AuthorisedTherapeutic confirmatory (Phase III)Interventional

IB1001-304: Effects of N-Acetyl-L-Leucine on CACNA1A Disorders: A Phase III, randomized, placebo-controlled, double-blind, crossover study

This study, called IB1001-304, is looking into a new medicine called N-Acetyl-L-Leucine for people with a condition called CACNA1A disorder. This condition can affect how well people move and coordinate their bodies. In this study, some people will get the new medicine, and others will get a dummy medicine (placebo) to see if the new medicine really helps. It’s a 'Phase III' study, which means it's a large, final step before a medicine might become widely available. Doctors will use a special rating scale called SARA to measure any changes in movement and coordination, aiming to see if the new medicine improves these symptoms.

At a glance

Status

Authorised

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Intrabio Limited, IntraBio Inc

Enrolment target

Start

25 Mar 2026

CACNA1A Disorders

What is this study about?

This study is investigating a potential new treatment for CACNA1A disorders. These conditions are genetic and can lead to problems with movement, balance, and coordination, often referred to as ataxia. The new medicine being tested is called N-Acetyl-L-Leucine. This study is designed to carefully compare the effects of this new medicine against a placebo, which is a dummy treatment. This helps researchers understand whether any improvements seen are truly due to the medicine or other factors.

The study involves a 'crossover' design. This means that at different times, participants will receive both the active medicine and the placebo. This allows researchers to compare how each individual responds to both treatments, making the results more reliable. All participants will be closely monitored, and their progress will be measured using specific tests. The main way they'll check for improvements is with a scale called SARA, which assesses how well someone can move and coordinate.

By running this study, researchers hope to determine if N-Acetyl-L-Leucine is a safe and effective treatment that could improve the quality of life for people living with CACNA1A disorders. Finding new treatments for conditions like this is a big step towards better care and support.

Key takeaways

This study is testing a new medicine for CACNA1A disorders.
It's a rigorous 'Phase III' study, meaning it's a late stage of research.
Participants will receive both the new medicine and a 'dummy' medicine (placebo) at different times.
A special scale called SARA will measure changes in movement and coordination.
The study aims to find out if the new medicine is safe and effective.

Who may be eligible?

To join this study, people generally need to be 18 years old or older. The study is open to both men and women, as CACNA1A disorders can affect anyone.

There might be other specific health requirements or conditions that would make someone suitable or unsuitable for this study. These could include the type or severity of their CACNA1A disorder, other medications they might be taking, or their general health. These details are important to ensure the study is safe for participants and that the results are clear.

If you're interested, it’s always best to discuss with your doctor whether you might be a good fit for this type of research.

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 old or older?
Do you have a diagnosis of a CACNA1A disorder?
Are you able to attend regular study appointments?
Are you willing to potentially take either the study medicine or a placebo?
Are you able to follow study instructions carefully?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would first have a screening visit to make sure you meet all the necessary requirements. Over the course of the study, you'd regularly attend clinic visits where doctors would check your health, measure your movement and coordination using scales like SARA, and ask about your general well-being. You would take the study medicine, N-Acetyl-L-Leucine, or the placebo, in granule form mixed into a liquid, as instructed. Because it's a 'crossover' study, at certain points you would switch between taking the active medicine and the placebo. The study will also involve follow-up visits to monitor your health after you've finished taking the study medication. The total duration of your involvement, including all visits and follow-ups, would be explained in detail before you decide to join.

Potential risks and benefits

Taking part in a clinical trial offers the chance to access a new potential treatment before it's widely available and contributes to medical knowledge that could help others. However, there's no guarantee the medicine will help you, and it might have side effects that aren't yet fully known. As with any medicine, you could experience discomfort or reactions. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

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

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Unverified
Italy
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Unverified
Germany
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Unverified
Austria

Common questions

What is a 'CACNA1A disorder'?

It's a health condition caused by a change in a specific gene called CACNA1A, which often leads to problems with movement, balance, and coordination.

What is 'N-Acetyl-L-Leucine'?

This is the name of the new medicine being tested in this study to see if it can help improve symptoms of CACNA1A disorders.

What does a 'Phase III' study mean?

It means this is a large, final stage of testing for the medicine, conducted after earlier smaller studies, to confirm if it's safe and effective.

What is a 'placebo'?

A placebo is a dummy treatment that looks just like the real medicine but contains no active ingredients. It helps researchers know if the real medicine is actually working.

What is 'SARA' and why is it used?

SARA is a special rating scale used by doctors to measure how well someone can move and coordinate. It helps them track any changes in symptoms during the study.