Neurodegenerative disease

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 study tests whether an additional dose of a COVID-19 vaccine can improve immune protection in adults and children with autoimmune diseases like rheumatoid arthritis, lupus, multiple sclerosis, and others who are taking immunosuppressive medications. Participants who had a we…

This study tested whether injections of MYOBLOC (botulinum toxin type B) can safely reduce muscle tightness in the arm and hand of adults with spasticity caused by stroke, brain injury, multiple sclerosis, or other conditions. The trial planned to enroll about 300 participants bu…

This study tested an oral form of edaravone in 384 adults with ALS to see if it could slow the disease's progression over 48 weeks. Participants were randomly assigned to different dosing schedules. The trial was terminated early, and results are not yet fully available.

This study tested two doses of oral edaravone in 202 people with ALS to see if it could slow disease progression. Participants were followed for up to 48 weeks. The trial was terminated early, so the full results are not available.

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 phase 3 trial tested lithium carbonate against placebo in 88 people with ALS to see if it could improve survival and daily function. The study was terminated early, so the full results are not available. The trial aimed to find a treatment that slows the disease, but its ear…

This study tested a new drug called exicorilant (CORT125281) combined with enzalutamide in men with metastatic castration-resistant prostate cancer. The goal was to find a safe dose and check for side effects. The trial was terminated early, so results are limited. It involved 39…

This study tested whether clemastine fumarate, an allergy medication, could improve eye movement problems in people with multiple sclerosis (MS). The trial planned to enroll 80 MS patients with a specific eye condition called internuclear ophthalmoparesis, but was terminated earl…

This study offered continued treatment with EryDex to 101 people with ataxia telangiectasia (A-T) who had finished a previous trial. The main goal was to monitor safety, including side effects and serious events. The study was terminated early, and it did not aim to cure the dise…

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 long-acting form of glatiramer acetate (GA Depot) given as a monthly shot to people with primary progressive multiple sclerosis (PPMS). The goal was to see if it could safely slow down worsening disability. The trial was stopped early, but it involved 30 adult…

This phase 2 trial tested telitacicept (RC18) in 8 people with relapsing-remitting multiple sclerosis. The drug aims to reduce relapses by targeting B cells. The study was terminated early, so we have limited data on safety and effectiveness.

This study tested a drug called TPN-101 (censavudine) in people with Aicardi-Goutières syndrome, a rare genetic disorder that causes severe brain inflammation. The trial enrolled only 4 participants and aimed to see if the drug could reduce immune system overactivity and check fo…

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 study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

This was a very early Phase 1 study testing the safety of a drug called bryostatin in just 4 people with multiple sclerosis. Participants received multiple doses over 26 weeks. The study was terminated, meaning it stopped early, so we don't have clear results on whether the drug …

This Phase II trial tested a drug called N-Acetyl-L-Leucine (IB1001) in 17 people with Ataxia-Telangiectasia, a rare genetic disease that affects movement and immunity. The study aimed to see if the drug could improve symptoms and slow the disease over time. The trial was termina…

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 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 tested whether adding melatonin to the standard drug ocrelizumab could safely slow disability in people with progressive primary multiple sclerosis. The trial was terminated early, so results are limited. Participants were adults aged 18–65 with this type of MS.

This early-phase study tested whether a fecal transplant from a healthy donor could be safe and affect the immune system in people with relapsing-remitting multiple sclerosis. Only 5 participants were enrolled before the study was stopped. The goal was to see if changing gut bact…

This study tested a new approach using a virus that attacks cancer cells (oncolytic immunotherapy) along with two standard drugs (atezolizumab and bevacizumab) in people with advanced colorectal cancer that had stopped responding to other treatments. The trial was stopped early a…

This early-stage trial tested a drug called nomlabofusp (CTI-1601) in 18 adolescents and children with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to check safety and how the body processes the drug. However, the study was term…

This Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…

This phase 1 trial tested a gene therapy called AVXS-101 (Zolgensma) in 32 children with spinal muscular atrophy (SMA) who could sit but not stand or walk. The therapy delivers a working SMN gene via a spinal injection to help improve muscle function. The study focused on safety …

This study was designed to let people who had already taken latozinemab in an earlier study continue receiving the drug. Only 17 people took part, and the study was stopped early. The goal was to track safety and how long people stayed on treatment, not to test if the drug could …

This phase 3 study tested a drug called AL001 in 119 people who carry a mutation in the progranulin gene, putting them at high risk for or already diagnosed with frontotemporal dementia. The goal was to see if AL001 could slow the worsening of memory, behavior, and language probl…

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 study tested a single dose of STR04, made from a patient's own bone marrow stem cells, for amyotrophic lateral sclerosis (ALS). Only one person enrolled before the study was stopped early. The goal was to see if the treatment was safe and could slow the disease, but no concl…

This study looked at the long-term safety and effectiveness of a daily oral medication called SPG302 in people with ALS who had already taken part in an earlier study. The trial was stopped early, and only 16 people were enrolled. Researchers tracked side effects, suicidal though…

This study aimed to see if the drug SAGE-718 is safe for people with Huntington's disease when taken over a long period. It was an open-label study, meaning everyone knew they were getting the drug. The study was terminated early, so results are limited.

This study tested a new drug called VRG50635 in 54 people with ALS (Lou Gehrig's disease). The main goal was to check if the drug is safe and how the body processes it. The trial was stopped early, but researchers were looking for side effects and changes in disease progression.

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 a blood product called immune globulin (Flebogamma) in 191 people with post-polio syndrome, a condition causing new muscle weakness years after polio. The goal was to see if it could improve walking distance and reduce pain. The trial was terminated early, so we…

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 the drug alemtuzumab in children aged 10 to 18 with relapsing-remitting multiple sclerosis (MS) whose disease was still active despite other treatments. The goal was to see if the drug could reduce new brain lesions and improve quality of life. However, the tria…

This study tested the drug natalizumab in 21 Japanese adults with relapsing-remitting multiple sclerosis (MS). The goal was to see if injections under the skin every 4 weeks could reduce new brain lesions over 24 weeks, and to check safety for up to 48 weeks. The study was termin…

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 early-stage trial tested a gene therapy called SBT101 for adrenomyeloneuropathy (AMN), a rare nerve disease that causes walking difficulties. Eight adults received either the therapy or a sham procedure. The study was terminated early, so we have limited data on safety and e…

This early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…

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 an oral drug called SAR443820 in 174 adults with multiple sclerosis (MS) to see if it could lower a marker of nerve damage in the blood. The trial was stopped early because the drug did not meet its main goal of reducing that marker. Participants were followed f…

This study tested an experimental drug called BIIB105 in adults with ALS, a progressive nerve disease. The trial had two parts: a 6-month placebo-controlled phase and a 3-year open-label extension where everyone received the drug. The goal was to check safety, how the body proces…

This early-stage trial tested an experimental drug called DNL747 in 15 people with ALS to see if it was safe and how the body processed it. The study was terminated before completion, so we don't have full results. It was a small, short-term safety study, not a test of whether th…

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 phase 3 trial tested the drug miglustat (Zavesca) in 30 infants with Sandhoff or Tay-Sachs diseases, rare genetic disorders that destroy nerve cells. The goal was to see if the drug could reduce hospitalizations, seizures, and feeding problems while improving motor function.…

This early-stage trial tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic brain disorders. The therapy delivers healthy genes directly into the brain and spinal fluid to try to restore a missing enzyme. The study was te…

This study tests whether a tear sample can diagnose primary progressive multiple sclerosis (MS) as accurately as a spinal tap. Currently, doctors need to collect spinal fluid to confirm this type of MS, which is invasive and uncomfortable. Researchers will compare proteins in tea…

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 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 tested whether a digital platform (m-Rehab) could help people with ALS who use non-invasive ventilation. The platform included educational content, remote monitoring, and video consultations. The goal was to see if it could slow the loss of physical function and improv…

