BI 3000202 for Aicardi-Goutières Syndrome

Recruiting at 26 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called BI 3000202, targeting conditions where the immune system attacks the body due to specific gene mutations. These conditions include Aicardi-Goutières syndrome, Coatomer subunit alpha (COPA) syndrome, and Familial chilblain lupus. The research aims to determine how well participants tolerate the treatment over approximately nine months. Participants must have one of these conditions confirmed by their medical history and may continue any regular treatment they are already receiving. As a Phase 1 trial, this research seeks to understand how the treatment works in people, offering participants the opportunity to be among the first to receive it.

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.

Is there any evidence suggesting that BI 3000202 is likely to be safe for humans?

Research is examining how well BI 3000202 is tolerated by individuals with rare conditions called type 1 interferonopathies, such as Aicardi-Goutières syndrome (AGS). The primary goal is to assess the treatment's safety. Researchers are closely monitoring for any side effects or health issues linked to BI 3000202.

As a Phase 1 study, it is in the early stages of research. Phase 1 studies typically involve small groups and aim to determine a treatment's safety. There is no detailed safety data from previous studies of BI 3000202. However, testing in humans suggests that earlier lab and animal tests did not reveal serious safety concerns.

Participants will be monitored closely, with doctors tracking any health issues that arise during the study. This careful monitoring is standard in early trials to ensure participant safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Aicardi-Goutières syndrome?

Researchers are excited about BI 3000202 for Aicardi-Goutières Syndrome because it offers a novel approach compared to existing treatments. Most current options for this condition focus on managing symptoms rather than addressing the underlying cause. BI 3000202 is different as it targets the root mechanisms at a molecular level, potentially altering the disease's progression. This could lead to more effective management and improved quality of life for patients with this rare genetic disorder.

What evidence suggests that BI 3000202 might be an effective treatment for Aicardi-Goutières syndrome?

Research has shown that BI 3000202 is designed to help with type 1 interferonopathies, such as Aicardi-Goutières syndrome (AGS), where the immune system causes inflammation. The treatment aims to reduce this inflammation by targeting specific parts of the immune system. While information on BI 3000202's effectiveness in humans is limited, the treatment is based on a strong understanding of the disease. This trial investigates how well people can tolerate BI 3000202, an important first step in determining its potential benefits. ^{3 4 6 7 8}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06878365

A Study to Test How Well BI 3000202 is Tolerated by ...

This study is open to adults with selected type 1 interferonopathies. People can join the study if they have Aicardi-Goutières syndrome (AGS), ...

2.

withpower.com

withpower.com/trial/phase-1-type-1-interferonopathies-2-2025-60b73

BI 3000202 for Aicardi-Goutières Syndrome

This Phase 1 medical study run by Boehringer Ingelheim is evaluating whether BI 3000202 will have tolerable side effects & efficacy for patients with ...

3.

clinicaltrials.ucsf.edu

clinicaltrials.ucsf.edu/trial/NCT06878365

Test How Well BI 3000202 is Tolerated by People With ...

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 ...

4.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC7617231/

Reverse transcriptase inhibitors in Aicardi–Goutières syndrome

There is insufficient evidence that ABC or 3TC is either effective or ineffective in reducing type I IFN signalling in AGS over 6 weeks. The ...

5.

inflammation-research.ed.ac.uk

inflammation-research.ed.ac.uk/child-life-and-health/people/principal-investigators/dr-jay-shetty

Dr Jayakara (Jay) Shetty | Child Life and Health

A Study to Test How Well BI 3000202 is Tolerated by People With ... Inhibition of Reverse Transcription in Aicardi-Goutières Syndrome (AGS ...

6.

ninds.nih.gov

ninds.nih.gov/health-information/disorders/aicardi-goutieres-syndrome

Aicardi-Goutières Syndrome

Aicardi-Goutières syndrome (AGS) is a rare inherited disease that affects the brain, immune system, and the skin.

7.

orpha.net

orpha.net/en/institutions/professional/634745

Pr Marie-Louise FRÉMOND

Single-arm Open-label Trial to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BI 3000202 in Adult Patients With Selected Type 1 ...

8.

medlineplus.gov

medlineplus.gov/genetics/condition/aicardi-goutieres-syndrome/

Aicardi-Goutières syndrome - Genetics

Aicardi-Goutières syndrome is a disorder with variable signs and symptoms, but it primarily affects the brain, the immune system, and the skin.