SKY-0515-004-WW
Research type
Research Study
Full title
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
IRAS ID
1013537
Contact name
Brian Yoon
Contact email
Sponsor organisation
Skyhawk Therapeutics, Inc.
Clinicaltrials.gov Identifier
Research summary
Huntington's Disease (HD) is an inherited condition that affects movement, thinking, and emotions. It gets worse over time, and currently there are no treatments that can slow down the disease itself - only medicines to help manage
SKY-0515 is an experimental tablet that may help slow down Huntington's Disease by reducing the amount of harmful protein that causes the condition. Early studies in healthy volunteers and people with HD showed it was generally well-tolerated and successfully reduced these harmful proteins.
We will compare SKY-0515 to placebo (dummy tablets with no active medicine) to see if SKY-0515 works and is safe. About 400 people with early-stage HD will take part across multiple countries. Participants will be randomly assigned to receive either SKY-0515 (at different dose strengths) or placebo once daily for 72 weeks (about 18 months). Neither participants nor doctors will know which treatment someone is receiving.
Adults aged 25 or over with confirmed Huntington's Disease who are in the early stages with mild symptoms but can still manage daily activities independently.
We will assess movement, thinking abilities, daily functioning, and safety through regular clinic visits including physical exams, blood tests, brain scans, and questionnaires.
This research could lead to the first treatment that actually slows down Huntington's Disease progression, potentially helping people maintain their abilities and quality of life longer. Currently, no such treatments exist, making this study crucial for the HD community.REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
26/NE/0082
Date of REC Opinion
3 Jun 2026
REC opinion
Further Information Favourable Opinion