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

NCT06878365 | Active Not Recruiting Phase 1 FDA Drug

• 3 other identifiers: 1509-00032024-514942-35-00U1111-1309-4909
• Study Type: interventional
• Target: 16
• Locations: 9 countries
• Sites: 20

Brief Summary

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.

Conditions

Type 1 Interferonopathies

Outcome Measures

Primary Outcomes (1)

• Occurrence of any treatment-emergent adverse events assessed as related to study drug

  Approximately 72 weeks

Secondary Outcomes (6)

• 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

• 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

Study Arms (1)

BI 3000202

EXPERIMENTAL

Drug: BI 3000202_low dose; Drug: BI 3000202_high dose

Interventions

BI 3000202_low dose DRUG

BI 3000202\_low dose

BI 3000202

BI 3000202_high dose DRUG

BI 3000202\_high dose

BI 3000202

Eligibility Criteria

• Age: 18 Years - 74 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Boehringer Ingelheim: lead

Study Sites (20)

University of California San Francisco

San Francisco, California, 94143, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Universitair Ziekenhuis Gent

Ghent, 9000, Belgium

Location

Hôpital Gui de Chauliac

Montpellier, 34295, France

Location

Hôpital Necker

Paris, 75015, France

Location

HOP Tenon

Paris, 75020, France

Location

Universitätsklinikum Carl Gustav Carus Dresden

Dresden, 01307, Germany

Location

Medizinische Hochschule Hannover

Hanover, 30625, Germany

Location

Universitätsklinikum Tübingen

Tübingen, 72076, Germany

Location

Barzilai Medical Center

Ashkelon, 7830604, Israel

Location

ASST degli Spedali Civili di Brescia

Brescia, 25123, Italy

Location

Azienda Sanitaria Universitaria Giuliano Isontina

Trieste, 34124, Italy

Location

ULS de Santa Maria, E.P.E

Lisbon, 1649-035, Portugal

Location

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

Porto, 4099-001, Portugal

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Virgen del Rocío

Seville, 41013, Spain

Location

Royal Infirmary of Edinburgh

Edinburgh, EH16 4SA, United Kingdom

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Related Links

• Related Info

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 13, 2025
• First Posted: March 17, 2025
• Study Start: July 29, 2025
• Primary Completion (Estimated): December 6, 2026
• Study Completion (Estimated): December 7, 2026
• Last Updated: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• 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.
• 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.

Locations

ES (3); DE (3); FR (3); IL (1); GB (2); PT (2); US (3); BE (1); IT (2)