RecruitingPHASE1INTERVENTIONAL

A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease

This is a new research study testing an investigational medicine called SRP-1005 for people living with Huntington's disease. It's an early-stage trial, meaning it's the very first time this specific medicine is being given to humans. Researchers want to understand if SRP-1005 is safe and how it works in the body of people with Huntington's. Some participants will receive SRP-1005, while others will receive a placebo (a dummy medicine with no active ingredients). This helps researchers compare the effects accurately. The study is being carried out in different medical centres.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Sarepta Therapeutics, Inc.

Enrolment target

Start

12 May 2026

Estimated completion

30 Nov 2027

Huntington's Disease

What is this study about?

This research is an important first step in understanding a new medicine called SRP-1005 for Huntington's disease. Huntington's is a condition that affects the brain and can cause uncontrolled movements, problems with thinking, and changes in mood. Currently, there is no cure for Huntington's disease, so finding new ways to help manage or slow down the condition is crucial.

The main goal of this study is to find out if SRP-1005 is safe for people to take and to see how their bodies react to it. Because it's the 'first-in-human' study, it means no one has received this particular medicine before. This is a standard process for all new medicines before they can be further developed. Participants in the study will either receive SRP-1005 or a placebo, which looks exactly like the study medicine but doesn't contain any active ingredients. This allows researchers to compare the effects and determine if the medicine is making a difference.

This early stage of research, called Phase 1, focuses on safety. If the medicine is found to be safe and shows promising signs, it might then move on to larger studies to test how well it works. This kind of research is vital for developing future treatments for Huntington's disease.

Key takeaways

This is an early-stage study testing a new medicine for Huntington's disease.
The main goal is to check the medicine's safety in people for the first time.
Participants will receive either the new medicine or a placebo.
It requires specific genetic diagnosis and stage of Huntington's disease.
Regular clinic visits, blood tests, MRIs, and lumbar punctures are part of the study.
You can stop participating at any time.

Who may be eligible?

To take part in this study, you would need to be diagnosed with Huntington's disease, and your diagnosis must be confirmed by genetic testing. Your condition should be at a specific stage, either what doctors call 'Stage 2' or 'Mild Stage 3' according to a special assessment tool for Huntington's disease.

There are also some things that might mean you can't join. For example, if you have other serious brain or body illnesses, or if you have certain infections like HIV or hepatitis. You also can't have recently taken part in other studies testing new medicines or specific therapies for Huntington's disease.

Your age is also a factor; participants need to be between 21 and 70 years old. If you, or your partner, could have children, you would need to agree to use effective contraception during the study and for about three months afterwards. Pregnant or breastfeeding individuals cannot participate. The study team will discuss all these points with you in detail.

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.

I have a confirmed genetic diagnosis of Huntington's disease.
My Huntington's disease is classified as Stage 2 or Mild Stage 3.
I am between 21 and 70 years old.
I don't have other serious medical conditions or infections like HIV.
I haven't recently taken part in other drug trials for Huntington's disease.
I am not pregnant or breastfeeding, and I understand contraception requirements.

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll have regular visits to a study clinic. These visits will involve various health checks, which might include blood tests, physical examinations, and possibly imaging scans like an MRI. You would also have a procedure called a lumbar puncture, which involves collecting a small sample of fluid from your spine, to understand how the medicine is affecting your body. This is a common procedure for studies looking at brain conditions.

During the study, you would receive either the investigational medicine, SRP-1005, or a placebo, which looks the same but contains no active medicine. Neither you nor your study doctor would know which one you are receiving. The study team will explain the schedule of visits, how often you'll take the medicine, and all the tests involved. The total duration of your participation, including follow-up, will be clearly explained by the research team.

Potential risks and benefits

Participating in any clinical trial involves both potential benefits and risks. For this early-stage study, the primary focus is on understanding the safety of SRP-1005. There is no guarantee that you will personally benefit from taking part, but your involvement could help researchers learn more about Huntington's disease and potentially contribute to developing future treatments for others. As with any medicine, there's a possibility of side effects from SRP-1005, and procedures like blood tests or lumbar punctures also carry minor risks, which the study team will explain fully. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Westmead Hospital
Verified postcode
Westmead, Australia· Recruiting
New Zealand Brain Research Institute
Verified postcode
Christchurch, New Zealand· Recruiting
Siloah AG
Verified postcode
Gümligen, Switzerland· Recruiting
University Hospital of Wales
Verified postcode
Cardiff, United Kingdom· Recruiting

Common questions

What is a 'first-in-human' study?

It means this is the very first time this particular medicine (SRP-1005) is being given to people. It's a key step to see if it's safe before more research can be done.

What is a 'placebo'?

A placebo is a 'dummy' medicine that looks just like the real medicine but contains no active ingredients. It helps researchers fairly compare the effects of the actual medicine.

Will I know if I'm getting the real medicine or the placebo?

No, during the study, neither you nor your study doctor will know whether you are receiving SRP-1005 or the placebo. This is done to ensure the study results are unbiased.

What is a lumbar puncture?

A lumbar puncture is a procedure where a small needle is used to collect a sample of fluid from your lower back, usually to check for certain markers or how a medicine is affecting your brain and spinal cord.

Can I stop participating in the study if I change my mind?

Yes, you are completely free to withdraw from the study at any time, for any reason. Your decision will not affect your routine medical care.

How to find out more

Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4

sareptally@sarepta.com 1-888-SAREPTA (1-888-727-3782) Official trial record

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