesis-stimulating agents. They were given luspatercept, and researchers tracked how many became free from red blood cell transfusions and whe…

This study tested a drug called ustekinumab in 8 people with a rare, severe form of Behçet's syndrome affecting the gut, along with a blood disorder called myelodysplastic syndrome. The goal was to see if the drug could control gut symptoms and reduce disease activity over 24 wee…

This phase 1 trial tested two immunotherapy drugs, nivolumab and ipilimumab, in 29 patients whose acute myeloid leukemia or myelodysplastic syndrome came back or did not respond after a donor stem cell transplant. The goal was to find the safest dose and see if the drugs could he…

This study tested a drug called pamiparib, given with radiation therapy, for people with a fast-growing brain cancer called glioblastoma. It included 50 patients, some newly diagnosed and some whose cancer had come back. The goal was to see how much drug reaches the tumor and whe…

This study tested the drug sunitinib (Sutent) in 71 people with advanced thyroid cancer that had spread or could not be removed by surgery. The goal was to see if the drug could shrink tumors when standard treatments like radioactive iodine no longer work. Participants took sunit…

This study tested whether an oral medication (CC-486) could help elderly patients with a serious blood disorder called myelodysplastic syndrome (MDS) maintain their improvement after initial injectable treatment. Eleven patients who had responded to injectable azacitidine switche…

This study tested a drug called luspatercept in 30 adults from China and Japan who have a bone marrow disorder (MDS) and need regular red blood cell transfusions. The goal was to see if the drug could help them go without transfusions for at least 8 weeks. The drug works by helpi…

This study tested a procedure called radiofrequency ablation, which uses heat to destroy a small adrenal gland tumor that causes high blood pressure. Researchers wanted to see if it could safely lower blood pressure in 31 patients with primary aldosteronism. The goal was to reduc…

This early-stage study tested a new drug called LYT-200 in 101 adults whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) had come back or stopped responding to treatment. The main goals were to check the drug's safety and find the right dose. While not…

This study tested pegloticase, a drug used for gout, in 10 cancer patients with high uric acid from tumor lysis syndrome. The goal was to see if it could quickly lower uric acid levels and protect kidney function. It was a small pilot trial to gather early data.

This early-stage trial tested an oral drug called NTX-301 in 22 adults with certain blood cancers (MDS, AML, or CMML) that had not responded to prior treatments. The main goal was to check safety and find the right dose. Researchers also looked for signs that the drug might help …

This study tested different drug combinations to prevent graft-versus-host disease (GVHD) in 174 people with blood cancers receiving stem cells from an unrelated donor. GVHD is a serious side effect where donor cells attack the patient's body. The goal was to find which drug regi…

This early-phase trial tested a combination of two cancer drugs, bortezomib and clofarabine, in 28 adults with advanced solid tumors, lymphomas, or myelodysplastic syndromes that had stopped responding to standard therapy. The main goal was to find the safest dose and check for s…

This early-phase trial tested the drug enasidenib in 17 people with low-risk myelodysplastic syndrome or a related leukemia who had anemia and did not have a specific gene mutation. The goal was to see if the drug could safely improve red blood cell counts and reduce the need for…

This study tested a two-step stem cell transplant for people with blood cancers like leukemia and lymphoma. First, patients received low-dose chemotherapy and radiation to prepare their bodies. Then, they got donor stem cells followed by drugs to prevent the donor cells from atta…

This pilot study tested a device called NaviFUS that uses focused ultrasound to temporarily open the blood-brain barrier, combined with the drug bevacizumab (Avastin), in 6 patients with recurrent glioblastoma (a type of brain cancer). The goal was to see if the combination is sa…

This phase 2 trial tested whether adding the drug veliparib to standard chemotherapy (cisplatin) helps people with metastatic triple-negative or BRCA-related breast cancer, including those with brain metastases. The study enrolled 344 participants to see if the combination slows …

This study tested a personalized geriatric optimization plan (GO!) in 30 adults aged 60 and older who were getting a donor stem cell transplant for blood cancers or bone marrow failure. The plan included tailored advice on diet, sleep, activity, and medications to improve physica…

This study tested whether adding pacritinib to standard immune-suppressing drugs (sirolimus and tacrolimus) could prevent graft-versus-host disease (GVHD) in 40 patients receiving stem cell transplants for blood cancers. GVHD occurs when donor immune cells attack the recipient's …

This study tested an oral drug called ASTX727 in people with lower-risk myelodysplastic syndromes (MDS), a bone marrow disorder. The goal was to see if a lower dose given over a longer schedule could improve blood cell counts and reduce the need for transfusions. The trial enroll…

