4 results
The objective of this pivotal study is to assess the PK and PD as well as safety and tolerability of RP103 compared to Cystagon® in patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support the registration…
The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…
The purpose of this first-in-human (FIH) study is to assess the safety, tolerability, pharmacodynamics (PD), and pharmacokinetics (PK) of subcutaneous (SC) VIS171 in healthy participants (single ascending dose [SAD] - Part A) as well as in…
The objection of the study is to assess the safety, tolerability, and effect on microvascular obstruction of study drug in subjects undergoing Percutaneous Coronary Intervention.NL specific sub-study:The aim of this substudy is to investigate…