Type 1 InterferonopathiesPhase 1NCT06878365
A Study to Test How Well BI 3000202 is Tolerated by People With Type 1 Interferonopathies
A Phase 1 study testing BI 3000202_low dose and BI 3000202_high dose in people with Type 1 Interferonopathies, run by Boehringer Ingelheim at 20 sites in 9 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 74 years
- Conditions the study is for
- Type 1 Interferonopathies
- What is being tested?
- BI 3000202_low dose and BI 3000202_high dose
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 20 sitesin 9 countries
- Study dates
- 29 Jul 2025to 7 Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 74 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
20 sites in 9 countries.
20 sites in 9 countries
United States3 sites
Texas Children's Hospital
Houston, Texas, 77030
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of California San Francisco
San Francisco, California, 94143
France3 sites
Hôpital Gui de Chauliac
Montpellier, 34295
Hopital Necker
Paris, 75015
HOP Tenon
Paris, 75020
Germany3 sites
Universitätsklinikum Carl Gustav Carus Dresden
Dresden, 01307
Medizinische Hochschule Hannover
Hanover, 30625
Universitätsklinikum Tübingen
Tübingen, 72076
Spain3 sites
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Hospital Universitario La Paz
Madrid, 28046
Hospital Virgen del Rocio
Seville, 41013
Italy2 sites
ASST degli Spedali Civili di Brescia
Brescia, 25123
Azienda Sanitaria Universitaria Giuliano Isontina
Trieste, 34124
Portugal2 sites
ULS de Santa Maria, E.P.E
Lisbon, 1649-035
ULS de Santo Antônio, E.P.E - Centro Hospitalar Universitário de Santo António
Porto, 4099-001
United Kingdom2 sites
Royal Infirmary of Edinburgh
Edinburgh, EH16 4SA
Royal Free Hospital
London, NW3 2QG
About this study
Official title: Single-arm Open-label Trial to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BI 3000202 in Adult Patients With Selected Type 1 Interferonopathies
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.
- Conditions
- Type 1 Interferonopathies
- Being tested
- BI 3000202_low dose, BI 3000202_high dose
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BI 3000202_low doseDrug
BI 3000202_low dose
BI 3000202_high doseDrug
BI 3000202_high dose
| Study group | Role | What they receive |
|---|---|---|
| BI 3000202 | Experimental | Not described Receives: Drug: BI 3000202_low dose; Drug: BI 3000202_high dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of any treatment-emergent adverse events assessed as related to study drug
Measured: Approximately 72 weeks
Secondary outcomes (6)
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)
Measured: At Day 1
Maximum measured concentration of BI 3000202 in plasma after administration of the first dose (Cmax)
Measured: 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)
Measured: At Days 29 and 85
Maximum measured concentration of BI 3000202 in plasma at steady state (Cmax,ss)
Measured: At Days 29 and 85
Predose concentration of BI 3000202 in plasma at steady state immediately before administration of the next dose (Cpre,ss)
Measured: At Days 29 and 85
Change from baseline in interferon gene score (IGS)
Measured: At baseline, at Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2025
- Study start29 Jul 2025
- Primary completion6 Dec 2026 (estimated)
- Study completion7 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Type 1 InterferonopathiesStudies of the same condition, open and under way.
- More trials run by Boehringer IngelheimThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boehringer Ingelheim
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06878365
- Sponsor's protocol ID
- 1509-0003
- Other IDs
- 2024-514942-35-00, U1111-1309-4909
- Status last verified
- Oct 2026
- First submitted
- 13 Mar 2025
- Last updated
- 14 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.clinicalstudies.boehringer-ingelheim.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement". Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single-arm Open-label Trial to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BI 3000202 in Adult Patients With Selected Type 1 Interferonopathies
- ClinicalTrials.gov ID
- NCT06878365
- Acronym / sponsor ID
- 1509-0003
- Conditions
- Type 1 Interferonopathies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Boehringer IngelheimIndustry (pharma, biotech, devices)
- Study start
- 29 Jul 2025 (actual)
- Primary completion
- 6 Dec 2026 (estimated)
- Study completion
- 7 Dec 2026 (estimated)
- First posted
- 17 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BI 3000202 | Experimental | Drug: BI 3000202_low dose, Drug: BI 3000202_high dose |
Who may qualify
- Ages
- 18 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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:
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BI 3000202_low doseBI 3000202_low dose | Drug | BI 3000202 |
| BI 3000202_high doseBI 3000202_high dose | Drug | BI 3000202 |
Primary outcome
Occurrence of any treatment-emergent adverse events assessed as related to study drug
Time frame: Approximately 72 weeks
6 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.clinicalstudies.boehringer-ingelheim.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement". Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BI 3000202_low dose and BI 3000202_high dose in people with Type 1 Interferonopathies, run by Boehringer Ingelheim at 20 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, France, Germany, Spain, Italy and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurrence of any treatment-emergent adverse events assessed as related to study drug (time frame: Approximately 72 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.