This study tested a device called Optune, which delivers electric fields to the brain, alongside radiation and chemotherapy for people newly diagnosed with glioblastoma, an aggressive brain cancer. About 981 adults took part. The goal was to see if adding Optune to standard treat…

This phase II trial tested whether adding the drug itacitinib to standard medications (tacrolimus and sirolimus) can better prevent graft-versus-host disease (GVHD) in 59 patients with blood cancers receiving donor stem cell transplants. GVHD occurs when donor immune cells attack…

This study tested a personalized vaccine made from a patient's own tumor cells and immune cells (dendritic cells) for people with newly diagnosed glioblastoma, an aggressive brain cancer. The goal was to see if the vaccine is safe and can help delay tumor growth. 76 participants …

This study tested a new method of cord blood transplantation in 13 children and young adults with high-risk myeloid cancers. The approach uses a molecule called UM171 to grow more stem cells from a single cord blood unit, aiming to speed up recovery and reduce complications. Earl…

This study looked at whether a modified Atkins diet combined with intermittent fasting is possible and has any effect in 25 people with glioblastoma, an aggressive brain cancer. Participants followed the diet for several weeks while researchers checked if they could stick with it…

This study tested a new way to prepare cord blood for transplant in 30 people with high-risk leukemia or myelodysplasia. The cord blood was treated with a molecule called UM171 to help it grow more stem cells. The goal was to see if this approach is safe and helps patients recove…

This phase 2 trial tested a two-step stem cell transplant approach for 47 patients with high-risk blood cancers like leukemia and lymphoma. The method uses the drug cyclophosphamide to lower the chance of graft-versus-host disease (GVHD), a common complication. The goal was to se…

This early-phase trial tested a new type of stem cell transplant called a microtransplant in 25 people with myeloid blood cancers (like leukemia) who were too old or frail for a standard transplant. Unlike a regular transplant, this one used stem cells from a partially matched fa…

This phase 2 trial tested the drug crizotinib in 12 people with neurofibromatosis type 2 (NF2) who had growing tumors in the ear area called vestibular schwannomas. Participants took crizotinib pills daily for up to 12 months. The main goal was to see if the drug could shrink the…

This phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…

This study tested whether matching chemotherapy intensity to a patient's genetic risk and overall health could improve outcomes for older adults (60+) with acute myeloid leukemia. 75 participants received either standard intensive or lower-intensity chemo based on their individua…

This early-phase trial tested a combination of two drugs, ibrutinib and lenalidomide, in 4 patients with myelodysplastic syndrome (MDS) who had not responded to or could not tolerate standard treatments. The main goal was to find the safest dose and identify side effects. While t…

This study tested whether physiotherapist-led pelvic floor and bowel training could improve bowel function in people who had ileo-anal pouch surgery for ulcerative colitis or familial adenomatous polyposis. Fifty-three participants were randomly assigned to either the behavioural…

This pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…

This study looked at whether a custom-made hip spacer can help people with infected hip replacements. 25 patients had their infected hip implant removed and replaced with a temporary custom spacer, followed by antibiotics. After the infection cleared, they received a new permanen…

This early-phase trial tested a cancer vaccine (CDX-1401) along with an immune booster, chemotherapy, and an immunotherapy drug in 8 people with myelodysplastic syndrome (MDS) or low-blast acute myeloid leukemia (AML). The goal was to see if the combination is safe and helps the …

This Phase 3 trial tested a special cord blood product called NiCord (omidubicel) against standard cord blood transplants in 125 people with blood cancers like leukemia and lymphoma. The goal was to see if NiCord helps the body rebuild its infection-fighting cells faster. Researc…

This early-phase trial tested a new treatment for people with myelodysplastic syndrome (MDS) or secondary acute myeloid leukemia (AML) who had not responded to prior therapy. The treatment involved giving specially prepared donor immune cells (with CD8 cells removed to reduce ris…

This phase 2 trial tested a gel called NFX-179 on skin tumors in 199 adults with neurofibromatosis type 1. Participants applied the gel daily for 26 weeks. The study measured safety and whether tumors shrank in size or height. Results suggest the gel may help reduce tumor volume,…

This study tested a gentler bone marrow transplant for people with blood cancers who don't have a perfectly matched family donor. Instead, it used donors who are less closely matched, like unrelated volunteers or distant relatives. The goal was to see if this approach could be sa…

