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Pharmacokinetics, safety and efficacy of methylphenidate during pregnancy in women with Attention-Deficit (Hyperactivity) Disorder

Primary objective is assessment of the pharmacokinetic profile of MPD in pregnancy. Secondary objectives are the safety and efficacy of any use of MPD during pregnancy.

An Open-label, Multinational, Multicenter, Intravenous
Infusion Study of the Efficacy, Safety, Pharmacokinetics,
and Pharmacodynamics of avalglucosidase alfa in
Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)

This study has been transitioned to CTIS with ID 2024-513859-33-00 check the CTIS register for the current data. The overall objective is to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and…

An exploratory pharmacokinetics and pharmacodynamics study of beta-lactam antibiotics in pediatric intensive care patients: is there a need for more precision? (EXPAT-Kids)

The aim of this study is to describe the PK/PD characteristics of frequently used beta-lactam antibiotics in PICU patients. The main objective is to identify whether current antibiotic dosing regimens of the selected beta-lactams achieve defined…

Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease

This study has been transitioned to CTIS with ID 2024-510603-11-00 check the CTIS register for the current data. Primary Objective: To evaluate the efficacy of ION373 in improving or stabilizing gross motor function in patients with Alexander…

Pharmacokinetics of tacrolimus during pregnancy in kidney and liver transplant recipients.

The overall aim of the study is to gain more insight in as well as understand and map the pharmacokinetics of tacrolimus during pregnancy. Therefore, we will study tacrolimus concentrations in maternal whole blood (routine care) and via area under…

Markers of Oxidative Stress from Supplemental Oxygen in critically-ill children

This study has two parts. The main objective of part 1 (in vivo) is todetermine changes in markers of oxidative stress in exhaled breath as a result of different short term oxygenation strategies (liberal versus conservative) . The main objective of…