En undersøgelse for at teste, hvor godt BI 3000202 tolereres af mennesker med type 1 -interferonopatier
Open-label-forsøg med enkeltarm for at vurdere sikkerhed, tolerabilitet, farmakokinetik og farmakodynamik af BI 3000202 hos voksne patienter med udvalgte type 1-interferonopatier
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.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Undersøgelsestype
Tilmelding (Faktiske)
Fase
- Fase 1
Kontakter og lokationer
Studiesteder
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Ghent, Belgien, 9000
- Universitair Ziekenhuis Gent
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Edinburgh, Det Forenede Kongerige, EH16 4SA
- Royal Infirmary of Edinburgh
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London, Det Forenede Kongerige, NW3 2QG
- Royal Free Hospital
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California
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San Francisco, California, Forenede Stater, 94143
- University of California San Francisco
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Pennsylvania
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Philadelphia, Pennsylvania, Forenede Stater, 19104
- Children's Hospital of Philadelphia
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Texas
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Houston, Texas, Forenede Stater, 77030
- Texas Children's Hospital
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Montpellier, Frankrig, 34295
- Hôpital Gui de Chauliac
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Paris, Frankrig, 75015
- Hopital Necker
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Paris, Frankrig, 75020
- HOP Tenon
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Ashkelon, Israel, 7830604
- Barzilai Medical Center
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Brescia, Italien, 25123
- ASST degli Spedali Civili di Brescia
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Trieste, Italien, 34124
- Azienda Sanitaria Universitaria Giuliano Isontina
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Lisbon, Portugal, 1649-035
- ULS de Santa Maria, E.P.E - Hospital de Santa Maria
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Porto, Portugal, 4099-001
- ULS de Santo Antônio, E.P.E - Centro Hospitalar Universitário de Santo António
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Barcelona, Spanien, 08035
- Hospital Universitari Vall d'Hebron
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Madrid, Spanien, 28046
- Hospital Universitario La Paz
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Seville, Spanien, 41013
- Hospital Universitario Virgen del Rocio
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Dresden, Tyskland, 01307
- Universitätsklinikum Carl Gustav Carus Dresden
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Hanover, Tyskland, 30625
- Medizinische Hochschule Hannover
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Tübingen, Tyskland, 72076
- Universitätsklinikum Tübingen
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Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
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Male and female adult patients from ≥18 years (or alternative age for adults based on local regulations) to <75 years.
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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.
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Patients may be either:
- On standard of care, provided it is on stable doses
- Not on standard of care
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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 Criteria:
- 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.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
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Eksperimentel: BI 3000202
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BI 3000202_low dose BI 3000202_high dose |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Tidsramme |
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Forekomst af eventuelle bivirkninger af behandlingsfremstilling vurderet som relateret til undersøgelsesmedicin
Tidsramme: Cirka 72 uger
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Cirka 72 uger
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Sekundære resultatmål
Resultatmål |
Tidsramme |
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Maksimal målt koncentration af BI 3000202 i plasma ved stabil tilstand (Cmax, SS)
Tidsramme: På dag 29 og 85
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På dag 29 og 85
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Predose -koncentration af BI 3000202 i plasma ved stabil tilstand umiddelbart før administration af den næste dosis (CPRE, SS)
Tidsramme: På dag 29 og 85
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På dag 29 og 85
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Skift fra baseline i Interferon Gene Score (IGS)
Tidsramme: Ved baseline, i uge 12
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Ved baseline, i uge 12
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Område under koncentrationstidskurven for BI 3000202 i plasma fra tid 0 til 4 timer efter administration af den første dosis (AUC0-4)
Tidsramme: På dag 1
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På dag 1
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Maksimal målt koncentration af BI 3000202 i plasma efter administration af den første dosis (Cmax)
Tidsramme: På dag 1
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På dag 1
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Område under koncentrationstidskurven for BI 3000202 i plasma fra tid 0 til 4 timer i Steady State (AUC0-4, SS)
Tidsramme: På dag 29 og 85
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På dag 29 og 85
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Samarbejdspartnere og efterforskere
Sponsor
Publikationer og nyttige links
Hjælpsomme links
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Andre undersøgelses-id-numre
- 1509-0003
- 2024-514942-35-00 (Registry Identifier: CTIS)
- U1111-1309-4909 (Anden identifikator: WHO International Clinical Trials Registry Platform (ICTRP))
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
IPD-planbeskrivelse
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.
IPD-delingstidsramme
After structured results have been posted, all regulatory activities are completed in the US and EU for the product and indication, and after the primary manuscript has been accepted for publication.
IPD-delingsadgangskriterier
For study documents - upon signing of a 'Document Sharing Agreement'. For study data - 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement.
IPD-deling Understøttende informationstype
- STUDY_PROTOCOL
- SAP
- CSR
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
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