Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase 2a Study of TPN-101 in Patients with Aicardi-Goutières Syndrome (AGS)

This study is looking into a new medicine called censavudine for people who have Aicardi-Goutières Syndrome (AGS). AGS causes inflammation and can affect the brain. The main goal is to see if censavudine can reduce this inflammation, which doctors measure using special blood tests. Researchers will also be checking very carefully for any side effects the medicine might cause over a period of up to 48 weeks. This is a Phase 2a study, meaning it's an early step to understand if the medicine is safe and if it works well enough to be studied further in a larger group of people. Both men and women aged 18 and older can take part.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Transposon Therapeutics Inc.

Enrolment target

Start

12 Sep 2024

Aicardi-Goutières Syndrome (AGS)

What is this study about?

Aicardi-Goutières Syndrome (AGS) is a rare condition that affects the brain and can cause inflammation. This inflammation happens because the body's immune system, which usually fights off germs, becomes overactive and starts attacking healthy cells. Scientists believe that reducing this overactive immune response could help people with AGS.

This study is testing a new medicine called censavudine. Researchers hope that censavudine can calm down the immune system in people with AGS and reduce the level of inflammation in their bodies. They will be measuring this inflammation using special blood tests that look at certain markers. If censavudine can successfully reduce these markers, it could mean a big step forward in finding new ways to help people with AGS.

Throughout the study, the research team will also be carefully watching for any changes in the patients' health, including how they feel and function day-to-day. They will use different tests, like brain scans and assessments of movement and development, to see if the medicine has any positive effects. The main focus, alongside looking at inflammation, is to make sure the medicine is safe and well-tolerated by patients.

Key takeaways

Tests a new medicine (censavudine) for Aicardi-Goutières Syndrome (AGS).
Aims to reduce inflammation in the brain and body.
Specifically for adults aged 18 and over.
Involves taking the medicine for up to 11 months.
Checks for how safe the medicine is and if it makes a difference.
Careful monitoring through blood tests, scans, and other assessments.

Who may be eligible?

To be considered for this study, you must be 18 years old or older. The study is open to both men and women.

The researchers will need to check your medical history and do some tests to make sure that taking part is safe and suitable for you. They will confirm that you have Aicardi-Goutières Syndrome.

There might be other specific reasons why someone couldn't join, for example, if they have certain other health conditions or are taking particular medications. The study team will discuss all of these details with you and your family.

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 Aicardi-Goutières Syndrome (AGS)?
Are you willing to attend regular hospital visits for up to 11 months?
Are you comfortable with blood tests, and potentially brain scans and spinal fluid tests?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you will be taking a new medicine called censavudine for up to 48 weeks, which is about 11 months. During this time, you will have regular visits to the clinic.

These visits will involve several assessments to check your health and how the medicine is working. You can expect blood tests to measure the inflammation markers and checks for any side effects. You might also have brain scans (MRIs), and some samples of your spinal fluid might be taken to measure levels of the medicine and other markers.

They will also do various tests to check your movement, brain function, and overall development. This could involve questionnaires for you and your caregivers, as well as specific tests designed to assess how you are doing. The doctors will explain all these tests in detail.

Potential risks and benefits

Taking part in a study like this means you might be among the first to try a new medicine that could potentially help reduce the inflammation linked to AGS. However, as with any new medicine, there's no guarantee it will work for everyone, and it might not improve your symptoms. There's also a chance of experiencing side effects, which could be mild or more serious, even though the study closely monitors for them. Your safety and well-being are the top priority, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (2)

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

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Unverified
France
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Unverified
Italy

Common questions

What is Aicardi-Goutières Syndrome (AGS)?

AGS is a rare genetic condition that primarily affects the brain, causing inflammation and developmental problems.

What is censavudine?

Censavudine is a new medicine being tested in this study. It's hoped to reduce the inflammation associated with AGS.

How long will the study last for each person?

Participants will take the medicine and be monitored for up to 48 weeks, which is about 11 months.

What kind of tests will I have?

You'll have blood tests, possibly brain scans (MRIs), and tests to check your movement, brain function, and overall health.

Can I stop being part of the study if I want to?

Yes, you can leave the study at any time, for any reason, and your medical care will not be affected.