This study tested a drug called dasatinib combined with standard chemotherapy in 30 adults newly diagnosed with a fast-growing leukemia (Ph+ ALL). The goal was to see if the combination helps people live longer and keeps the cancer away. Patients took dasatinib daily for up to 2 …

This phase II study tested a combination of three drugs—ibrutinib, fludarabine, and pembrolizumab—in 15 people with high-risk or relapsed/refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The goal was to see if the combo could produce a complete r…

This early-stage study tested a new drug called PRT2527, alone or with other cancer drugs (zanubrutinib or venetoclax), in 86 people with various blood cancers that had returned or stopped responding to standard treatments. The main goals were to check safety, find the best dose,…

This early-phase study tested an experimental oral drug called LP-108, given alone or with another drug (azacitidine), in 32 adults with blood cancers (MDS, CMML, or AML) that returned or didn't respond to prior therapy. The main goals were to find the safest dose and understand …

This early-phase trial tested a combination of two chemotherapy drugs, bortezomib (Velcade) and lenalidomide (Revlimid), in 22 adults whose acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) had returned after a stem cell transplant. The main goal was to find the safe…

This early-phase study tested the drug regorafenib in 16 adults with advanced blood cancers like acute myeloid leukemia (AML) that had stopped responding to other treatments. The main goal was to find the safest dose and see how the drug affects cancer-related cell signals. While…

This early-phase trial tested a combination of three drugs—olaparib, palbociclib, and fulvestrant—in 9 people with BRCA-mutated, hormone-positive, HER2-negative metastatic breast cancer. The main goal was to see if the combination is safe. Researchers also looked at how long the …

This early-phase study tested an experimental drug called INCB7839 in 13 children whose high-grade gliomas (aggressive brain tumors) had come back or worsened after standard treatment. The main goals were to find a safe dose and check for side effects. The drug works by blocking …

This phase 2 trial tested the drug sirolimus (rapamycin) in 61 children and young adults with complicated vascular anomalies—abnormal blood vessel growths that can cause pain, bleeding, or organ problems. The goal was to see if sirolimus could safely shrink these anomalies and im…

This study tested whether a low dose of the drug cyclophosphamide, given after a stem cell transplant, can prevent graft-versus-host disease (GVHD) — a common and serious complication where donor cells attack the patient's body. The trial involved 35 adults with various blood dis…

This study tested whether the antibiotic levofloxacin can prevent fevers and infections in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) who are receiving the chemotherapy drug azacitidine. Infections are a major risk for these patients. The trial ran…

This study tested the drug dasatinib in 68 adults with several types of blood and bone marrow disorders, including certain leukemias, myelofibrosis, and mastocytosis. The goal was to see if dasatinib could help control the disease and to check its safety. Participants took dasati…

This study tested giving a second round of radiation to people whose glioblastoma (a fast-growing brain tumor) came back after standard treatment. Seventeen adults aged 18 to 50 received daily radiation for 2-3 weeks. The goal was to find the safest highest dose and see how it af…

This study tested a new drug called VIC-1911 added to standard medications to prevent graft-versus-host disease (GVHD) in 16 adults with blood cancers receiving a stem cell transplant. The goal was to find a safe and effective dose that reduces GVHD and relapse. Results are still…

This study tested whether adding ready-made natural killer (NK) cells from a donor to a standard stem cell transplant could safely reduce the chance of cancer returning in people with high-risk myeloid cancers like acute myeloid leukemia, myelodysplastic syndrome, and chronic mye…

This early-stage trial tested a new oral drug, JNJ-64619178, in 114 people with advanced cancers (including solid tumors, lymphoma, and myelodysplastic syndromes) that had stopped responding to standard treatments. The main goals were to find a safe dose and check for side effect…

This study tested whether adding a transanal (through the anus) approach to standard laparoscopic pelvic pouch surgery could improve outcomes for patients with ulcerative colitis or familial adenomatous polyposis. The transanal method may give surgeons better control and visibili…

This study tested whether adding the drug abatacept to standard medications (calcineurin inhibitor and methotrexate) could better prevent severe acute graft-versus-host disease (aGVHD) after a stem cell transplant from an unrelated donor. A total of 186 participants aged 6 and ol…

This phase II trial tested two drugs—sirolimus and azacitidine—in 57 people with high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that had come back or didn't respond to standard treatment. The goal was to see if the combination could shrink or control the…

This early-phase study tested a new drug called tinostamustine in 10 people with a specific type of glioblastoma, a severe brain cancer. The drug was given after standard treatment to see if it is safe and to find the right dose. The goal is to control the disease and delay its r…

