Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

This research study is looking into a new treatment called zilganersen for Alexander Disease (AxD). The main goal is to find out if this medicine is safe and if it can help improve or stop the worsening of motor skills – that's your ability to move your body – for people living with AxD. The study involves a large number of participants and compares zilganersen with a placebo, which looks like the real medicine but contains no active ingredients. Participants won't know if they're getting the medicine or the placebo at first. After this initial period, everyone will have the chance to receive zilganersen. The study is particularly interested in how the medicine affects different aspects of movement and functioning in daily life.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Ionis Pharmaceuticals, Inc.

Enrolment target

Start

01 Jun 2021

Estimated completion

01 Sep 2029

Alexander Disease

What is this study about?

This study is investigating a new medicine called zilganersen specifically for people who have Alexander Disease (AxD). Alexander Disease is a very rare condition that affects the brain and can cause problems with movement, balance, and other body functions. There isn't a cure for AxD yet, so researchers are always looking for new ways to help.

The main purpose of this study is to see if zilganersen is safe for people with AxD, and if it can help improve or at least stabilise their movement abilities. This means looking at things like walking, using your hands, and other physical activities that might be affected by the disease. Half of the participants will receive the new medicine, while others will receive a placebo – a dummy treatment that looks just like the real medicine but has no active ingredients. This helps researchers truly understand if the medicine is making a difference.

This is a long-term study, and for the first part, neither you nor your doctor will know if you are receiving the medicine or the placebo. After this initial period, all participants will be offered the opportunity to receive zilganersen. The study also includes an option for younger children with AxD to take part. The hope is that zilganersen could offer a new way to manage the symptoms of Alexander Disease and improve the quality of life for those affected.

Key takeaways

This study is testing a new medicine, zilganersen, for Alexander Disease.
It aims to see if the medicine is safe and can help improve or stabilise your movement.
You might receive the medicine or a placebo (dummy treatment) initially.
Participation involves regular visits, assessments, and tests like MRI scans and lumbar punctures.
The study is long-term, lasting several years, with options for continued treatment.
It's crucial to meet specific health and age requirements to be eligible.

Who may be eligible?

To join this study, participants need to have a clear diagnosis of Alexander Disease, confirmed by brain scans and genetic tests showing a specific change in the GFAP gene. Participants can be between 2 and 65 years old. It's important that you and your family (if you're under 18) are able to attend all the necessary appointments, which will involve travel to the study centre.

However, there are certain reasons why someone might not be able to join. For example, if you have other serious health problems, unusual blood test results, or can't have an MRI scan, you might not be eligible. Also, if you've recently taken part in other drug studies (within the last month to a year, depending on the drug) or had certain brain surgeries, gene therapies, or a specific type of brain shunt, you wouldn't be able to participate. The study team will review your full medical history to make sure it's safe for you to take part.

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.

Do you have a confirmed diagnosis of Alexander Disease based on brain scans and genetic tests?
Are you between 2 and 65 years old?
Are you able to attend all required appointments at the study centre?
Have you not participated in other drug studies recently (check with your doctor)?
Have you not had certain brain surgeries, gene therapies, or shunts?
Are you generally in good health, without other serious medical conditions that would prevent participation?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be one of about 73 people with Alexander Disease in this study. For the first 60 weeks (about 14 months), you'll either receive the study medicine, zilganersen, or a placebo (a dummy treatment). You won't know which one you're getting. After this, everyone will then receive zilganersen for a further 60 weeks, and potentially for an even longer period of up to 120 weeks in an extension part of the study. There will also be a 28-week follow-up period after you stop taking the medicine.

Taking part will involve regular visits to the study centre. These visits will include checks of your health, physical examinations, and possibly tests like brain scans (MRIs) and lumbar punctures (where a small sample of fluid is taken from your spine). These are done to see how the medicine is affecting your body and the disease. If you are under 18, a parent or guardian will need to be with you and involved in the study requirements.

Potential risks and benefits

There is a chance that zilganersen could help improve or steady your movement abilities, or it might not have any effect. We don't yet know what specific benefits, if any, the medicine might offer. As with all medicines, there's a risk of side effects, which the study will carefully monitor. You will undergo various medical procedures, some of which carry their own small risks. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (13)

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

Lucile Packard Children's Hospital Stanford
Verified postcode
Palo Alto, United States
Children's Hospital of Atlanta
Verified postcode
Atlanta, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
Children's Hospital of Philadelphia
Verified postcode
Philadelphia, United States
Murdoch Children's Research Institute
Verified postcode
Parkville, Australia
McGill University Health Centre
Verified postcode
Montreal, Canada
Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center
Verified postcode
Tel Aviv, Israel
Ospedale dei Bambini Vittore Buzzi
Verified postcode
Milan, Italy
Ospedale Pediatrico Bambino Gesù
Verified postcode
Roma, Italy
National Center of Neurology and Psychiatry
Verified postcode
Tokyo, Japan
Amsterdam Universitair Medische Centra - Academisch Medisch Centrum
Verified postcode
Amsterdam, Netherlands
University College London Hospitals NHS Foundation Trust
Verified postcode
London, United Kingdom

Common questions

What is a placebo?

A placebo is a substance that looks just like the real study medicine but doesn't contain any active ingredients. It helps researchers compare how effective the new medicine is against not receiving the active treatment.

What does 'Phase 3' mean for a study?

Phase 3 is usually one of the final stages of testing a new medicine before it can be approved for wider use. It involves a larger number of patients and aims to confirm the medicine's effectiveness and safety over a longer period.

Will I have to travel for this study?

Yes, you will need to travel to a designated study centre for your appointments, tests, and to receive your medication.

What is a lumbar puncture?

A lumbar puncture is a medical procedure where a small needle is used to collect a sample of spinal fluid from your lower back. This fluid can tell doctors important information about your brain and spinal cord.

What happens if I'm under 18?

If you're under 18, a parent or guardian will need to provide permission for you to join the study and will be involved in managing your participation and appointments.