IRB Study Number 26-128
Status Recruiting
Phase Phase 3
Location Cleveland Clinic Main Campus
Institute Neurological Institute
Description
This study will compare the safety and effects of Votoplam to a placebo in people with Huntington's disease (HD). Votoplam is an investigational (experimental) drug, which means it is not approved by the Food and Drug Administration (FDA). It is taken by mouth and intended to lower the amount of a protein called huntingtin (HTT), which plays a role in HD. By reducing this protein, Votoplam may help slow disease progression.
The following assessments will be performed during the study: blood and urine sample collection, brain magnetic resonance imaging (MRI), electrocardiograms (ECG/EKG), HD related scales and health questionnaires, ophthalmological (eye) exams, and lumbar punctures.
Participation will last from around 24-36 months. You will need to visit the study site between 11-15 times. The study staff may ask you to visit more often if they need to check your health more closely.
Inclusion Criteria
- Age 21-70 years
- Genetically confirmed HD diagnosis with a CAG repeat length of at least 40
- Ability to walk
Exclusion Criteria
- History of gene therapy, cell transplantation, or other experimental brain surgery for HD
- Serologic evidence for active viral hepatitis
- History or current diagnosis of ECG or cardiac abnormalities indicating significant safety risk
- Other criteria as listed in protocol
