NCT07378644 · Phase 2 · RECRUITING
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
This trial tests an oral drug called SKY-0515 in people with Huntington's disease. Researchers want to see whether it lowers harmful proteins linked to the condition and whether it improves symptoms. It is a Phase 2 trial, meaning it is testing whether the drug works and is safe. It is not yet proven or approved.
You may qualify if
- 25 years or older.
- Huntington's Disease confirmed through genetic testing, with a specific change in exon 1 of the HTT gene (CAG repeat of 40 or more).
- Total Functional Capacity (TFC) score of 10 or more).
- Total Motor Score (TMS) of 6 or more).
- Independence Score (IS) of 70 or more).
- Women who can have children must have a negative pregnancy test before starting and use two types of birth control during the study and for 30 days after the last dose of the study drug.
- Men must agree to use birth control during the study and for 90 days after the last dose.
- Agree to sign a consent form and follow the study's rules and schedule.
You're excluded if
- Other Serious health problems or brain/spinal issues that could interfere with the study or make procedures unsafe.
- Conditions that interfere with protocol-specified assessments, like an implanted medical device or difficulty getting an MRI.
- Cancer, except for some types of skin cancer, or a history of cancer in the last five years.
- Severe allergies or have reacted badly to similar drugs in the past.
- Taking medications or treatments that might interfere with the study.
- Participated in another study or taken experimental drugs in the last two months (or longer for some drugs).
- Any kind of gene therapy.
- History of suicidal thoughts, severe depression, or have attempted suicide in the past year.
- Liver function tests show significant abnormalities.
- Positive for hepatitis B, hepatitis C, or HIV.
- Pregnancy, breastfeeding, or planning to become pregnant during the study.
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2026-10-16