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National observational study to monitor the new guideline concerning treatment of atypical hemolytic uremic syndrome

Monitoring and evaluation of the Dutch guideline for treatment of aHUS in children and adults during two years.

Correcting mutations in vitro using CRISPR-Cas9; towards autologous stem cell transplantation in sickle cell disease and X-linked severe combined immunodeficiency

Preparation for in vivo correction of SCD and X-SCID causing mutations by CRISPR-Cas9 by in vitro studies in cell lines

B7 Coreceptor Molecules as Clinically-Relevant Surrogate Biomarkers in the Hyper IgD Syndrome (HIDS) form of Mevalonate Kinase Deficiency (MKD)

The objective of this study is to assess potentially new and unique biomarkers that will be specific to patients with HIDS as surrogate outcomes for eventual larger, cohort-controlled clinical studies. Our longitudinal design in a small pilot group…

Pharmacokinetics and safety of the intravenous human immunoglobulin product Nanogam 100 mg/ml

The primary objective is to examine the pharmacokinetics of Nanogam 100 mg/ml and compare these with Nanogam 50 mg/ml. The secondary objective is safety and tolerability of Nanogam 100 mg/ml. Aim is to show bioequivalency between Nanogam 50 mg/ml…

Pedigree research in families with Common Variable Immunodeficiency Disease.

Using pedigree research in two CVID-families to find clues for genetic testing.

CFH polymorphism and laser coagulation of drusen

Assess correlation between CFH polymorphism and the effect of laser treatment of drusen.

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…

Study to estimate the minimum clinically different difference in visual analogue scale scores for skin swelling, skin pain and abdominal pain in subjects with hereditary angio-edema

The objective of the study is to estimate the utility and the minimally clinically significant symptom difference in VAS and verbal descriptor scores as the basis for the onset of relief for skin swelling, skin pain or abdominal pain in patients…

An open-label study to characterize the absorption, distribution, metabolism and elimination of a single oral dose of 14C-leniolisib in healthy male subjects

In this study we will investigate how safe the new compound leniolisib is and how well it is tolerated by healthy male participants.We also investigate how quickly and to what extent leniolisib is absorbed, transported, and eliminated from the body…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in patients with WHIM Syndrome with Open-Label Extension

This study has been transitioned to CTIS with ID 2024-518461-10-00 check the CTIS register for the current data. • Randomized Placebo-Controlled Period:Primary:- to demonstrate the efficacy of mavorixafor in patients with Warts,…

C1-inhibitor improves efficacy of red blood cell transfusion in patients suffering from autoimmune hemolytic anemia * an open-labeled pilot trial

The aim of the present *proof-of-principle* study is to test in patients suffering from AIHA whether co-administration of C1-inh improves the recovery of RBC transfusion by the inhibition of the activation of the classical pathway of complement. In…

A multicenter, Phase 2a, open-label, non-randomized study evaluating the efficacy, safety, and tolerability of BIVV020 in adults with persistent/chronic immune thrombocytopenia (ITP)

To evaluate the effect of BIVV020 on the durabilityof platelet response in participants withpersistent/chronic immune thrombocytopenia (ITP)Secondary• To assess the safety and tolerability of BIVV020• To assess the pharmacokinetics (PK) of BIVV020•…

An Open-Label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 4 to 11 Years) With APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed By an Open-Label Long-Term Extension

Objective Endpoint• To assess the clinical safety and tolerability of leniolisib in pediatric patients (aged 4 to 11 years) with APDS• Incidence of treatment-emergent AEs (TEAEs), SAEs, and AEs leading to discontinuation of study drug• Change from…

Diagnostic value of array-based genotyping in autoinflammatory syndromes

Objective: The primary objective of this study is to investigate the potential of the custom global screening array (GSA) in patients with autoinflammatory disorders. We aim to investigate whether the GSA can pick up genetic variants in genes known…

A bioequivalence study comparing 70 mg leniolisib capsules with leniolisib tablets in healthy volunteers

In this study we will compare leniolisib as capsules with leniolisib as tablets. We compare this in terms of how quickly and to what extent each form of leniolisib is absorbed, transported, and eliminated from the body (this is called…

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…

An Open-label, Sequential, Ascending, Repeated Dose-finding Study of Sarilumab, Administered with Subcutaneous (SC) Injection, in Children and Adolescents, Aged 1 to 17 Years, with Systemic Juvenile Idiopathic Arthritis (sJIA), Followed by an Extension Phase

To describe the pharmacokinetic (PK) profile and effectiveness of sarilumab in patients with sJIA in order to identifythe dose and regimen for continued development in this population.

An Open-label, Sequential, Ascending, Repeated Dose-finding Study of Sarilumab, Administered with Subcutaneous (SC) Injection, in Children and Adolescents, Aged 2 to 17 Years, with Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA) Followed by an Extension Phase

To describe the pharmacokinetic (PK) profile and effectiveness of sarilumab in patients with pcJIA in order to identifythe dose and regimen for continued development in this population.