This study tests a mobile telehealth app called Brain CareNotes designed to help unpaid Hispanic caregivers manage challenging behaviors in people with Alzheimer's or related dementias. The app offers coaching and tools to handle symptoms like agitation or wandering. Researchers …

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 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 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 whether EEG neurofeedback—a type of brain training that uses real-time brainwave readings—can help people with aphasia (trouble speaking or understanding language) improve their communication, sleep, and anxiety. Seven adults with aphasia from stroke or primary …

This study tested a rehabilitation program for hand sensitivity problems in people with multiple sclerosis. The program used TENS (a mild electrical stimulation device) and hand exercises to improve the ability to feel and recognize objects. The goal was to improve quality of lif…

This study aimed to see if the drug ozanimod could help reduce fatigue in people with multiple sclerosis (MS). Researchers planned to follow 26 adults with MS for three months, measuring fatigue using special questionnaires and brain scans. The study was terminated early, so resu…

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 used a special MRI technique to look at tiny brain changes in people who recently had a first episode of symptoms that could lead to multiple sclerosis (MS). Researchers compared 84 participants (patients and healthy controls) to see if early memory problems are linked…

This study tested whether special insoles with pressure sensors (FEETME®) can accurately measure walking speed in people with CIDP, a nerve condition that causes walking difficulties. Researchers compared the insoles to a standard walkway system in 18 adults aged 18-75 with CIDP.…

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 small, early-terminated study explored whether measuring certain receptors on immune cells could predict disease activity and rebound risk in multiple sclerosis patients taking or stopping ozanimod. Researchers analyzed blood samples from 23 adults with relapsing-remitting M…

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 tested whether two gut hormones, GIP and GLP-2, could reduce bone breakdown in children with spinal muscular atrophy, cerebral palsy, or Duchenne muscular dystrophy who use wheelchairs. Participants received a liquid meal and then either a hormone injection or a placeb…

This study looked at how stress, hormonal cycles, illness, and missed medications might affect when symptoms appear or get worse in women with relapsing-remitting multiple sclerosis (RR-MS). Researchers planned to track 80 women over time, recording daily symptom changes. The stu…

This study was a registry that collected information from over 900 adults being treated for degenerative spine conditions. Researchers focused on the use of bone graft materials and how patients felt after treatment. The study did not test any new treatment and was stopped early.

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 looked at whether special MRI scans can detect early brain changes in people with multiple sclerosis who are starting treatment with Tysabri. Researchers planned to follow 70 patients for two years, comparing MRI images before and after treatment. The goal was to find …

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 looked at how satisfied people with ALS (Lou Gehrig's disease) were when they had doctor visits by video from home, with a nurse or physiotherapist there to help. About 38 patients who used a breathing machine at home took part. The goal was to see if this kind of care…

This study followed 7 people with Mucolipidosis Type IV (MLIV) to learn how the disease naturally progresses. Researchers used tests like neuropsychological exams, blood and urine tests, and brain MRIs to find better ways to measure the disease. The goal was to improve future cli…

This study tested a wearable device called Syde® in 86 people with multiple sclerosis. Participants wore the device for one month every six months over two years. The goal was to see if the device's activity data matches standard clinic tests like walking speed and disability sco…

This study followed 65 adult men with a rare inherited nerve disease called AMN (a form of spastic paraplegia) to understand how their symptoms change over time. Researchers collected data on walking ability and quality of life. The goal was to fill gaps in knowledge about the di…

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 tested an experimental drug called BLZ945 in 28 people with ALS to see if it could reduce brain inflammation. Researchers used special PET scans to measure inflammation. The study was terminated early, so the full results are not available, but it aimed to gather safet…

This study follows up on children with Krabbe disease who received a one-time gene therapy infusion (FBX-101) in earlier trials. Researchers will monitor safety and measure motor skills over time. Only 2 participants are enrolled, so results are very limited.