RecruitingPHASE2, PHASE3INTERVENTIONAL

Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease

This clinical trial is looking into a new oral medication called SKY-0515 for people with Huntington's disease (HD). HD is a serious condition that affects the brain, causing movement, thinking, and mental health problems. The drug aims to lower specific harmful proteins, which are thought to cause damage in the brain. By reducing these proteins, the researchers hope to improve symptoms for people living with HD. This study involves men and women aged 25 and older who have a confirmed genetic diagnosis of HD and meet certain health and physical ability requirements. Participants will be randomly given either the study drug or a placebo (a dummy pill) to compare their effects. Safety and effectiveness will be carefully monitored throughout the trial.

At a glance

Status

Recruiting

Phase

PHASE2, PHASE3

Sponsor

Skyhawk Therapeutics, Inc.

Enrolment target

400

Start

06 Jan 2026

Estimated completion

01 Aug 2029

Huntington Disease

What is this study about?

This study is focused on Huntington's disease (HD), a serious inherited illness that affects a person's brain over time. HD is caused by a faulty gene that leads to the build-up of a problematic protein called huntingtin, which damages nerve cells and causes the wide range of symptoms seen in HD, such as problems with movement, thinking, and mood. The new medication, SKY-0515, is an oral drug, meaning it's taken by mouth, and it's designed to specifically target and reduce the levels of this harmful huntingtin protein in the body. Researchers believe that by lowering these protein levels, the drug could slow down the progression of HD and help improve symptoms for those affected.

The trial is divided into two main stages (Phase 2 and Phase 3), which means it's a detailed investigation into whether SKY-0515 is safe and effective enough to be considered a new treatment for HD. Participants will be randomly assigned to receive either the active drug at different doses or a placebo (a dummy pill with no active ingredient). This comparison helps the researchers understand the true effects of the drug. They will closely monitor participants' health, changes in their symptoms, and how the drug affects their bodies using various tests.

Ultimately, the main goal of this research is to find new and better ways to treat Huntington's disease. If SKY-0515 proves to be safe and effective, it could offer a new hope for managing this challenging condition and improving the daily lives of people living with HD. This is why careful and thorough research like this study is so important.

Key takeaways

New drug (SKY-0515) for Huntington's disease being tested.
Aims to reduce harmful proteins and improve symptoms.
Participant age: 25 years and older with confirmed HD diagnosis.
Involves regular clinic visits, blood tests, and MRI scans.
You might receive the drug or a placebo (dummy pill).
Participation is voluntary, and you can withdraw anytime.

Who may be eligible?

To be considered for this study, you need to be at least 25 years old and have a confirmed diagnosis of Huntington's disease through a genetic test. Your genetic test results should show a specific change in the huntingtin gene (called a CAG repeat of 40 or more). You also need to have certain levels of physical ability and independence, which will be assessed by the study team.

There are also some health conditions or situations that would prevent you from joining. For example, if you have other serious health problems, certain types of cancer, or a history of severe allergies to similar drugs, you would not be able to take part. Also, if you've recently been in another clinical trial or taken experimental drugs, there might be a waiting period before you can join this one. Your doctor will carefully review your full medical history to see if this study is right for you.

Additionally, if you are a woman who could become pregnant, you'll need to have a negative pregnancy test and agree to use two reliable forms of birth control throughout the study and for 30 days after your last dose. Men will also need to agree to use birth control during the study and for 90 days after their last dose.

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 25 years old or older?
Do you have a genetically confirmed diagnosis of Huntington's disease?
Do you meet certain independence and physical activity levels?
Are you able to attend regular clinic visits and have MRI scans?
Are you free from other serious health conditions like certain cancers or severe allergies?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be given either the study drug (SKY-0515) or a placebo (a dummy pill), which you will take by mouth. The study involves regular visits to the clinic for various assessments. These will include blood tests, brain scans (MRIs), and detailed evaluations of your Huntington's disease symptoms, such as your movement, thinking, and overall daily function. These assessments help the doctors understand how the drug is affecting your body and your symptoms.

Because this is a combined Phase 2 and Phase 3 study, it typically means it will last for a significant period, potentially several months to a few years, with follow-up appointments after you stop taking the study drug. The exact number of visits and the full duration of your participation will be clearly explained to you by the study team. You will receive all necessary information and support throughout your involvement in the trial.

Potential risks and benefits

Like all clinical trials, there are potential benefits and risks to consider if you participate. You might personally benefit if SKY-0515 is effective in reducing your harmful proteins and improving your symptoms. However, it's also possible that the drug may not help you, or you might experience side effects. The study team will carefully monitor your health throughout and inform you of any potential risks. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (8)

Buenos Aires British Hospital
City only
CABA, Argentina· Recruiting
Instituto Argentino de Investigación Neurológica
City only
CABA, Argentina· Recruiting
CINME Centro de Investigaciones Metabólicas
City only
CABA, Argentina· Recruiting
Fundación Scherbovsky
Verified postcode
Mendoza, Argentina· Recruiting
PSEG Centro de Pesquisa Clinica
Verified postcode
São Paulo, Brazil· Not yet recruiting
North York General Hospital
Verified postcode
Toronto, Canada· Not yet recruiting
Pineo Medical Ecosystem
Verified postcode
Tbilisi, Georgia· Recruiting
Simon Khechinashvili University Hospital
Verified postcode
Tbilisi, Georgia· Recruiting

Common questions

What is SKY-0515?

It's a new oral medication being tested to see if it can lower specific harmful proteins linked to Huntington's disease and improve symptoms.

Is this drug guaranteed to work?

No, this is a research study to find out if it works. There's no guarantee it will help you, but your progress will be closely monitored.

Will I know if I'm getting the drug or a dummy pill?

Neither you nor the study doctors will know, as this is a 'blinded' study. This helps ensure results are accurate.

How long will the study last?

The study will last for a significant period, potentially several months to a few years, including follow-up appointments. The exact duration will be explained by the study team.

Can I leave the study at any time?

Yes, you have the right to withdraw from the study at any point, for any reason, and it won't affect your regular medical care.

How to find out more

Study Director, MD

sky0515trials@skyhawktx.com 6178580041 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.