This study tested a stem cell transplant from half-matched (haploidentical) family donors for 34 people with various blood cancers. Patients received a reduced-intensity conditioning regimen of fludarabine, melphalan, and total body irradiation before the transplant. The goal was…

This study tested a new combination of chemotherapy drugs (nab-paclitaxel, cisplatin, and fluorouracil) given before standard chemoradiation in 60 people with advanced nasopharyngeal cancer. The goal was to see if this approach could shrink tumors and improve survival. The trial …

This study tested the long-term safety of a drug called LP352 (bexicaserin) in 41 people aged 12 to 65 with severe epilepsy syndromes like Dravet or Lennox-Gastaut. Participants took the drug three times daily for up to 52 weeks. The main goal was to check for side effects, while…

This study tested adding a drug called thiotepa to the usual chemotherapy given before a donor stem cell transplant for people with leukemia. The goal was to see if this stronger mix could help more patients stay cancer-free one year after the transplant. The study included 40 ad…

This early-phase trial tested a combination of two drugs—veliparib and temozolomide—in 66 adults with relapsed or refractory acute leukemias. Veliparib blocks a DNA repair enzyme, while temozolomide damages cancer cell DNA. The goal was to find the safest dose and check for side …

This early-phase trial tested replacing the standard drug cyclophosphamide with bendamustine after a half-matched bone marrow transplant in 50 patients with various blood cancers. The goal was to see if this change could reduce side effects like graft-versus-host disease while st…

This study tested whether the immunotherapy drug nivolumab, alone or with other drugs, can shrink tumors in people with colon cancer that has returned or spread. It included 385 participants whose tumors had a specific genetic feature called MSI-H or were non-MSI-H. The goal was …

This study tested a drug called guadecitabine combined with donor lymphocyte infusions in 55 patients whose acute myeloid leukemia or myelodysplastic syndrome came back after a stem cell transplant. The goal was to see if the drug could help the donor immune cells fight the cance…

This study tested whether a daily oral pill can safely and temporarily lower sperm counts in healthy men to prevent pregnancy. Ninety-two men took either the experimental drug (DMAU) alone, DMAU plus another hormone (LNG), or a placebo for 12 weeks. The goal was to see if sperm c…

This study tested a two-step stem cell transplant for people with high-risk blood cancers like leukemia and lymphoma. First, patients got low-dose chemotherapy and radiation to prepare the body. Then, they received donor stem cells and immune cells to help destroy remaining cance…

This study tested whether adding pevonedistat to the standard drug azacitidine helps people with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, or low-blast acute myeloid leukemia live longer without their disease getting worse. About 454 adults took part…

This phase 1 study tested the drug enasidenib as a maintenance therapy after a stem cell transplant in 23 people with IDH2-mutant acute myeloid leukemia or chronic myelomonocytic leukemia. The goal was to find the safest dose and see how well patients tolerated it. Enasidenib wor…

This study tested an experimental drug called SPI-62 in 26 adults with Cushing's syndrome caused by a non-adrenal tumor. The goal was to see if SPI-62 could lower cortisol levels safely. Participants received either SPI-62 or a placebo for 24 weeks, with an option to continue lon…

This study tested a drug called MEK162 in 105 children with low-grade gliomas or other tumors that have a specific overactive growth signal. The goal was to find the best dose, check for side effects, and see if the drug could shrink tumors. Children took the drug by mouth twice …

This study tested two daily doses (150mg vs 300mg) of the drug vandetanib in 81 adults with advanced medullary thyroid cancer that had spread or caused symptoms. The goal was to see which dose better shrinks tumors and how side effects compare. Patients took the drug for up to 24…

This early-stage study tested a new drug called JNJ-74856665 in 153 people with acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or chronic myelomonocytic leukemia (CMML). The main goals were to find safe doses and check for side effects when given alone or with oth…

This study tested whether a combination of two drugs, cyclophosphamide and abatacept, could lower the risk of chronic graft-versus-host disease (GVHD) after a stem cell transplant in people with high-risk blood cancers. GVHD happens when donor cells attack the patient's body. The…

This study tested whether the drug palifermin could make stem cell transplants safer and reduce chronic graft-versus-host disease (GVHD), a serious complication where donor cells attack the patient's body. Thirty-four adults with blood cancers received palifermin before transplan…

This phase 3 trial tested a daily oral drug called vandetanib (ZD6474) against a placebo in 331 people with advanced medullary thyroid cancer that could not be removed by surgery. The main goal was to see if the drug could delay tumor growth. Researchers also measured how many pa…

