All studies
Active not recruitingPHASE1INTERVENTIONAL

A Study to Test How Well BI 3000202 is Tolerated by People With Type 1 Interferonopathies

This research study is looking at a new medicine called BI 3000202 for adults who have certain rare conditions known as Type 1 Interferonopathies. These include conditions like Aicardi-Goutières syndrome (AGS), COPA syndrome, or Familial chilblain lupus. The main aim is to find out how well people tolerate this new medicine. Participants will take a low dose of BI 3000202 tablets for four weeks, then a higher dose for 36 weeks. They may continue treatment for longer. Throughout the study, participants will have regular check-ups to monitor their health and any effects of the medicine, while potentially continuing their standard treatments.

At a glance

Status
Active not recruiting
Phase
PHASE1
Sponsor
Boehringer Ingelheim
Enrolment target
16
Start
29 Jul 2025
Estimated completion
07 Dec 2026

What is this study about?

This study is investigating a new medicine called BI 3000202 for adults living with certain rare conditions known as Type 1 Interferonopathies. These conditions, which include Aicardi-Goutières syndrome (AGS), COPA syndrome, or Familial chilblain lupus, stem from specific genetic changes within the body's immune system.

The main purpose of this research is to understand how well people tolerate BI 3000202. This means checking for any side effects or discomfort while taking the medicine. By carefully monitoring participants, the researchers hope to gather important information about its safety and how the body copes with different doses. Understanding how well a new medicine is tolerated is a crucial first step in its development.

Taking part in this study could help scientists learn more about Type 1 Interferonopathies and develop new ways to manage them in the future. It's a vital step in clinical research, aiming to improve future treatment options for people affected by these rare conditions.

Key takeaways

  • This study is for adults with specific rare conditions called Type 1 Interferonopathies.
  • It aims to see how well a new medicine, BI 3000202, is tolerated at different doses.
  • Participation involves taking tablets daily for several months and attending regular check-ups.
  • You might be able to continue your usual treatments during the study.
  • The study helps gather important information to potentially find new ways to manage these conditions.
  • You can withdraw from the study at any time.

Who may be eligible?

This study is designed for adults aged 18 to 74 years old. To join, you must have a confirmed genetic diagnosis of a Type 1 Interferonopathy, such as Aicardi-Goutières syndrome (AGS), COPA syndrome, or Familial chilblain lupus. Your doctor will need to confirm the specific genetic change related to your condition.

You can participate whether you are currently receiving standard treatment for your condition, as long as it's at a stable dose, or if you are not on specific treatment. If you are a woman who could become pregnant, you must agree to use highly effective birth control methods during the study. Similarly, men in the study whose partners could become pregnant must also use effective contraception.

However, you won't be able to join if you have a major ongoing inflammatory or connective tissue disease other than a Type 1 Interferonopathy, or if your doctor believes you have a high risk of infections. Also, if there are signs of moderate to severe kidney problems, you would not be eligible.

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.

  1. Are you an adult between 18 and 74 years old?
  2. Have you been diagnosed with a Type 1 Interferonopathy (like AGS, COPA, or Familial chilblain lupus) with a confirmed genetic change?
  3. Are you able to use effective birth control if you are a woman of childbearing potential, or a man whose partner could become pregnant?
  4. Do you not have another major inflammatory or connective tissue disease?
  5. Do you not have a high risk of infections or moderate to severe kidney problems?
Answer every question to see your result.

What does participation involve?

If you join the study, you'll start by taking a lower dose of BI 3000202 tablets every day for four weeks. After this, you'll switch to a higher dose of the tablets and continue taking them for an additional 36 weeks. You might continue receiving the study medicine for longer, until all participants have completed 40 weeks of treatment (which is about 9 months).

During the study, you will have regular check-ups at the study site. This will involve at least 13 visits, but potentially more, depending on when you start. These visits are important for the doctors to monitor your health, check how your body is responding to the medicine, and record any new symptoms or health problems you might experience. You may also continue your usual treatments for your condition alongside the study medicine.

Potential risks and benefits

Taking part in a clinical study can offer potential benefits, such as access to a new medicine that isn't widely available and close medical monitoring of your health. However, like all medicines, BI 3000202 may have side effects, and some effects might not be fully known yet. The study team will carefully monitor you for any potential risks or side effects throughout your participation. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (20)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • University of California San Francisco
    Verified postcode
    San Francisco, United States
  • Children's Hospital of Philadelphia
    Verified postcode
    Philadelphia, United States
  • Texas Children's Hospital
    Verified postcode
    Houston, United States
  • Universitair Ziekenhuis Gent
    Verified postcode
    Ghent, Belgium
  • Hôpital Gui de Chauliac
    Verified postcode
    Montpellier, France
  • Hôpital Necker
    Verified postcode
    Paris, France
  • HOP Tenon
    Verified postcode
    Paris, France
  • Universitätsklinikum Carl Gustav Carus Dresden
    Verified postcode
    Dresden, Germany
  • Medizinische Hochschule Hannover
    Verified postcode
    Hanover, Germany
  • Universitätsklinikum Tübingen
    Verified postcode
    Tübingen, Germany
  • Barzilai Medical Center
    Verified postcode
    Ashkelon, Israel
  • ASST degli Spedali Civili di Brescia
    Verified postcode
    Brescia, Italy

Common questions

What are Type 1 Interferonopathies?

These are a group of rare genetic conditions where the body's immune system is overactive in a specific way, leading to inflammation and various health problems.

What is BI 3000202?

BI 3000202 is the new medicine being tested in this study. Researchers want to see how well people tolerate it.

How long will I take the study medicine?

You will take a low dose for 4 weeks, then a higher dose for 36 weeks. You might continue for longer, up to around 9 months in total, depending on when you start.

Can I continue my current treatments?

Yes, in most cases, you can continue your regular treatments for your condition during the study, as long as they are at a stable dose.

What does 'tolerated' mean in this study?

It means the study is looking to see how well people cope with the new medicine, checking for any side effects or discomfort, and how safe it is for them.

How to find out more

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

Discussion

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