Active not recruitingPHASE3INTERVENTIONAL

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)

This important study, known as a Phase 3 trial, is looking into a new medicine called N-Acetyl-L-Leucine (also called IB1001) for people aged 4 and over who have Ataxia-Telangiectasia (A-T). We want to find out if this medicine is safe to use, how well people tolerate it, and if it helps with the symptoms of A-T. To do this, some participants will receive the active medicine, and others will receive a dummy treatment (placebo). This is a 'blinded' study, meaning neither the patient nor the study team will know who is getting which treatment until after the study ends. This helps us get the most accurate results about the medicine's effects.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

IntraBio Inc

Enrolment target

Start

18 Mar 2025

Estimated completion

01 Jun 2028

Ataxia-Telangiectasia Ataxia-Telangiectasia (A-T)

What is this study about?

Ataxia-Telangiectasia, often shortened to A-T, is a rare genetic condition that affects the brain and other parts of the body. It can cause problems with movement, balance, and speech, and can also impact the immune system. Researchers are always looking for new and better ways to help manage the symptoms of A-T and improve the quality of life for those living with it.

This study is a 'Phase 3' trial, which means it's a key step in testing whether a new medicine is ready to be used more widely. It's designed to compare the new medicine, N-Acetyl-L-Leucine (IB1001), with a ‘placebo’ – a treatment that looks just like the real medicine but contains no active drug. This is the best way to understand if any changes seen are truly due to the new medicine or if they might have happened anyway.

The main goal of this study is to find out if N-Acetyl-L-Leucine is safe for people with A-T, how well their bodies handle it, and most importantly, if it can make a positive difference to their symptoms. The results from this study could help decide if this medicine could one day become a new treatment option for A-T.

Key takeaways

This study is testing a new medicine for Ataxia-Telangiectasia (A-T).
It's a Phase 3 trial, a crucial step before broader use.
Participants will receive both the active medicine and a placebo at different times.
The study aims to check safety, how well it's tolerated, and if it helps A-T symptoms.
It involves regular check-ups over about 6 months.
You can stop participating at any time.

Who may be eligible?

To join this study, people need to be at least 4 years old and have a confirmed diagnosis of Ataxia-Telangiectasia (A-T) through genetic testing. Parents or legal guardians will need to give their permission for children to take part, and adults will give their own consent.

There are also some specific rules about pregnancy and contraception for both women and men. Women who could become pregnant must agree to use effective birth control during the study and for a period afterwards. This is to ensure the safety of any potential future pregnancies. Women who are past menopause or have had certain surgeries are also eligible.

Men participating in the study who are able to father children will also need to use condoms with spermicide during the study and for a period after their last dose of medication. These measures are common in clinical trials to protect participants and ensure the study's safety.

Quick self-check

Are you at least 4 years old?
Do you have a genetically confirmed diagnosis of Ataxia-Telangiectasia (A-T)?
Are you able to agree to the study's contraception guidelines (if applicable)?
Can you commit to around 6 months for the main study with regular visits?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, the study will involve a few different stages. First, there's a short 'baseline' period of about two weeks, where doctors will get a clear picture of your current health and symptoms. After that, you'll be randomly assigned to receive either the study medicine (IB1001) or a dummy treatment (placebo) for about 12 weeks. This is done by chance, like flipping a coin. Neither you nor the study team will know which treatment you are getting at this stage.

After the first 12 weeks, you'll switch over to the other treatment – so if you had the medicine, you'll now get the placebo, and vice versa. This second treatment period will also last about 12 weeks. During each treatment period, you'll have two check-ups with the study team. In total, the main part of the study will last around 26 weeks. After the main study, some people might be given the chance to continue receiving the medicine in a special 'extension phase', which means they'd have continued access to IB1001.

Potential risks and benefits

Taking part in a clinical trial may offer some potential benefits, such as access to a new medicine before it's widely available and closer monitoring of your health by medical professionals. However, there are also potential risks, including side effects from the medication or the inconvenience of study visits. While the study medicine is being tested for safety, it's possible that unknown risks might emerge. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (11)

University of California Los Angeles
Los Angeles, United States
Massachusetts General Hospital
Boston, United States
The University of Texas Health (UT Health)
Houston, United States
University of Cologne
Cologne, Germany
University of Giessen
Giessen, Germany
Comenius University Bratislava
Bratislava, Slovakia
University Hospital L. Pasteur
Košice, Slovakia
Hospital Universitario La Paz
Madrid, Spain
University of Bern
Bern, Switzerland
Royal Papworth Hospital
Cambridge, United Kingdom
University of Nottingham
Nottingham, United Kingdom

Common questions

What is a 'placebo'?

A placebo is a dummy treatment that looks exactly like the study medicine but contains no active drug. It helps researchers see if the real medicine is having an effect.

What does 'double-blind' mean?

It means that neither you (the patient) nor the study doctors will know whether you are receiving the active medicine or the placebo. This helps prevent any bias.

Will I eventually get the actual medicine if I'm on the placebo?

Yes, because this study uses a 'cross-over' design, you will receive both the active medicine (IB1001) and the placebo at different times during the study.

How long will I be in the study?

The main part of the study will last about 26 weeks (roughly 6 months), including a short initial period and two 12-week treatment periods.

What happens after the study ends?

After the main study, you might have the option to join an 'extension phase' to continue receiving the study medicine, if the researchers decide it's appropriate.