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New pill aims to tame rare immune diseases

NCT ID NCT06878365

Disease control Sponsor: Boehringer Ingelheim Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

16 people

The number who actually took part.

Started

Jul 2025

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 74 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Male and female adult patients from ≥18 years (or alternative age for adults based on local regulations) to \<75 years. * Genetic diagnosis with mutations in the following affected genes: three prime repair exonuclease 1 (TREX1), ribonuclease H2 subunit A, B or C (RNASEH2B, RNASEH2C, RNASEH2A), SAM And HD domain containing deoxynucleoside triphosphate triphosphohydrolase 1 (SAMHD1), U7 Small Nuclear RNA Associated sm-like protein (LSM11), RNA component of the U7 snRNP (RNU7-1) for AGS; Coatomer subunit alpha (COPA) for COPA syndrome; TREX1, SAM And HD domain containing deoxynucleoside triphosphate triphosphohydrolase 1 (SAMHD1) for Familial chilblain lupus (FCL); DNA nuclease 2 (DNASE2), Adenosine triphosphate synthase family AAA domain containing 3A (ATAD3A) for other type 1 interferonopathies. Genotype documented in medical history is sufficient for eligibility determination and does not require confirmation. Variant identification as "pathogenic" or "likely pathogenic" is preferred according to a joint consensus recommendation of the American College of Medical Genetics and Genomics and the Association for Molecular Pathology. In the absence of such identification, clinical assessment of pathogenicity is required to be documented in the medical records. * Patients may be either: * On standard of care, provided it is on stable doses * Not on standard of care * If women of childbearing potential (WOCBP): must be ready and able to use highly effective methods of birth control. Non-vasectomised male trial participants whose sexual partner is a woman of childbearing potential must be ready and able to use male contraception. Exclusion Criteria: * Major chronic inflammatory or connective tissue disease other than selected type 1 interferonopathies, as assessed by the investigator. * Increased risk of infectious complications based on investigator's judgement. * Evidence of potential moderate to severe loss of kidney function. * Evidence of hepatic impairment. * Further exclusion criteria apply.

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Conditions

The condition(s) this trial relates to.

Aicardi-Goutieres syndrome autoimmune interstitial lung disease-arthritis syndrome familial chilblain lupus type 1 interferonopathy

As listed by the trial registrant

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

Type 1 interferonopathies

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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