New pill aims to tame rare immune diseases

NCT ID NCT06878365

First seen Jun 27, 2026 · Last updated Jul 08, 2026 · Updated 2 times

Summary

This early-stage trial tests an experimental drug called BI 3000202 in 16 adults with rare type 1 interferonopathies, such as Aicardi-Goutières syndrome. Participants take a low dose for 4 weeks, then a higher dose for 36 weeks. The main goal is to see if the drug is safe and how the body processes it.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
BI 3000202 (an experimental drug taken as tablets)
What this could lead to
If it works, this could point toward a treatment that helps control type 1 interferonopathies, rare immune disorders.
What could go wrong
This is a very early, small Phase 1 trial with only 16 participants. It primarily tests safety, not effectiveness, so it may not lead to a working treatment.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • ASST degli Spedali Civili di Brescia

    Brescia, 25123, Italy

  • Azienda Sanitaria Universitaria Giuliano Isontina

    Trieste, 34124, Italy

  • Barzilai Medical Center

    Ashkelon, 7830604, Israel

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • HOP Tenon

    Paris, 75020, France

  • Hopital Necker

    Paris, 75015, France

  • Hospital Universitari Vall d'Hebron

    Barcelona, 08035, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Virgen del Rocío

    Seville, 41013, Spain

  • Hôpital Gui de Chauliac

    Montpellier, 34295, France

  • Medizinische Hochschule Hannover

    Hanover, 30625, Germany

  • Royal Free Hospital

    London, NW3 2QG, United Kingdom

  • Royal Infirmary of Edinburgh

    Edinburgh, EH16 4SA, United Kingdom

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • ULS de Santa Maria, E.P.E

    Lisbon, 1649-035, Portugal

  • ULS de Santo Antônio, E.P.E - Centro Hospitalar Universitário de Santo António

    Porto, 4099-001, Portugal

  • Universitair Ziekenhuis Gent

    Ghent, 9000, Belgium

  • University of California San Francisco

    San Francisco, California, 94143, United States

  • Universitätsklinikum Carl Gustav Carus Dresden

    Dresden, 01307, Germany

  • Universitätsklinikum Tübingen

    Tübingen, 72076, Germany

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