This Phase 3 trial tested a drug called relacorilant in 137 people with hypercortisolism caused by adrenal adenomas or hyperplasia. The goal was to see if it could improve blood sugar control and lower high blood pressure. Participants received either relacorilant or a placebo fo…

This phase 2 trial tested a drug called VAL-083 combined with standard radiation in 29 people with newly diagnosed glioblastoma, a fast-growing brain cancer. The study focused on patients whose tumors have a specific genetic marker (unmethylated MGMT) that makes them resistant to…

This early-stage trial tested whether adding chlorpromazine, an old antipsychotic drug, to standard treatment (radiation and chemotherapy) is safe for people newly diagnosed with glioblastoma, an aggressive brain cancer. Ten adults took part. The main goal was to find a safe dose…

This study tests a new, less invasive way to measure stomach acid using a small wireless capsule (SmartPill) that patients swallow. It compares the capsule's accuracy to the standard method, which involves a tube placed through the nose into the stomach. The study includes health…

This study tested a radioactive tracer called [68Ga]FAPI-46, used with a PET scan, to see if it can better detect pancreatic cancer cells. 63 adults with early-stage pancreatic cancer received the tracer before surgery. The goal was to see how well the scan matches the actual can…

This study tested whether using artificial intelligence (AI) during colonoscopy helps find more polyps in people with Lynch syndrome, a genetic condition that raises colon cancer risk. One hundred participants each had two colonoscopies back-to-back, with the second using either …

This study tested whether a special type of PET/MRI scan using a radioactive tracer called FET can better map glioblastoma brain tumors after surgery compared to standard MRI. The goal was to see if this could help doctors plan radiation therapy more accurately. Eleven adults who…

This study looked at whether a device called Nevisense, which measures the electrical properties of moles, can help doctors better assess suspicious moles during routine skin checks. Forty adults with many moles (at least 100) and several large moles took part. The goal is to imp…

This study looked at a new test called Synovasure that checks joint fluid for signs of infection after hip or knee replacement. Researchers compared its accuracy to current clinical guidelines using 810 stored samples. The goal was to see if the new test agrees with standard meth…

This study tests whether the drug SENS-401 can prevent hearing loss caused by cisplatin, a common chemotherapy. Adults with cancer who are scheduled to receive cisplatin take SENS-401 before, during, and after treatment. Hearing is measured with a standard test to see if the drug…

This study tested a drug called abatacept, given as a shot under the skin, to prevent a serious complication called graft-versus-host disease (GVHD) in 29 adults with blood cancers. Participants received a stem cell transplant from a half-matched donor. The goal was to see if aba…

This pilot study tested a virtual program designed to help older men at high risk for fractures reduce their risk of falls and broken bones. The program included personalized exercise plans delivered through a smartphone app and virtual nutrition counseling. Researchers enrolled …

This study tested two 8-week stress management programs delivered via secure video calls for 196 adolescents aged 12–17 with neurofibromatosis (NF1 or NF2). The goal was to see if these programs could improve emotional, social, and physical quality of life. Both programs taught c…

This study looked at whether adding Epidiolex (a CBD-based medicine) to usual treatment can improve seizures, behavior, sleep, and quality of life in people aged 1 to 65 with tuberous sclerosis complex (TSC). 79 participants took the drug and were monitored for changes in symptom…

This study tested whether N-acetylcysteine (NAC), an over-the-counter antioxidant, could improve learning and motor skills in children with neurofibromatosis type 1 (NF1). Five children aged 8 to 16 took NAC or a placebo to see if it helped with movement and attention problems. T…

This completed phase 3 trial tested whether a patient outreach program—regular phone calls every two weeks—could help people with advanced medullary thyroid cancer spend less time experiencing moderate-to-severe side effects from the drug vandetanib. The study enrolled 205 adults…

This study looked at behavior problems in 101 children aged 3 to 6 with tuberous sclerosis complex (TSC). Families completed assessments online, and those with elevated behavior issues were offered a parent-training therapy (PCIT) over the internet. The goal was to help parents m…

This study looked at whether providing free blood transfusions to blood cancer patients would encourage them to enroll in hospice care earlier. Many patients delay hospice because they need transfusions for symptom relief, but hospices often cannot afford to provide them. The stu…

This study tested two stress and symptom management programs for adults with neurofibromatosis (NF1, NF2, or schwannomatosis). The programs were delivered through live videoconferencing over 8 weekly sessions. Researchers measured changes in quality of life and emotional well-bei…

