Status:
RECRUITING
A Study to Test How Well BI 3000202 is Tolerated by People With Type 1 Interferonopathies
Lead Sponsor:
Boehringer Ingelheim
Conditions:
Type 1 Interferonopathies
Eligibility:
All Genders
18-74 years
Phase:
PHASE1
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 chilblai...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
July 29 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 7 2026
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT06878365
Start Date
July 29 2025
End Date
December 7 2026
Last Update
January 9 2026
Active Locations (19)
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1
University of California San Francisco
San Francisco, California, United States, 94121
2
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
3
Texas Children's Hospital
Houston, Texas, United States, 77030
4
Universitair Ziekenhuis Gent
Ghent, Belgium, 9000