Ex Vivo Evaluation of JAK-inhibitor and Gene Therapeutical Approach in JAK-STAT Related Disorders

Plain-language summary

This study evaluates, in the laboratory (ex vivo) using blood samples, a JAK inhibitor and a gene therapy approach in disorders that lead to an overactive JAK-STAT pathway. It enrolls adult patients with a genetically confirmed or strongly suspected disorder causing excessive JAK-STAT activation, along with healthy controls. It is a study with no assigned phase and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Not Applicable

Status

Recruiting

Sponsor

prof. dr. Rik Schrijvers

Intervention

blood sampling

Start date

11/21/2024

Estimated completion

12/1/2030

Countries

Belgium

Related genes

Locations

• Leuven, Belgium

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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