Not yet recruitingPHASE3INTERVENTIONAL

Pivotal Study of N-acetyl-L-leucine for CACNA1A

This study is a research trial exploring a new potential treatment called N-acetyl-L-leucine (also known as IB1001) for individuals aged 4 and older who have a genetic condition related to CACNA1A. These conditions can include types of ataxia (problems with movement and balance) and certain severe migraines. Researchers want to understand if this new treatment is safe to use, how well people tolerate it, and if it can help improve symptoms compared to a placebo (a dummy pill with no active medicine). It's a 'Phase 3' study, meaning it's a key step in testing the treatment before it might be considered for wider use. The study is designed very carefully to get reliable results, involving participants receiving both the study medicine and the placebo at different times.

At a glance

Status

Not yet recruiting

Phase

PHASE3

Sponsor

IntraBio Inc

Enrolment target

Start

01 Sep 2026

Estimated completion

01 Nov 2028

CACNA1A Spinocerebellar Ataxia Type 6 Episodic Ataxia Type 2 Familial Hemiplegic Migraine-1

What is this study about?

This study is looking into a new medicine called N-acetyl-L-leucine, sometimes called IB1001, for people who have conditions linked to a gene called CACNA1A. These conditions can cause issues with movement and balance, like spinocerebellar ataxia type 6 and episodic ataxia type 2, or a type of severe headache known as familial hemiplegic migraine-1. Finding better ways to manage these conditions is really important for those affected and their families.

The main goal of this study is to see if N-acetyl-L-leucine is safe to use, if people can take it without too many difficulties (what we call 'tolerability'), and if it actually helps improve symptoms. To do this fairly, the new medicine is being compared to a 'placebo', which is a pill that looks the same but doesn't contain any active medicine. This helps researchers understand if any changes people feel are truly due to the new medicine or other reasons.

This is a 'Phase 3' study, which means it's a large and important step in testing the medicine. If it proves to be safe and effective in this phase, it could potentially become a treatment doctors can prescribe in the future. The study is being carried out in different centres and designed to be very thorough to make sure the results are as clear and reliable as possible.

Key takeaways

This study is testing a new medicine (IB1001) for CACNA1A-related conditions.
It's a 'Phase 3' trial, a key step in developing new treatments.
Participants will receive both the study medicine and a dummy pill at different times.
Children aged 4 and older, as well as adults, can participate.
Safety, tolerability, and effectiveness are the main things being studied.

Who may be eligible?

To join this study, you need to be at least 4 years old and have a confirmed diagnosis of a CACNA1A-related condition. This diagnosis must be based on genetic tests. Examples of these conditions include certain types of ataxia (problems with movement and balance) and some severe forms of migraine.

If you are a female and could potentially get pregnant, you can still take part, but you'll need to either be sexually inactive throughout the study or use a highly effective method of contraception. This is to ensure your safety and the accuracy of the study results. Your doctor can help explain which contraception methods are considered highly effective.

Females who are unable to get pregnant, either because they have had certain sterilisation procedures at least six months before starting the study, or who have gone through menopause at least a year ago, can also participate.

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 aged 4 years or older?
Do you have a genetically confirmed diagnosis of a CACNA1A-related condition?
If you are female, are you using highly effective contraception or unable to get pregnant?
Are you able to provide written consent for yourself or through a legal guardian?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will involve three main periods. First, there's a short 'baseline' period of about two weeks where doctors will get a starting picture of your health. After this, you'll be randomly assigned to either receive the study medicine (IB1001) or a placebo (a dummy pill) for about 12 weeks. Neither you nor your study doctor will know which one you are taking – this is called 'double-blinded'.

After these 12 weeks, you'll then 'switch over' to the other treatment (if you had the medicine, you'll get the placebo, and vice versa) for another 12 weeks. So, everyone in the study will get both the active medicine and the dummy pill at different times. During each of these periods, you'll have two assessment visits.

Once you complete the main part of the study, you might be offered the chance to join an 'Extension Phase'. This phase is designed to allow you to continue receiving the IB1001 medicine if you wish, providing continued access to the potential treatment.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of joining this study could be access to a new treatment that might help with your condition, which isn't yet widely available. However, there's no guarantee the treatment will work for you, and you might receive the placebo for a period. Potential risks involve side effects from the experimental medicine, which will be carefully monitored. You will be closely watched by medical staff throughout the study, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (10)

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

Johns Hopkins University
Verified postcode
Baltimore, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
The University of Texas Health (UT Health)
Verified postcode
Houston, United States
Medical University of Innsbruck
Verified postcode
Innsbruck, Austria
University of Cologne
Verified postcode
Cologne, Germany
University of Athens
Verified postcode
Athens, Greece
University of Milan
Verified postcode
Milan, Italy
Bambino Gesu' Children's Research Hospital
Verified postcode
Rome, Italy
University Hospital Bern Inselspital
Verified postcode
Bern, Switzerland
University of Oxford - John Radcliffe Hospital
Verified postcode
Oxford, United Kingdom

Common questions

What is CACNA1A?

CACNA1A is a gene, and problems with this gene can lead to conditions that affect movement, balance, and sometimes cause severe headaches.

What does 'placebo-controlled' mean?

It means some people will get the active medicine, and some will get a dummy pill with no medicine inside. This helps researchers see if the real medicine is actually making a difference.

What is a 'Phase 3' study?

Phase 3 is an important stage where a new medicine is tested on a larger group of people to confirm its safety and how well it works before it can be considered for general use.

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

No, neither you nor your study doctor will know which treatment you are receiving at any given time. This is done to keep the study fair and unbiased.

What is the 'Extension Phase'?

If you complete the main study, an Extension Phase might be offered, allowing you to continue receiving the active study medicine if you choose.

How to find out more

Taylor Fields

tfields@intrabio.com +447426956369 Official trial record

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