Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease
A Phase 2/3 Randomized, Double Blind, Placebo-Controlled, Dose Ranging Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
Skyhawk Therapeutics, Inc.
120 participants
May 27, 2025
INTERVENTIONAL
Conditions
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. This study includes men and women aged 25 and older who have HD confirmed by genetic testing and meet certain requirements for physical ability and independence.
Eligibility
Inclusion Criteria8
- 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 Criteria11
- 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.
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Interventions
Study Drug Name (INN): SKY-0515 Type: Drug (small molecule) Dosage Level(s): Low, mid, high Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 12 months Use: Experimental Sourcing: SKY-0515 will be provided centrally by the Sponsor or subsidiary, or designee Packaging and Labeling: Study drug will be provided in blinded bottles labeled as required per country requirement. All packaging and labeling operations for the study drug will be performed according to current Good Manufacturing Practice for Medicinal Products and the relevant regulatory requirements.
Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 12 months Use: Experimental Sourcing: SKY-0515 placebo will be provided centrally by the Sponsor or subsidiary, or designee Packaging and Labeling: Study drug will be provided in blinded bottles labeled as required per country requirement. All packaging and labeling operations for the study drug will be performed according to current Good Manufacturing Practice for Medicinal Products and the relevant regulatory requirements
Locations(12)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06873334