Ex vivo testing of JAK inhibitors and a gene-therapy approach for JAK-STAT related immune disorders

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

Researchers will take blood samples from adults with genetically confirmed or suspected JAK-STAT pathway disorders (including STAT1 gain-of-function) and from healthy controls and expose bulk and sorted immune cells ex vivo to different JAK inhibitors. They will measure pSTAT signaling, transcriptional profiles, and cytokine production after stimulation to see which cell types normalize and which remain refractory. The team will also test the technical feasibility of a gene-therapeutic correction strategy for STAT1 GOF in patient cells outside the body. Findings will be used to map treatment blind spots and inform whether specific JAK inhibitors or gene approaches warrant further clinical development.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Cases (A): adult patients presenting with a genetically confirmed or highly suspected disorder leading to an exagerated JAK-STAT pathway.
* 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:

* Children (\< 18 years at time of recruitment)
* Persons unable or unwilling to give informed consent

Where it's running

• University Hospitals Leuven,
  Leuven, Vlaams-Brabant, Belgium Recruiting

Study contacts

• Principal investigator: Rik Schrijvers, MD, PhD — UZ Leuven
• Study coordinator: Rik Schrijvers, MD, PhD
• Email: rik.schrijvers@uzleuven.be
• Phone: +3216342985

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Evaluating the safety of a 20% Immune Globulin solution in patients with primary immunodeficiency

  This study is trying to see if a 20% immune globulin solution is safe for people with primary immunodeficiency diseases by looking at past patient data from Japan.

  Recruiting Takeda · Tokyo, Tokyo · NCT06565078

• Data collection for patients with non-malignant disorders undergoing stem cell transplantation

  This study is collecting information from patients with non-cancerous disorders who are getting stem cell transplants to see if a new treatment plan helps them recover better and avoid complications.

  Recruiting University of Pittsburgh · Pittsburgh, Pennsylvania · NCT04528355

• Reduced intensity conditioning for patients with non-malignant disorders undergoing stem cell transplants

  This study is testing a gentler approach to stem cell transplants for people with non-cancerous disorders to see if it helps them recover better with fewer risks.

  Recruiting Phase 2 University of Pittsburgh · Pittsburgh, Pennsylvania · NCT01962415

• T-cell therapy for chronic norovirus in immunocompromised patients

  This study is testing a new T-cell therapy for people with chronic norovirus infections who have weakened immune systems to see if it's safe for them.

  Recruiting at 3 sites Phase 1 Children's National Research Institute · Washington D.C., District of Columbia and 2 other locations · NCT04691622

• BP-SCIG 20% treatment for primary immunodeficiency

  This will test BP-SCIG 20%, a 20% subcutaneous immunoglobulin given weekly (with an optional loading dose), in children and adults with primary immunodeficiency who need IgG replacement.

  Recruiting at 5 sites Phase 3 Biopharma Plasma LLC · Cherkasy and 11 other locations · NCT07346859

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.