Disease by molecular mechanism

This study tests an experimental drug called GSK4527226 in 367 people with early Alzheimer's (mild memory problems or mild dementia). Participants receive either a low dose, high dose, or placebo through an IV. The goal is to see if the drug can slow down declines in memory and d…

Researchers are testing whether piromelatine 20 mg, a tablet that acts on melatonin and serotonin receptors, can slow cognitive decline in people aged 60 to 85 with mild dementia from Alzheimer's disease. The study enrolls 216 participants who do not carry a specific genetic vari…

This pilot study tests whether a special PET scan tracer called 18F florbetapir can detect changes in amyloid protein deposits in the heart of people with AL cardiac amyloidosis who are receiving chemotherapy. Four participants will have a PET/CT scan before starting chemotherapy…

This trial tests whether a light-activated cream (Levulan Kerastick) followed by red light therapy can slow the growth of benign skin tumors called neurofibromas in people with neurofibromatosis type 1 (NF1). About 30 participants aged 14 and older with superficial tumors on the …

This early-stage trial tested an oral drug called TNO155, alone or with another drug (nazartinib), in 227 adults with advanced solid tumors like lung cancer, melanoma, and head/neck cancer. The main goal was to check safety and find the right dose. The study was terminated early,…

This study observes children aged 3 to 18 with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas and are starting treatment with selumetinib. Researchers will track how well the drug shrinks or stabilizes tumors, how long patients stay on tre…

This study tested the long-term safety of AXS-05, a combination of dextromethorphan and bupropion, for treating agitation in people with Alzheimer's disease. It enrolled 139 participants who had completed earlier studies. The trial was terminated early, so the full safety picture…

This study looked at the long-term safety and effects of ALZ-801, a tablet taken twice daily, in 163 people with early Alzheimer's disease who have the APOE4/4 gene. Participants had already completed a previous phase 3 study. The goal was to see if the drug could slow cognitive …

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-stage study tested a new drug called SAR445514, which helps the body's natural killer (NK) cells attack cancer cells in people with multiple myeloma or light-chain amyloidosis that had returned or stopped responding to other treatments. The study was terminated early, …

This study tested different warming methods—like forced air blankets and warmed IV fluids—to prevent hypothermia in women having planned C-sections. Only 16 women participated before the trial was stopped early. The goal was to see which method best keeps mother and baby warm and…

This study tested an experimental drug called SHR-1707 in people with mild memory problems or mild Alzheimer's disease. The goal was to see if it could safely reduce harmful protein clumps in the brain. The trial was stopped early, and it is not expected to cure the disease.

This study looked at the long-term safety of an experimental drug called TB006 (oloctinebart) in people with Alzheimer's disease. It included 119 participants who had either completed a previous TB006 study or were new to the drug. The study was terminated early, so the full safe…

This early-stage study tested a new drug, ADEL-Y01, in healthy volunteers and people with mild Alzheimer's or mild cognitive impairment. The goal was to check safety and how the drug moves through the body. The trial was stopped early, so results are limited.

This study tested a drug called PrimeC in people with mild to moderate Alzheimer's disease. The goal was to see if it is safe and if it helps with thinking and daily activities. The study was stopped early and only 8 people took part, so results are very limited.

This study tested an experimental antibody called JNJ-63733657 in 523 people with early Alzheimer's disease. The antibody aims to clear tau protein clumps in the brain, which are linked to cognitive decline. Participants received either the antibody or a placebo by IV infusion. T…

This early study tested a weekly injection of allopregnanolone, a natural brain steroid, in 6 people with early Alzheimer's or mild cognitive impairment. The goal was to find a safe dose and check for side effects. The trial was terminated early, so we have limited information on…

This study gave Alzheimer's patients who had already completed earlier XPro1595 trials an extra 55 to 74 weeks of the drug to check long-term safety and effects on memory and brain health. Only 11 people took part, and the trial was terminated early, so the findings are limited. …

This study was a long-term extension of an earlier trial, testing the safety and tolerability of AL002 in 197 people with early Alzheimer's disease. Participants received the drug by IV infusion. The trial was terminated, meaning it stopped before completion, so the full safety a…

This study tested a drug called inotersen in 31 people with a rare condition where a faulty protein builds up in the heart, causing damage. The drug is designed to lower levels of that protein. The trial was stopped early, so it's unclear if it works, but researchers hoped to see…

This early-stage study tested a drug called ION269 in adults with Down syndrome who also showed signs of Alzheimer's disease. The goal was to check if the drug was safe and tolerable when injected into the spinal fluid. Only one person took part, and the study was stopped early, …

This study aimed to check the long-term safety and effects of a daily pill called SPG302 in people with mild-to-moderate Alzheimer's disease. It included 12 participants who had already been in a previous SPG302 study. The study was terminated early, so results are limited.

