Huntington disease and related disorders

This early-stage trial tests a single injection of ARO-ATXN2 in 36 adults with spinocerebellar ataxia type 2, a rare genetic disorder that affects movement and coordination. The main goal is to see if the drug is safe and how the body processes it. Participants are randomly assig…

This phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo dail…

This study looks at whether the drug troriluzole can slow the progression of spinocerebellar ataxia (SCA), a rare genetic disease that affects movement and balance. Researchers will compare 909 patients who took troriluzole for up to three years with similar patients who did not …

This study tests an oral drug called SKY-0515 in 500 adults with Huntington's disease who have already completed a prior study of the same drug. The goal is to see if the drug can safely lower harmful proteins and improve symptoms over the long term. Participants must be 25 or ol…

This phase 2 trial tests an experimental drug called tominersen in 301 people with early-stage Huntington's disease. The drug aims to lower levels of the harmful mutant protein that causes the disease. Researchers will compare tominersen against a placebo to see if it can slow do…

This early-stage study tests a gene therapy called AB-1001 in 5 adults with early Huntington's disease. The treatment is injected directly into the brain to potentially slow the disease. The main goal is to check safety, but researchers will also look at brain changes on MRI and …

This study tests an oral drug called SKY-0515 in 120 adults with Huntington's disease. The goal is to see if it can lower harmful proteins in the blood, slow brain changes, and improve symptoms. Participants must be 25 or older with a confirmed genetic diagnosis and a certain lev…

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic disorder that affects movement and brain function. The drug aims to reduce the harmful effects of the mutated AT…

This study tests a custom-made genetic medicine (called an antisense oligonucleotide) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare inherited brain disorder. The treatment aims to reduce seizures and improve quality of life. Only one participan…

This early-stage study tests an experimental drug, ER2001, in 27 adults with early Huntington's disease. The main goal is to check if the drug is safe and how the body processes it. Participants receive either the drug or a placebo once a week for 6 weeks, with follow-up for 6 mo…

This study looks at the long-term safety and effects of the drug votoplam in people with Huntington's disease. About 144 participants who finished a previous votoplam study will take part. The goal is to see how the drug affects Huntington's protein levels and brain changes over …

This early-stage trial tests a one-time gene therapy called AMT-130, injected directly into the brain, to lower the production of the harmful huntingtin protein. It involves 43 adults with early Huntington's disease. The study is primarily checking safety, but also looks for sign…

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 looks at the long-term safety of valbenazine for people with Huntington disease who have involuntary jerking movements (chorea). It includes 7 participants from Canada who were in a previous valbenazine study. Everyone gets the drug, and researchers track side effects …

This study was designed to test whether a reusable pouch called DoddleBags could help people with Huntington's disease eat more easily and safely. The pouch holds pureed food and can be used with an anti-choking cap or spoon attachment. The trial was withdrawn before any particip…

This study tests whether deutetrabenazine can improve speech clarity and walking ability in people with Huntington disease. About 30 adults with early to moderate symptoms will take the drug and be evaluated using speech tests and motion capture technology. The goal is to see if …

This study tests whether a home-based brain stimulation device (tDCS) combined with activity therapy can help improve cognitive and language problems in people with stroke, brain tumors, or neurodegenerative conditions like Parkinson's or Alzheimer's. Fifty-five participants will…

This study tests a short guide called the 'Jumpstart Guide' to help doctors have better conversations with dementia patients and their families about healthcare goals. Over 1,800 patients and their caregivers are taking part. Researchers will compare whether those whose doctors u…

This study looks at 40 people with early-stage Huntington's disease and healthy volunteers to see how well two types of brain scans can detect changes over 2 years. Participants get PET and MRI scans plus clinical exams at the start and after 2 years. The goal is to find better w…

This observational study follows 37 children and young adults with juvenile-onset Huntington's disease (JoHD) to understand how their brains develop differently from adult-onset cases. Participants undergo cognitive tests, behavioral assessments, and MRI scans over time. The goal…

This study aims to create more sensitive cognitive tests for Huntington's disease by combining brain imaging (PET and MRI) with blood markers. Researchers will study 90 participants—healthy controls, pre-symptomatic gene carriers, and early-stage patients—to understand how brain …