Huntington’s Disease is a rare, inherited brain disorder that causes problems with movement, thinking and mood. The study looks at a new oral tablet called votoplam (code name HTT227) compared with a placebo. The medication is taken by mouth as a tablet.
The purpose of the study is to see whether votoplam can slow the progression of Huntington’s Disease. Participants will be randomly assigned to receive either the study drug or the placebo, and neither the participants nor the researchers will know which one is given. The study lasts about three years, with regular visits to check health, perform simple tests, and collect blood samples.
During the visits, doctors will use a questionnaire called the Unified Huntington’s Disease Rating Scale to track changes in movement, thinking and daily abilities. Blood will be checked for a protein called mutant huntingtin (a faulty version of a normal brain protein), and safety will be monitored by recording any side effects. The information collected will help understand if the drug is safe and helpful.



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