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Clinical Trials in the UK / NCT06878365
Active, not recruiting Phase 1

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

NCT06878365 · tracked via the Priya Life Science UK tracker
Sponsor
Boehringer Ingelheim
Phase
Phase 1
Started
2025-07-29
Last updated
2026-09-15

Condition(s) studied

Type 1 Interferonopathies

Investigational drug(s) / intervention(s)

BI 3000202_low doseBI 3000202_high dose

BI 3000202_low dose: BI 3000202\_low dose

BI 3000202_high dose: BI 3000202\_high dose

Study 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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
74 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (20)

FacilityCityRegionStatus
University of California San Francisco San Francisco California
Children's Hospital of Philadelphia Philadelphia Pennsylvania
Texas Children's Hospital Houston Texas
Universitair Ziekenhuis Gent Ghent Belgium
Hôpital Gui de Chauliac Montpellier France
Hopital Necker Paris France
HOP Tenon Paris France
Universitätsklinikum Carl Gustav Carus Dresden Dresden Germany
Medizinische Hochschule Hannover Hanover Germany
Universitätsklinikum Tübingen Tübingen Germany
Barzilai Medical Center Ashkelon Israel
ASST degli Spedali Civili di Brescia Brescia Italy
Azienda Sanitaria Universitaria Giuliano Isontina Trieste Italy
ULS de Santa Maria, E.P.E Lisbon Portugal
ULS de Santo Antônio, E.P.E - Centro Hospitalar Universitário de Santo António Porto Portugal
Hospital Universitari Vall d'Hebron Barcelona Spain
Hospital Universitario La Paz Madrid Spain
Hospital Virgen del Rocio Seville Spain
Royal Infirmary of Edinburgh Edinburgh United Kingdom
Royal Free Hospital London United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06878365 on ClinicalTrials.gov ↗ ← All trials in the UK