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

Single-arm Open-label Trial to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BI 3000202 in Adult Patients With Selected Type 1 Interferonopathies

This study is open to adults with selected type 1 interferonopathies. People can join the study if they have Aicardi-Goutières syndrome (AGS), Coatomer subunit alpha (COPA) syndrome, Familial chilblain lupus (FCL), or another type 1 interferonopathy with a specific gene mutation.

The purpose of this study is to find out how BI 3000202 is tolerated in people with selected type 1 interferonopathies. Participants take a lower dose of BI 3000202 as tablets for 4 weeks. Afterwards, they take a higher dose of BI 3000202 as tablets for 36 weeks. They may continue with the study treatment until every participant has completed 40 weeks of treatment (about 9 months). The participants may also continue their regular treatment for their condition during the study.

During this study, participants visit the study site 13 times or more, depending on when they start their participation. The doctors check the health of the participants and note any health problems that could have been caused by BI 3000202.

Study Overview

• Status: Active, not recruiting
• Conditions: Type 1 Interferonopathies
• Intervention / Treatment: Drug: BI 3000202_low dose; Drug: BI 3000202_high dose

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 1

Contacts and Locations

Study Locations

• Belgium
  • Ghent, Belgium, 9000
    • Universitair Ziekenhuis Gent
• France
  • Montpellier, France, 34295
    • Hopital Gui de Chauliac
  • Paris, France, 75015
    • Hôpital Necker
  • Paris, France, 75020
    • HOP Tenon
• Germany
  • Dresden, Germany, 01307
    • Universitatsklinikum Carl Gustav Carus Dresden
  • Hanover, Germany, 30625
    • Medizinische Hochschule Hannover
  • Tübingen, Germany, 72076
    • Universitätsklinikum Tübingen
• Israel
  • Ashkelon, Israel, 7830604
    • Barzilai Medical Center
• Italy
  • Brescia, Italy, 25123
    • Asst Degli Spedali Civili Di Brescia
  • Trieste, Italy, 34124
    • Azienda Sanitaria Universitaria Giuliano Isontina
• Portugal
  • Lisbon, Portugal, 1649-035
    • ULS de Santa Maria, E.P.E
  • Porto, Portugal, 4099-001
    • ULS de Santo Antônio, E.P.E - Centro Hospitalar Universitário de Santo António
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d'Hebron
  • Madrid, Spain, 28046
    • Hospital Universitario La Paz
  • Seville, Spain, 41013
    • Hospital Virgen Del Rocio
• United Kingdom
  • Edinburgh, United Kingdom, EH16 4SA
    • Royal Infirmary of Edinburgh
  • London, United Kingdom, NW3 2QG
    • Royal Free Hospital
• United States
  • California
    • San Francisco, California, United States, 94143
      • University of California San Francisco
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Texas
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BI 3000202	Drug: BI 3000202_low dose; BI 3000202_low dose; Drug: BI 3000202_high dose; BI 3000202_high dose

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Occurrence of any treatment-emergent adverse events assessed as related to study drug	Approximately 72 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Maximum measured concentration of BI 3000202 in plasma at steady state (Cmax,ss)	At Days 29 and 85
Predose concentration of BI 3000202 in plasma at steady state immediately before administration of the next dose (Cpre,ss)	At Days 29 and 85
Change from baseline in interferon gene score (IGS)	At baseline, at Week 12
Area under the concentration-time curve of BI 3000202 in plasma from time 0 to 4 hours after administration of the first dose ( AUC0-4)	At Day 1
Maximum measured concentration of BI 3000202 in plasma after administration of the first dose (Cmax)	At Day 1
Area under the concentration-time curve of BI 3000202 in plasma from time 0 to 4 hours at steady state (AUC0-4,ss)	At Days 29 and 85

Collaborators and Investigators

• Sponsor: Boehringer Ingelheim

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): July 29, 2025
• Primary Completion (Estimated): December 6, 2026
• Study Completion (Estimated): December 7, 2026

Study Registration Dates

• First Submitted: March 13, 2025
• First Submitted That Met QC Criteria: March 13, 2025
• First Posted (Actual): March 17, 2025

Study Record Updates

• Last Update Posted (Actual): July 7, 2026
• Last Update Submitted That Met QC Criteria: July 6, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Other Study ID Numbers: 1509-0003; 2024-514942-35-00 (Registry Identifier: CTIS); U1111-1309-4909 (Other Identifier: WHO International Clinical Trials Registry Platform (ICTRP))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.clinicalstudies.boehringer-ingelheim.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement".

Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.

• IPD Sharing Time Frame: After structured results have been posted, all regulatory activities are completed in the US and EU for the product and indication, and after the primary manuscript has been accepted for publication.
• IPD Sharing Access Criteria: For study documents - upon signing of a 'Document Sharing Agreement'. For study data - 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No