Status:
RECRUITING
Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease
Lead Sponsor:
Skyhawk Therapeutics, Inc.
Conditions:
Huntington Disease
Huntingtons Disease
Eligibility:
All Genders
25+ years
Phase:
PHASE2
PHASE3
Brief Summary
The goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD. T...
Detailed Description
This clinical trial aims to evaluate the safety, pharmacodynamics, and efficacy of SKY-0515, a novel, orally-administered small molecule mRNA-splicing modulator for treating Huntington's Disease (HD)....
Eligibility Criteria
Inclusion
- You must be 25 years or older.
- You must have Huntington's Disease confirmed through genetic testing, with a specific gene change (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.
- You must agree to sign a consent form and follow the study's rules and schedule.
Exclusion
- You have other serious health problems or brain/spinal issues that could interfere with the study or make procedures unsafe.
- You have a condition that interferes with protocol-specified assessments, like an implanted medical device or difficulty getting an MRI.
- You have cancer, except for some types of skin cancer, or a history of cancer in the last five years.
- You have severe allergies or have reacted badly to similar drugs in the past.
- You are taking medications or treatments that might interfere with the study.
- You've been in another study or taken experimental drugs in the last two months (or longer for some drugs).
- You have had any kind of gene therapy.
- You have a history of suicidal thoughts, severe depression, or have attempted suicide in the past year.
- Your liver function tests show significant abnormalities.
- You have tested positive for hepatitis B, hepatitis C, or HIV.
- You are pregnant, breastfeeding, or planning to become pregnant during the study.
Key Trial Info
Start Date :
May 27 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2027
Estimated Enrollment :
120 Patients enrolled
Trial Details
Trial ID
NCT06873334
Start Date
May 27 2025
End Date
December 1 2027
Last Update
November 17 2025
Active Locations (10)
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1
John Hunter Hospital
New Lambton Heights, New South Wales, Australia, 2305
2
Westmead Hospital
Westmead, New South Wales, Australia, 2145
3
The University of Queensland
Herston, Queensland, Australia, 4006
4
Flinders Medical Centre
Adelaide, South Australia, Australia, 5042