Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

An Open-Label Extension Study of EryDex in Patients with Ataxia-Telangiectasia Following Participation in Study IEDAT-04-2022 (NEAT)

This study is an extension of a previous trial for people with Ataxia-Telangiectasia (A-T) who have already received an experimental medicine called EryDex. It’s an 'open-label' study, meaning everyone involved knows they are receiving the medicine. The main goal is to carefully check the long-term safety of EryDex. Researchers will be looking for any side effects, changes in body measurements like blood pressure and lab results, and how participants generally feel. They will also assess important body functions such as hormone levels, immune cells, bone strength, and overall growth and development. This will help us understand more about how EryDex affects people over time.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Quince Therapeutics S.p.A.

Enrolment target

Start

23 Oct 2024

Ataxia Telangiectasia (A-T)

What is this study about?

This research is an extension of a previous study for people living with a rare genetic condition called Ataxia-Telangiectasia, often shortened to A-T. A-T can cause problems with movement, balance, and the immune system. In the previous study, participants received a new experimental medicine called EryDex.

This current study, known as an 'open-label extension', means that all participants will continue to receive EryDex, and everyone involved (researchers, doctors, and participants) will know they are receiving the treatment. The main purpose is to carefully monitor the safety of EryDex over a longer period. This is a very important step in developing new treatments, as it helps doctors understand how a medicine affects people over time, not just for a short period.

Researchers will be looking closely at any side effects that might happen. They will also check a range of health markers, including blood tests, physical examinations, and how the medicine might affect things like hormone levels, bone health, and how a person grows and develops. This detailed monitoring helps to build a complete picture of the medicine's long-term effects.

Key takeaways

This study is for people who have already been in the NEAT study for A-T.
It's an extension, meaning participants continue with treatment and long-term monitoring.
The main focus is to understand the long-term safety of EryDex.
Researchers will check for side effects and how the medicine affects your body over time.
It's an 'open-label' study, so everyone knows they are receiving the treatment.

Who may be eligible?

To be considered for this study, you must have previously taken part in the earlier EryDex study (called IEDAT-04-2022 or NEAT) for Ataxia-Telangiectasia. This means that if you haven't been in that specific study before, you won't be able to join this extension study.

Participants must be 18 years old or older. There is no upper age limit for joining this study. Both men and women are welcome to participate.

Quick self-check

Have I previously participated in the NEAT study (IEDAT-04-2022) for Ataxia-Telangiectasia?
Am I 18 years old or older?
Do I have Ataxia-Telangiectasia?
Am I able to attend regular study appointments?

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

What does participation involve?

If you join this study, you will continue to receive the experimental medicine EryDex. Researchers will regularly check your health and look for any side effects. This will involve regular visits where they might take blood samples, check your vital signs (like blood pressure), and perform physical and neurological examinations. They will also assess things like your balance and coordination using specific scales. Additionally, they will check your hormone levels, immune cells, and measure your bone density using a special X-ray called a DEXA scan. Your growth and development will also be monitored. The total length of your participation will be discussed with the study team.

Potential risks and benefits

Potential benefits of participating could include continued access to the experimental medicine EryDex, which may offer therapeutic effects for your Ataxia-Telangiectasia. You will also receive careful health monitoring and contribute valuable information that could help others with A-T in the future. However, as with any medicine, there are potential risks, including side effects from EryDex, some of which could be serious. Researchers will explain all known potential side effects. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (6)

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Denmark
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Norway
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Germany
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Italy
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Poland
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Spain

Common questions

What is EryDex?

EryDex is an experimental medicine being studied for Ataxia-Telangiectasia. It involves a medicine called dexamethasone packaged into your own red blood cells.

What does 'open-label' mean?

It means that both you and the study doctors will know you are receiving the active medicine (EryDex). No one will be receiving a placebo.

Will I have to pay to be in this study?

No, you will not have to pay to receive the study medicine or for the study-related appointments and tests.

How long will this study last for me?

The exact duration of your participation will be explained by the study team, as it's an extension of a previous study.

What kind of tests will I have?

You'll have regular physical and neurological exams, blood tests, measurements of vital signs, and scans to check bone density, among other assessments.