About Huntington's Disease
Huntington's disease is an autosomal dominant neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the HTT gene encoding huntingtin protein. It causes progressive decline in movement, cognition, and psychiatric function. There is currently no disease-modifying treatment, making the clinical trial landscape extremely active. Approaches under investigation include antisense oligonucleotides (ASOs) targeting huntingtin mRNA, RNA interference, small molecule lowering agents, and neuroprotective strategies.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Huntington's Disease trials.
Predictive genetic testing is available for at-risk individuals — genetic counselling is strongly recommended before testing.
Many HD trials now enrol pre-manifest gene carriers years before symptom onset — a positive gene test may make you eligible sooner.
The ENROLL-HD global registry is the most important resource for HD trial access — enrol even if you have no current symptoms.
Patient Resources
Find recruiting Huntington's Disease trials
Search 500,000+ studies from ClinicalTrials.gov, filtered for Huntington's Disease. Updated daily.