AuthorisedPhase II and Phase III (Integrated)Interventional

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

Huntington’s disease is a genetic condition that damages nerve cells in the brain over time. It can cause involuntary movements, emotional changes, and difficulties with memory and daily tasks. This clinical trial is researching an experimental medicine called SKY-0515 to see if it can help slow down this damage. Participants will take either SKY-0515 (at one of three different doses) or an inactive dummy pill called a placebo once a day. A computer chooses who receives the medicine and who receives the placebo, and neither participants nor doctors will know the choice during the study. Over 72 weeks (about 18 months), specialist doctors will regularly check participants' movement, thinking speed, mood, and daily independence. The aim is to find out if SKY-0515 is safe, well-tolerated, and effective at helping people stay independent for longer.

At a glance

Status

Authorised

Phase

Phase II and Phase III (Integrated)

Sponsor

Skyhawk Therapeutics Inc.

Enrolment target

110

Start

23 Jul 2026

Huntington’s Disease

What is this study about?

Huntington’s disease is an inherited illness that gradually damages nerve cells in specific parts of the brain. Over time, this damage leads to uncontrolled muscle movements, changes in mood and behaviour, and problems with thinking, planning, and memory. While there are treatments that help control some symptoms, there are currently very few options that can slow down the disease itself.

This trial is testing an experimental oral medicine known as SKY-0515. Researchers want to know whether SKY-0515 can protect nerve cells and reduce the rate at which Huntington’s disease progresses. The study tests three different strengths of the tablet (4 mg, 6 mg, and 9 mg) to find the dose that offers the best balance between helping symptoms and minimising side effects.

To be sure that any health improvements are truly caused by SKY-0515, the study compares the drug to a placebo. A placebo looks identical to the real tablet but contains no active medicine. Over roughly 18 months, doctors will track changes in physical coordination, memory, and everyday independence to see if those taking SKY-0515 do better than those taking the dummy pill.

Key takeaways

Tests a new experimental tablet (SKY-0515) for adults with Huntington’s disease.
Compares three different doses of the medicine against a dummy pill (placebo).
Tracks changes in movement, thinking, and daily independence over 72 weeks.
Open to adults aged 18 and over with a confirmed diagnosis.
Participation is voluntary, and you are free to leave the trial at any point.

Who may be eligible?

This study is open to men and women aged 18 and older who have been diagnosed with Huntington’s disease.

Because the study involves regular checks of movement, memory, and daily tasks, participants need to be at a stage of the condition where they can reliably complete these clinic visits and tests. Having a family member, partner, or carer who can support you through visits is often very helpful.

The medical team will check your full medical history and any current medicines you take. Certain other serious health conditions or medications may mean you cannot take part, as your safety is the top priority.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years of age or older?
Have you been diagnosed with Huntington’s disease?
Are you able to swallow tablets every day?
Are you able to attend regular clinic visits over an 18-month period?
Do you have a family member or carer who can help support your study visits if needed?

Answer every question to see your result.

What does participation involve?

Taking part in this study lasts for about 72 weeks (nearly a year and a half). During this time, you will take a tablet by mouth every day. You will be randomly assigned to take one of three doses of SKY-0515 or a placebo tablet. You and your trial doctor will not know which one you are taking.

You will need to attend regular clinic appointments so doctors can monitor your health. Visits will involve physical check-ups, simple movement tests, pen-and-paper thinking exercises, and questions about how you manage your day-to-day routine. You will also have standard safety checks, such as routine blood tests, urine tests, and heart tracings (ECGs).

Potential risks and benefits

Taking part in this study may or may not help your condition directly, particularly if you are assigned the placebo or a low dose. However, you will receive close, expert medical care throughout the trial, and the findings may help scientists develop better treatments for people with Huntington’s disease in the future. As with any new medicine, there is a risk of unwanted side effects, which the doctors will monitor carefully. Joining is entirely your choice, and you can withdraw from the study at any time without your routine NHS medical care being affected.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany
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Unverified
Spain

Common questions

What is a placebo?

A placebo is a dummy pill that looks exactly like the study medicine but contains no active drug. It allows doctors to see whether changes in health are due to the real medicine or other factors.

Will I know if I am taking SKY-0515 or the placebo?

No. This is a double-blind trial, meaning neither you nor your study doctor will know which treatment you receive until the trial is complete, unless there is a medical emergency.

How long will I be in the study?

The trial lasts for about 72 weeks, which is roughly 18 months, including regular clinic visits and daily medication.

Do I have to pay to take part?

No, all study tablets, medical tests, and clinic check-ups are free of charge. Travel costs to attend clinic visits are also usually reimbursed.

Can I leave the study early if I want to?

Yes. Participation is completely voluntary. You are free to stop taking part at any time without giving a reason, and your regular medical care will continue as normal.