Ex Vivo Evaluation of JAK-inhibitor and Gene Therapeutical Approach in JAK-STAT Related Disorders
NCT ID: NCT07261891
Last Updated: 2025-12-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
NA
20 participants
INTERVENTIONAL
2024-11-21
2030-12-01
Brief Summary
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Detailed Description
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* To study pSTAT, transcriptional profile and cytokine production on bulk and sorted peripheral blood cell populations following stimulation in the presence or absence of different jakinibs
* To evaluate to what extent jakinibs can normalize the transcriptional in different cell types (or not and identify blind spots of this treatment strategy)
* To evaluate ex vivo the impact of a gene therapeutic approach for STAT1 GOF.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Patients with a monogenic IEI with an hyperactive JAK-STAT pathway
Adult patients presenting with a genetically confirmed or highly suspected disorder leading to an exagerated JAK-STAT pathway.
blood sampling
Blood/serum samples will be collected during routine clinical visits at the time of planned peripheral venous blood sampling. Samples will be processed and either used immediately (flow cytometry-based cell sorting, gDNA extraction, in vitro functional assays, primary cell culture or single-cell applications) or stored for later analysis.
Healthy controls
Healthy controls (without immune-mediated disease)
blood sampling
Blood/serum samples will be collected during routine clinical visits at the time of planned peripheral venous blood sampling. Samples will be processed and either used immediately (flow cytometry-based cell sorting, gDNA extraction, in vitro functional assays, primary cell culture or single-cell applications) or stored for later analysis.
Interventions
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blood sampling
Blood/serum samples will be collected during routine clinical visits at the time of planned peripheral venous blood sampling. Samples will be processed and either used immediately (flow cytometry-based cell sorting, gDNA extraction, in vitro functional assays, primary cell culture or single-cell applications) or stored for later analysis.
Eligibility Criteria
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Inclusion Criteria
* Controls (B): participants eligible for inclusion in this study must fall in one of the following categories:
* Healthy controls (without immune-mediated disease)
Exclusion Criteria
* Persons unable or unwilling to give informed consent
18 Years
ALL
Yes
Sponsors
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Research Foundation - Flanders (Fonds Wetenschappelijk Onderzoek)
OTHER
prof. dr. Rik Schrijvers
OTHER
Responsible Party
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prof. dr. Rik Schrijvers
Prof. dr.
Principal Investigators
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Rik Schrijvers, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
UZ Leuven
Locations
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University Hospitals Leuven,
Leuven, Vlaams-Brabant, Belgium
Countries
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Central Contacts
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Facility Contacts
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References
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Staels F, Roosens W, Giovannozzi S, Moens L, Bogaert J, Iglesias-Herrero C, Gijsbers R, Bossuyt X, Frans G, Liston A, Humblet-Baron S, Meyts I, Van Aelst L, Schrijvers R. Case report: Myocarditis in congenital STAT1 gain-of function. Front Immunol. 2023 Mar 20;14:1095595. doi: 10.3389/fimmu.2023.1095595. eCollection 2023.
Giovannozzi S, Demeulemeester J, Schrijvers R, Gijsbers R. Transcriptional Profiling of STAT1 Gain-of-Function Reveals Common and Mutation-Specific Fingerprints. Front Immunol. 2021 Feb 17;12:632997. doi: 10.3389/fimmu.2021.632997. eCollection 2021.
Giovannozzi S, Lemmens V, Hendrix J, Gijsbers R, Schrijvers R. Live Cell Imaging Demonstrates Multiple Routes Toward a STAT1 Gain-of-Function Phenotype. Front Immunol. 2020 Jun 9;11:1114. doi: 10.3389/fimmu.2020.01114. eCollection 2020.
Other Identifiers
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G054022N
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
S69751
Identifier Type: -
Identifier Source: org_study_id
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