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 Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
BI 3000202\_low dose
BI 3000202\_high dose
University of California San Francisco
San Francisco, California, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
Universitair Ziekenhuis Gent
Ghent, Belgium
Hôpital Gui de Chauliac
Montpellier, France
Hôpital Necker
Paris, France
HOP Tenon
Paris, France
Universitätsklinikum Carl Gustav Carus Dresden
Dresden, Germany
Medizinische Hochschule Hannover
Hanover, Germany
Universitätsklinikum Tübingen
Tübingen, Germany
...and 10 more locations
Occurrence of any treatment-emergent adverse events assessed as related to study drug
Time frame: Approximately 72 weeks
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)
Time frame: At Day 1
Maximum measured concentration of BI 3000202 in plasma after administration of the first dose (Cmax)
Time frame: 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)
Time frame: At Days 29 and 85
Maximum measured concentration of BI 3000202 in plasma at steady state (Cmax,ss)
Time frame: At Days 29 and 85
Predose concentration of BI 3000202 in plasma at steady state immediately before administration of the next dose (Cpre,ss)
Time frame: At Days 29 and 85
Change from baseline in interferon gene score (IGS)
Time frame: At baseline, at Week 12
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