This study tested whether cooling the skin before or during laser and injection treatments for neurofibromatosis type 1 (NF1) skin bumps can reduce pain. Thirteen adults with NF1 received four different treatments (two lasers and two injections) on both cooled and non-cooled skin…

This study followed 17 adults with neurofibromatosis type 1 (NF1) over two years to learn how their skin tumors (dermal neurofibromas) grow and change. Researchers used special cameras and skin biopsies to measure tumor growth and look for genes that might influence it. The goal …

This study looked at nearly 1,400 people with rare conditions like PPNAD, Carney Complex, and Peutz-Jeghers syndrome. The goal was to find the genetic causes and link them to specific symptoms. Researchers used clinical exams and genetic testing to better understand how these dis…

This completed study looked at how to best find and manage tumors in people with Zollinger-Ellison syndrome, a rare condition that causes too much stomach acid. Researchers used imaging tests and tissue samples to locate tumors and check for a related genetic condition called MEN…

This study looked at people with neurofibromatosis type 1 (NF1) to better understand how harmless tumors (plexiform neurofibromas) turn into a rare cancer called MPNST. Ten participants had MRI, PET scans, and tumor biopsies. Researchers tested whether a special PET scan (FLT PET…

This study looks at people with neurofibromatosis type 1 (NF1) and their families to find out why some have more severe symptoms than others. Researchers will collect medical history, blood samples, and images to study genes and physical traits. The goal is to identify genes that…

This study tested a web-based tool called MeTree that collects family health history to estimate a person's risk for inherited cancer. Over 1,800 adults from three medical centers participated. The goal was to see if the tool helps identify high-risk patients more efficiently tha…

This completed study looked at red and white blood cells in people with a rare blood disorder called PNH who also have bone marrow failure. Over 5,500 participants aged 10 and older with aplastic anemia or related conditions provided blood samples. The goal was simply to learn mo…

This study looks at the DNA of 100 children (and young adults up to age 17) who have cancer, along with the DNA of their healthy parents. The goal is to find genetic changes that may have caused the cancer. By comparing the child's DNA to both parents', researchers hope to discov…

This study tested three types of messages to encourage genetic testing in 1,283 people at risk for hereditary breast and ovarian cancer. Participants received an electronic health record message, then a text message, then a doctor's reminder if needed. The goal was to see which a…

This completed study from France examined whether a specific type of immune cell (innate CD8+ T cells) is linked to how quickly resistance mutations develop in patients with chronic myeloid leukemia or Philadelphia-positive acute lymphoblastic leukemia. Researchers analyzed blood…

This study compared two methods for measuring bioavailable testosterone in the blood: the standard lab test (radioimmunoassay) and a mathematical calculation. Researchers included 270 men and women aged 18 to 90. The goal was to see if the calculation matches the lab test closely…

This study surveyed 231 adults with Lynch syndrome, an inherited condition that raises bowel cancer risk. Researchers wanted to find out how many people follow the UK recommendation of a colonoscopy every two years, and what makes it harder or easier to keep up with these check-u…

This study looked at 545 people with hereditary cancer syndromes and their relatives. It compared the usual method of asking patients to share testing information with family members to a new method where a registry also sends reminders. The goal was to see if the registry-aided …

This study looked at whether a 3-session peer coaching program over the phone could help young women (ages 21–30) who have a family history of BRCA mutations. The goal was to see if it reduces cancer-related distress and helps them make informed decisions about genetic counseling…

This completed study enrolled 377 individuals and families with a high risk of breast or ovarian cancer due to known or suspected genetic factors. Researchers collected medical records, questionnaires, and biological samples to better understand the disease's natural history and …

This study collected information from 733 people with a personal or family history of hereditary stomach cancer. The goal was to better understand how these cancers develop and what genes are involved. Participants provided medical history, blood samples, and genetic testing. The…

This completed study looked at how immune cells recover in 61 adults who received a stem cell transplant for blood cancers. Researchers measured the function of T and NK cells by testing their cytokine profiles after stimulation. The goal was to see if these measurements could he…

This completed study in Japan looked at how low-risk myelodysplastic syndrome (MDS) or unexplained anemia affects patients' daily lives. Researchers used questionnaires and interviews with 56 participants to understand symptoms, quality of life, and unmet needs. The goal was to c…

This study tested whether digital care plans and text message reminders help families of children with cancer predisposition syndromes better understand their child's condition. Researchers enrolled 177 parents and measured changes in knowledge and how acceptable the digital tool…

This study looked at how kidney function and race/ethnicity affect the drug treosulfan in 20 patients with AML or MDS getting a stem cell transplant. The goal was to find safe doses for those with kidney problems. It was a small, early-phase study that did not test a new treatmen…

