7 results
The objectives of this open-label study are to evaluate the efficacy (GL-3 clearance), pharmacokinetics (PK), and safety parameters (including immunogenicity) for 2 alternative dose regimens of Fabrazyme (0.5 mg/kg every 2 weeks [q2w] and 1.0 mg/kg…
The primary objectives of this study are: * To characterize the effects of 150 mg and 450 mg of AT1001 administered 2 hours before administration of agalsidase on the safety and plasma pharmacokinetics of agalsidase in subjects with Fabry Disease*…
In this study we want to investigate whether the submission of zoledronic acid to neoadjuvant chemotherapy benefits the pathological complete response, and thus favors a better clinical outcome in patients with large ressectable or locally advanced…
To evaluate the safety and efficacy of PRX-102 compared to agalsidase beta in Fabry disease patients with impaired renal function.
One of the primary goals is to establish whether students in the first grade of secondary vocational education receiving the multimodal school-based stepped approach towards preventing suicidal behaviours and depression show a clinically significant…
The purpose of the overall project is to adapt an existing self management tool for adults to the needs of young people and how to implement the adapted app within the youth mental health. The overall project consists of three different phases with…
This study will evaluate the safety, tolerability, and pharmacokinetics of idasanutlin as a single agent and the safety, tolerability, pharmacokinetics, and preliminary efficacy of idasanutlin in combination with either chemotherapy or venetoclax in…