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The role of TSH in the human immune system, a randomized, controlled, trial

The main objective of this study is to determine the role of TSH in the generation of new T cells by thymopoiesis

Glivec (imatinib mesylate) in systemic sclerosis, a pilot study

To investigate the efficacy (and the toxicity) of Glivec in systemic sclerosis by examining clinical outcomes (clinical and laboratory findings).

Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD).

Primary: Dose finding for a twice daily regimen for PSZ as prophylactic treatment in children with CGD, based on the exposure to PSZ measured by PSZ trough levels.Secondary:To determine the tolerability of PSZ as prophylactic treatment in children…

Effects of nicotinamide riboside (vitamin B3) in patients with Ataxia Telangiectasia

The purpose of this research is to investigate whether treatment with nicotinamide riboside (vitamine B3) has a positive effect on the disease course of patients with ataxia telangiectasia.

PHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY

This study has been transitioned to CTIS with ID 2023-510204-50-00 check the CTIS register for the current data. Primary: The main objectives of the study are to demonstrate feasibility and safety of gene corrected autologous CD34+-selected…

An open-label, 8-week, proof of concept trial on thymosin-α1 (thymalfasin) in the treatment of primary antibody deficiency (PAD) associated mood disorders (TIDAM).

Based on the effects of Tα1 on immune cell function, the known disturbances in T lymphocyte numbers and subsets in patients with CVID and the increased prevalence of mood disorders in these patients the following research questions have been…

An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (activated phosphoinositide 3-kinase delta syndrome/p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency)

This study is an open-label, non-randomized extension to study CCDZ173X2201. It aims to provide treatment with CDZ173 to patients withAPDS/PASLI who participated in study CCDZ173X2201 or who were treated previously with PI3Kδ inhibitors other than…

Prospective Analysis of an individualized dosing Regimen of ATG (Thymoglobulin) in Children Undergoing HCT: redUcing Toxicity and improving Efficacy * a single arm phase II study

To investigate whether an individualized dosing regimen for Thymoglobulin leads to a better immune reconstitution after HCT (definition as in primary endpoint), as compared to historically non-individualized treated patients receiving Thymoglobulin…