BI 3000202 for Aicardi-Goutières Syndrome

Recruiting at 17 trial locations

Overseen By Boehringer Ingelheim

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Boehringer Ingelheim

Disqualifiers: Chronic inflammatory disease, Kidney impairment, Hepatic impairment, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 9

18 Participants Needed

Trial Summary

What is the purpose of this trial?

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 8 weeks. The participants may continue their regular treatment for their condition during the study. Participants are in the study for about 6 months. During this time, they visit the study site 9 times. The doctors check the health of the participants and note any health problems that could have been caused by BI 3000202.

Will I have to stop taking my current medications?

You can continue your regular treatment for your condition during the study, as long as the doses are stable.

Eligibility Criteria

Adults with type 1 interferonopathies like Aicardi-Goutières syndrome, COPA syndrome, Familial chilblain lupus, or those with specific gene mutations can join. They must be able to take BI 3000202 tablets and continue their regular treatments.

Inclusion Criteria

My genetic test results are already in my medical records.

Women of childbearing potential must be ready and able to use highly effective methods of birth control

My genetic test shows a harmful mutation.

Exclusion Criteria

Increased risk of infectious complications based on investigator's judgement

Further exclusion criteria apply

AGS Severity Scale >3 for patients diagnosed with Aicardi-Goutières syndrome or other interferonopathy with neurological involvement

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment - Lower Dose

Participants take a lower dose of BI 3000202 as tablets

4 weeks

4 visits (in-person)

Treatment - Higher Dose

Participants take a higher dose of BI 3000202 as tablets

8 weeks

5 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• BI 3000202

Trial Overview The trial tests the tolerability of a drug called BI 3000202 at two different doses in people with certain autoimmune disorders. Participants first take a low dose for 4 weeks followed by a high dose for another 8 weeks.

Participant Groups

Group I: BI 3000202 Experimental Treatment 2 Interventions

Who Is Running the Clinical Trial?

Boehringer Ingelheim

Lead Sponsor

Trials

2,566

Recruited

16,150,000+