This study looked at how often people with certain blood cancers or related conditions have an acquired deficiency of an enzyme called pyruvate kinase, which can cause anemia. Researchers took a single blood sample from 18 participants to measure enzyme activity and check for rel…

This study tested a digital health platform to help people with a hereditary risk of breast and ovarian cancer share their genetic test results with family members. Researchers worked with 128 participants in Switzerland and Korea to see if the tool reduced distress and improved …

This study looked at whether a special walking test (3D gait analysis) can help identify movement problems in people with rare genetic diseases like Tuberous Sclerosis and STXBP1. About 40 participants aged 6 and older who could walk without help took part. The goal was to see if…

This study looked at 308 people with myelodysplastic syndromes (MDS) who had a stem cell transplant. Researchers identified each patient's unique genetic mutations and then monitored those mutations in blood and bone marrow samples after transplant. The goal was to see if rising …

This study collected blood, bone marrow, skin, and cheek cell samples from 650 adults with acute leukemia, myelodysplastic syndromes, or myeloproliferative disorders. Researchers analyzed the samples to find genetic, epigenetic, and drug sensitivity patterns that might predict ho…

This study tested whether a 'Disclosure Toolkit'—including a family letter, a chatbot, and a website—helps people with BRCA1 or BRCA2 mutations share their genetic test results with at-risk relatives. Researchers enrolled 52 participants to see if the toolkit improves communicati…

This completed study brought together men who have had suicidal thoughts to help design suicide prevention tools. Over up to 10 meetings, 13 men gave feedback on tool drafts, focusing on usability, privacy, and support. The goal was to create resources that truly fit their needs.

This study explored how teenagers and their parents react emotionally and mentally when they learn about genetic risks for conditions like hereditary breast cancer, Lynch syndrome, or high cholesterol. Researchers surveyed 162 participants using questionnaires and interviews to m…

This completed study tested whether an online self-guided program could effectively return genetic test results for hereditary breast and ovarian cancer to African American women. Over 900 women from the Black Women's Health Study were offered their results online or by printed m…

This study tested whether a telehealth program could help older patients (60+) with acute myeloid leukemia or myelodysplastic syndrome discuss and document their future medical wishes. Researchers enrolled 36 patients, caregivers, and clinicians to see if the program was easy to …

This study tested a new type of MRI scan that shows how brain tumors use energy, which could help doctors make better treatment decisions. Seventeen adults with glioblastoma received an injection of a special substance before imaging. The goal was to see if this method is safe an…

This study looked for new genes that cause a rare condition called syndromic congenital neutropenia, where children are born with low infection-fighting white blood cells and other developmental issues. Researchers used advanced gene sequencing on 25 participants to find the gene…

This study looked at how a genetic test that checks 25 genes linked to hereditary cancers is used in clinics. Over 1,500 people with a personal or family history of cancer took part. Researchers collected blood samples and had participants fill out questionnaires over five years …

This study looked back at medical records of 418 patients with lower-risk myelodysplastic syndromes (MDS) who had not previously used erythropoiesis-stimulating agents (ESAs). Researchers examined how luspatercept was used in everyday practice, including dosing, treatment changes…

This completed study used Danish national health records to compare infection rates after total knee replacement in obese (BMI ≥30) versus non-obese patients. Researchers analyzed data from over 100,000 adults who had knee replacement for osteoarthritis between 2011 and 2021. The…

This study tested whether a short video sent by text message could help people with BRCA gene mutations encourage their relatives to get tested. Fifty-eight participants completed the trial. The goal was to see if the video made it easier to share important health information wit…

This study measured how often iron builds up in the heart muscle of people who receive many blood transfusions. Researchers used MRI scans to check heart iron levels in 110 patients with thalassemia, sickle cell disease, or myelodysplasia. The goal was to understand how common th…

This completed study enrolled over 2,100 people with or suspected of having myelodysplastic syndromes (MDS), a group of blood disorders. Researchers collected medical data, blood, and tissue samples over time to track how the disease develops and progresses. The goal was to creat…

This study compared robot-assisted laparoscopy using the Senhance Surgical System to conventional laparoscopy for hysterectomy and other uterine surgeries. Researchers measured operating time, blood loss, and safety in 78 patients. The goal was to see if the robot offers advantag…

This study tested whether an educational session at a church could help Latino adults learn more about colorectal cancer and feel more willing to join cancer clinical trials. Sixty Spanish-speaking participants watched videos and completed surveys. The goal was to understand barr…

