Early-onset progressive encephalopathy-spastic ataxia-distal spinal muscular atrophy syndrome

This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…

This study will test whether living in a self-contained, AI-managed community (called a Microcity) can improve mental health and reduce addiction in adults with conditions like autism, ADHD, substance use disorders, and behavioral addictions. Up to 1 million participants will liv…

This study tests a short case management program for people who have been involuntarily admitted to a psychiatric hospital. The program, designed with input from people who have experienced coercion, provides up to five sessions with a nurse or social worker starting during the h…

This trial will test an artificial intelligence system designed to interpret routine newborn screening tests for inherited metabolic disorders. Researchers will compare the AI's accuracy to standard manual review by trained staff. The study plans to include 200,000 newborns in Ch…

This study tests an 8-week program called KINDER, designed to help family caregivers of people with dementia reduce psychological elder abuse and improve the quality of care. The program includes group discussions over Zoom and a self-paced workbook. Researchers will enroll 260 c…

This study looks at whether high-intensity walking training can help people with cerebellar damage (a part of the brain that controls balance and coordination) improve their walking ability. Twenty participants will either do intense walking exercises or standard training. The go…

This study tests whether a virtual cognitive behavioral therapy for insomnia (CBT-I) program, called REPAIR Sleep, can improve sleep and emotional well-being in people with dementia and their live-in caregivers. Over 5 weekly video sessions, participants learn techniques like set…

This pilot study will test whether a non-invasive brain stimulation technique called transcranial alternating current stimulation (tACS) can improve working memory in 30 adults with mild cognitive impairment or traumatic brain injury. Participants will receive low-level electrica…

This study tests whether a type of cognitive training called Action-Based Cognitive Remediation (ABCR) can improve thinking skills in people with various psychiatric conditions like depression, schizophrenia, anxiety, PTSD, and autism. Participants will complete computer exercise…

This pilot study will test a digital platform called the Dementia Journey Companion (DJC) that helps people with dementia and their caregivers plan future care. Researchers will enroll 150 participants with mild to moderate dementia living at home or in care facilities. The study…

This study tests whether laser acupuncture—using a pen-like device that shines light on specific points on the body—can improve behavioral and psychological symptoms in people with dementia. Sixty participants will receive either real or sham laser treatment three times a week fo…

This study aims to adapt and validate a French version of the Recovery Assessment Scale (RAS), a questionnaire that measures how people feel about their own recovery from mental health conditions. Researchers will ask 260 adult psychiatric patients in France to fill out the RAS a…

This study will test an Italian version of a questionnaire called the dynamic Neurocognitive Adaptation (dNA) scale in 265 older adults, including those with normal aging, mild memory complaints, or dementia. The goal is to see if higher scores on the scale—reflecting more engage…

This study will test blood samples from 450 people aged 6 to 60 with inherited neuromuscular diseases to see if they have antibodies that could block gene therapy viruses (AAVs). The goal is to understand how common these antibodies are and which patients might be good candidates…

This study will test a tool called the Health Improvement Profile (HIP) in a mental health inpatient unit. The HIP is a checklist that helps clinicians spot physical health problems like high blood pressure or poor diet in people being treated for mental illness. Researchers will…

This study aims to better understand psychiatric disorders, thinking problems, and treatment side effects in 2000 cancer patients and their close relatives. Researchers will compare those with and without mental health issues to find patterns and improve care. No new treatments a…

This study is testing a digital toolkit that uses AI and wearable sensors to assess pain and distress in people with mild to moderate dementia. The goal is to see if the toolkit is easy to use and accurate. If it works, it could help caregivers better understand and respond to th…

This study will compare how well an AI program (Blings) measures speech clarity in 40 adults with speech disorders from stroke or other neurological conditions. Two speech therapists will also rate the same speech samples. The goal is to see if the AI can reliably replace or assi…

This study will check how much psychiatric clients know about occupational therapy (OT) and whether their expectations are realistic. It will also evaluate how well OT interns explain health information and conduct interviews. Researchers will develop new tools to measure these s…

This 10-year observational study will follow 10,000 adults with diabetes, metabolic disorders, and related conditions like high blood pressure and fatty liver disease. Researchers will collect routine medical data to identify markers of disease severity and activity. The goal is …