Rett syndrome

This study tests a gene therapy called NGN-401 in 33 girls with Rett syndrome, a rare genetic disorder that affects development and movement. The therapy delivers a working copy of the MECP2 gene to try to improve skills and daily function. Researchers will measure success by whe…

This phase 2 trial tests whether lamivudine, an antiviral drug, can improve symptoms in people with Rett syndrome, a rare genetic disorder that affects movement, communication, and behavior. Ten participants will take the drug by mouth for a set period, and researchers will measu…

This early-phase trial tests a single injection of AAV-MECP2 gene therapy into the spinal fluid of 8 girls aged 4-10 with Rett syndrome, a severe neurodevelopmental disorder. The goal is to see if the treatment is safe and can reduce disease severity. The study is not yet recruit…

This study tests whether intensive home-based psychiatric treatment can help children and teens with mental health disorders as an alternative to being in the hospital. Over 3 months, participants receive 3 to 7 home visits per week from a team of doctors, therapists, and other s…

This study compares two ways to help people with both substance use and mental health disorders: a full team-based program called MISSION that provides counseling, peer support, and community outreach for 6 months, versus just linking them with a peer specialist. Researchers hope…

This study tests a new way of delivering mental health and addiction services to youth aged 14-17. Instead of standard hospital outpatient care, an integrated team offers rapid access to various services like therapy, peer support, and primary care in a youth-friendly setting. Re…

This study looks at whether having a trained peer (someone with similar experiences) as a case manager can help people with HIV who also have mental health or substance use problems get the care they need. About 405 adults from an HIV clinic will be randomly assigned to either us…

This study tests a new gene therapy called TSHA-102 for girls aged 5 to 8 with Rett syndrome, a severe genetic disorder that affects brain development. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and side effects, while also …

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study tests whether an AI tool can help radiologists read brain CT and MRI scans more accurately and quickly. Researchers will compare how well doctors, AI alone, and doctors using AI together can spot abnormalities, urgent findings, and classify diseases. The goal is to red…

This study tests whether wearing a virtual reality (VR) headset during dental treatment can reduce fear and stress in children with mild intellectual disabilities or autism spectrum disorder. Two hundred children aged 6 to 12 will either receive standard dental care or use a VR h…

This pilot study tests a specific type of drama therapy for people who have both a mental health condition (like depression or bipolar disorder) and a substance use disorder. Ten participants will attend weekly group drama therapy sessions for 12 weeks, then put on a performance.…

This study tests whether personalized computer games can help people with Rett syndrome use their arms more purposefully and reduce repetitive hand movements. Fourteen participants will play games that reward them for separating their hands and reaching for targets. The training …

This study tests a group therapy called Awareness, Courage, and Love (ACL) for older adults with psychiatric disorders who feel lonely. The therapy focuses on mindfulness, sharing feelings, and building connections. Researchers want to see if the program is practical and helpful …

This study tests a wearable robotic exoskeleton called EXPLORER in 15 children with movement problems from conditions like cerebral palsy. The goal is to see if it helps them walk better in their everyday environments, such as home and the community. The study focuses on safety, …

This study tests a robotic feeding device called Obi for people who cannot use their arms to eat on their own. About 50 participants, including patients, caregivers, and healthcare providers, will use the device at home or in care settings for one week. Afterward, they will fill …

This study tests a 10-week program called 'Grow to Recovery' for people with mental illness. The program is co-led by a peer (someone who also has a mental illness) and a professional. Researchers want to see if it helps participants feel more hopeful and capable. 138 people are …

This study tests whether an early assessment of activity limitations by occupational therapists can help people with mental disorders return to work. 150 participants in specialized psychiatric care will either receive the assessment or usual care. The goal is to see if this lead…

This study looks at whether an 8-week program of daily brain training exercises, combined with a gentle electrical current applied to the scalp (tDCS), can improve thinking, reduce falls, and help seniors with mental health conditions stay independent. About 270 older adults livi…

This study tests whether a 12-week program that combines brain training, social activities, and exercise is practical and liked by seniors with mental health conditions living in community housing. About 18 participants will try the program in small groups, guided by a support wo…

This study is developing and testing a smartphone app called R-Assist for people recently released from jail who have both substance use and mental health disorders. The app tracks symptoms, connects users to resources like housing and recovery groups, and measures how easy it is…

This study looks at tobacco, cannabis, and other drug use among homeless and at-risk youth in downtown Ottawa. Researchers will follow 520 participants aged 16 and older who are homeless or insecurely housed and have a history of cannabis use and mental illness. The goal is to un…

This study tests a method called SAIA-MH to help clinics in Mozambique better diagnose, treat, and follow up with people who have mental health conditions. Researchers will compare clinics using this approach to those that don't, involving about 155 patients. The goal is to see i…

This study follows 192 teens aged 12 to 20 who are already receiving either Cognitive Behavioral Therapy (CBT) or Brief Strategic Therapy (BST) for anxiety, depression, or OCD. Researchers will track their progress using questionnaires and interviews over 9 months. The goal is to…

This study gathers MRI, CT, and ultrasound images from 300 adults—some healthy, some with kidney or brain disease—to build a collection for future not-for-profit research. No new treatments are being tested; the goal is to make medical images available to scientists for advancing…