This small, completed study tested a device called NuView in 3 people with familial adenomatous polyposis (FAP). The goal was to see if the device helps doctors view the papilla, a small opening in the intestine, using a standard forward-facing endoscope. Participants had their u…

This completed study looked at 1,200 people with neurocutaneous syndromes (NF1, TSC, SWS, VHL) in Western China. Researchers compared those who received genetic testing, targeted drugs, and coordinated specialist care against those who got standard care. The goal was to see if pr…

This study looked at 1,753 patients needing a stem cell transplant for blood cancers or other serious blood disorders. Researchers compared two strategies: first searching for a matched unrelated donor, then trying an alternative donor if needed, versus going straight to an alter…

This study looked at the bacteria in the mouth and gut of 126 people with and without serrated polyposis syndrome, a condition that raises the risk of colorectal cancer. Researchers compared the bacteria in people with the syndrome to those with other types of polyps or no polyps…

This early-stage study tested the safety and tolerability of a new drug called LP-001 in 68 healthy adults aged 18 to 50. The goal was to see how the body handles single and multiple doses, not to treat any disease. Results will help guide future research for myelodysplastic synd…

This study tested whether a mindful self-compassion program could be adapted for young adults aged 18 to 29 with Li-Fraumeni syndrome, a genetic condition that greatly raises cancer risk. Seven participants attended a one-day demonstration and gave feedback on how to make the pro…

This completed study followed 183 cancer patients starting checkpoint inhibitor therapy to see how often they develop autoantibodies—proteins that can mistakenly attack the body's own tissues. Researchers collected blood samples to track changes in several types of autoantibodies…

This study looked at how 177 people with lower-risk myelodysplastic syndromes (MDS) are treated in real-world clinics across Japan. Researchers collected data on treatments, blood transfusions, and healthcare costs to understand current practice patterns. The goal was to describe…

This completed study at Stanford University looked at the DNA of over 1,000 adults with Neurofibromatosis Type 1 (NF1) to find genetic differences that might explain why some people develop more skin neurofibromas than others. Participants provided blood or saliva samples for gen…

This study looked at whether briefly pressing on the phrenic nerve during lung surgery could help reduce complications like air leaks. Researchers compared 55 patients who had this temporary nerve compression with those who did not. They measured diaphragm activity, lung function…

This study looked at tiny, lab-grown versions of the intestine called organoids, made from tissue samples of people with inflammatory bowel disease (Crohn's or ulcerative colitis), a genetic polyp condition (FAP), and healthy volunteers. The goal was to understand how intestinal …

This study tested how the experimental drug radiprodil interacts with five common medications (warfarin, midazolam, digoxin, rosuvastatin, and omeprazole) in 18 healthy adults. Participants took radiprodil alone and then with each of these drugs to measure changes in drug levels …

This study looked at 126 people aged 60 and older who had a bone marrow transplant for blood cancers like leukemia or lymphoma. Researchers measured physical abilities, mental health, and stress levels before and after transplant to find traits that help people recover better. Th…

This study tested over 1,000 women with endometrial cancer for DNA repair problems that can be inherited (Lynch syndrome). By analyzing tumor DNA, researchers aimed to personalize cancer treatment and identify family members who might also be at risk. The goal was to improve care…

This completed Phase 2 trial tested whether a special PET/CT scan using 11C-methionine could help doctors better plan radiation therapy for people with glioblastoma, the most common and aggressive brain cancer. The study focused on patients whose tumors grew back quickly after su…

This study looked at whether offering exome sequencing (a detailed genetic test) along with tailored genetic counseling helps people at high risk for hereditary cancer syndromes understand their risk and take action. Nearly 1,000 adults from diverse backgrounds in Colorado and Or…

This completed study looked at how the pigment layer of the eye works in 30 people with neurofibromatosis type 1. Researchers used special eye tests to measure electrical signals from the pigment layer and compared them to images of the back of the eye. The goal was to confirm ea…

This study enrolled 349 people with myelodysplastic syndromes (MDS) to see how certain gene mutations affect survival. Researchers collected blood samples at diagnosis and tracked patients over time. The goal was to understand which mutations are linked to better or worse outcome…

This study looked at whether mailing at-home genetic testing kits to relatives of people with hereditary cancer gene variants could increase the number of relatives who get tested. Researchers compared this approach to usual care in 108 participants. The goal was to see if making…

This completed study involved 146 patients with blood cancers who received a stem cell transplant from a donor. Researchers collected blood and tissue samples over a year to study immune cells linked to graft-versus-host disease (GVHD), a common complication where donor cells att…