This phase 3 study tested an experimental drug called birtamimab in 208 people with advanced AL amyloidosis, a rare disease where abnormal proteins damage organs like the heart. Participants received either birtamimab or a placebo, plus standard chemotherapy. The goal was to see …

This study tested a drug called varoglutamstat (PQ912) in people with early Alzheimer's disease. The goal was to see if it is safe and if it could slow memory and thinking problems. The trial was stopped early, so we have less information than planned. About 112 people took part,…

This study tested an experimental drug called REM0046127 in people with mild to moderate Alzheimer's disease. The goal was to see if it was safe and how it affected brain chemicals and brain wave activity. The trial was stopped early and included only 14 participants instead of t…

This study tested an experimental drug called IMG-007 in 13 adults with moderate-to-severe atopic dermatitis (eczema) who did not get enough relief from topical treatments. The main goal was to check the drug's safety, and researchers also looked at how the body processes it and …

This study tested a drug called AVP-786 to see if it could safely reduce agitation in people with Alzheimer's dementia over the long term. It was an extension of earlier studies, enrolling over 1,100 participants who had already completed a prior trial. However, the study was ter…

This study tested a drug called simufilam in over 1,000 people with mild-to-moderate Alzheimer's disease. Participants took either 50 mg, 100 mg, or a placebo twice daily for 76 weeks. The goal was to see if simufilam could slow memory loss and help with daily activities. However…

This study tested a drug called ixazomib plus dexamethasone against other standard treatments for people with relapsed AL amyloidosis, a rare disease where abnormal proteins build up in organs. The trial aimed to see if the combination improved blood markers and slowed heart or k…

This study explored whether a special eye scan (hyperspectral retinal imaging) can detect beta-amyloid, a protein linked to Alzheimer's disease, in the retina. Researchers aimed to see if this non-invasive test could distinguish people with Alzheimer's from healthy individuals. T…

This study tested a wearable device that sits on the neck to detect swallowing problems in people with conditions like stroke, Parkinson's, multiple sclerosis, Alzheimer's, or dementia. The device was compared to a standard X-ray test. Unfortunately, the device did not meet the a…

This study tested whether a wearable EEG device can help doctors diagnose Alzheimer's disease and mild cognitive impairment. Researchers compared brain wave data from the EEG with standard memory and thinking tests in 148 participants. The goal was to see if the EEG could improve…

This study looks at whether a ketogenic diet (high-fat, low-carb) is practical and safe for people with early-stage Alzheimer's disease over one year. The diet may provide an alternative energy source for the brain and reduce inflammation. Researchers will track diet adherence, b…

This study tested a fast-dissolving film (BXCL501) placed under the tongue to quickly reduce severe agitation in older adults with dementia. The trial aimed to enroll many participants but was stopped early after only 5 people joined. Because it ended so soon, we cannot draw reli…

This study tests whether adding MK-1167 to standard Alzheimer's treatment can improve memory and thinking in people with mild to moderate Alzheimer's dementia. About 350 participants will receive either MK-1167 or a placebo for 24 weeks. Researchers will measure changes in cognit…

This study looked at whether a drug called K0706 is safe and tolerable for people with Dementia with Lewy Bodies (DLB). Researchers planned to give 45 participants either a low dose, high dose, or placebo for 12 weeks, then check for side effects and measure changes in brain and …

This study tested a drug called AVP-786 to see if it could safely reduce disinhibition—impulsive or inappropriate behavior—in people with neurodegenerative disorders like Alzheimer's or frontotemporal dementia. The trial planned to include many participants but was terminated ear…

