Disease susceptibility

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 looked at whether using antibiotic ear drops during and after ear tube surgery reduces the chance of tube blockage or ear drainage in children. Only 9 children took part, and each child received drops in one ear but not the other. The study was stopped early, so the fi…

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 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 study tested an experimental drug called TOL-3021 in people with type 1 diabetes to see if it could help preserve the body's ability to produce insulin. The trial enrolled 78 participants aged 12 to 41 who had been diagnosed within the last 5 years. The study was terminated …

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 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 early-stage study tested a new treatment called VCTX211 for people with type 1 diabetes. The goal was to see if implanting these special cells could help the body make insulin and reduce the need for insulin shots. Only 5 people took part, and the study was stopped early. Th…

This study tested a low dose of a drug called ATG to see if it could delay or prevent type 1 diabetes in people at high risk. Only 2 people enrolled, so the study was stopped early. The goal was to measure how long it took for participants to develop full-blown diabetes.

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 study tested a drug called OMS721 in 31 people with certain kidney diseases, including IgA nephropathy and lupus nephritis. The main goal was to check if the drug is safe and if it reduces protein in the urine, a sign of kidney damage. The study was stopped early, so results…

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 aimed to see if Hiprex (methenamine hippurate) could reduce recurrent urinary tract infections in women aged 50 to 85. Participants took Hiprex daily for a year, and researchers counted infections and antibiotic use. However, the trial was terminated early with only 14…

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 daily probiotic supplement containing Lactobacillus plantarum, cranberry, and vitamin C to see if it could prevent recurrent urinary tract infections (UTIs) in women. The trial planned to enroll 106 women with a history of frequent UTIs, but it was terminated …

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 a new drug called CX-072 combined with other cancer treatments for adults with solid tumors or advanced melanoma. The goal was to see if the combination could shrink tumors. However, the study was stopped early and only 3 people took part, so we don't have enoug…

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 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 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 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 study tested a drug called ALXN2050 in people with kidney inflammation caused by lupus (lupus nephritis) or IgA nephropathy. The goal was to see if it could reduce protein in the urine, a sign of kidney damage. About 100 adults were planned to take the drug or a placebo alon…

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 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 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 study tested whether gloves with tiny semiconductors embedded in the fabric could reduce pain and improve hand function in people with hand osteoarthritis. Sixteen participants wore either the active gloves or identical-looking placebo gloves. The trial was terminated early,…

This study tested a new way to inject the drug ZILRETTA into the hip joint for people with hip osteoarthritis. The goal was to see if using different needle sizes could improve the success rate of the injection. Only one person took part before the study was stopped early.

This study tested whether low level laser therapy (LLLT) could reduce pain and improve hand function in people with hand osteoarthritis. About 83 military members and their families received either real or sham laser treatment weekly for six weeks. The trial was terminated early,…

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 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 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 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…

This study looked at adults with severe uncontrolled asthma in Germany to see how well their asthma was controlled over 12 months. Researchers used a simple questionnaire called AIRQ® to track symptoms, medication use, and doctor visits. The goal was to gather real-world informat…

This study tested whether two medications, naloxone (a nasal spray) and diazoxide (an oral pill), could prevent the body from losing its ability to sense low blood sugar—a condition called hypoglycemia unawareness. Only 4 healthy volunteers were enrolled before the trial was stop…