This study tested a personalized touchscreen device (My PATI) to help people with Alzheimer's or related dementias communicate their care preferences. The goal was to improve quality of life for both patients and their caregivers. The study was terminated early, so results are li…

This study tested a drug called CVL-871 to see if it is safe and can help reduce apathy (lack of motivation or interest) in people with dementia. The trial included 41 participants with mild to moderate dementia and clinically significant apathy. The main goal was to check for si…

This study tested whether a gentle, non-invasive brain stimulation technique (tDCS) done at home, along with computer-based brain games, could improve thinking and language skills in people with primary progressive aphasia, mild cognitive impairment, or dementia. Participants use…

This study tested a behavioral program using bright light therapy and sleep coaching to improve sleep and thinking in older adults with mild cognitive impairment or mild Alzheimer's. Only 15 people were enrolled before the study was stopped early. The goal was to see if the progr…

This pilot study tested a special helmet and abdominal panel that shines near-infrared light and uses a static magnetic field on people with mild-to-moderate Alzheimer's disease. The goal was to see if it could safely improve memory and thinking. The study planned to enroll 60 pa…

This study tested a digital health app called medidux™ in 196 people with HER2-positive breast cancer. The app was used for up to 16 weeks during chemotherapy to help track and manage side effects. The goal was to see if using the app reduced the number of severe side effects. Ho…

This study investigates how Alzheimer's disease and frontotemporal dementia alter a person's sense of self and ability to understand others. Researchers will compare patients with healthy older adults using questionnaires and brain imaging. The goal is to link changes in self-awa…

This early-stage study aimed to compare how a tablet form of tafamidis is absorbed in the body compared to the existing capsule form. It involved 24 healthy adults who took a single dose of each form under fed conditions. The study was terminated early, so results may be limited.

This study looks at the tiny hair-like structures (cilia) inside the fallopian tubes of women who have had surgery to remove a tube or uterus. Researchers want to see if differences in these cilia help explain why some women have ectopic pregnancies (when a fertilized egg grows o…

This study collects small skin samples from children aged 2 to 17 with Costello syndrome or a related condition. Researchers will analyze the cells to understand how a mutation in the HRAS gene affects energy use and mitochondria. The goal is to learn more about the disease's und…

This study tested whether a visual consent form with pictures helps patients with cancer or Alzheimer's understand research better than standard text. Researchers planned to enroll 266 adults across three U.S. sites. The trial was terminated early, so results may be limited.

This pilot study measured brain activity (EEG) in 6 healthy volunteers while they performed computer tasks that test attention and memory. The goal was to develop better ways to measure thinking skills that could be used in future Alzheimer's drug trials. The study was terminated…

This study looked at why people with type 1 diabetes often fail to sense dangerously low blood sugar (hypoglycemia). Researchers tested how opioid drugs like morphine and naloxone affect the body's ability to respond to low sugar. The goal was to better understand the problem, no…

This study looked at how early Alzheimer's disease affects a person's ability to make decisions. Researchers tested 100 people with Alzheimer's and healthy volunteers using memory and thinking tasks, plus a special decision-making test. The goal was to understand which brain func…

This early-stage study aimed to see if taking memantine, an Alzheimer's drug, for 15 days would change certain brain and cognitive markers in healthy young men aged 18-30. The goal was to validate a test battery for future Alzheimer's drug development. However, the study was term…

This study explored whether a non-invasive technique called transcranial magnetic stimulation (TMS) could improve memory in healthy older adults and those with mild memory problems due to Alzheimer's. Researchers used TMS to stimulate brain networks linked to memory and measured …

This study tested whether a robot that moves on its own could patrol nursing home rooms and detect when a resident with Alzheimer's has fallen. The robot was designed to turn on lights, alert staff, and let them see and talk to the resident through its camera and screen. The stud…

This study tested whether anti-inflammatory drugs could help people with early Alzheimer's or mild cognitive impairment. It was stopped early, so only one drug was studied in 34 participants. The main goal was to measure changes in thinking and memory, but the early termination l…

This study aims to find out how common transthyretin amyloidosis cardiomyopathy (ATTR-CM) is in Russian patients with a certain type of heart failure. Researchers will review medical records and then invite some patients for extra heart tests to confirm or rule out ATTR-CM. The g…