4 results
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
The purpose of the study is to compare the hemodynamic effects (study of blood flow or circulation, such as blood pressure, heart rate, cardiac output (the volume of blood being pumped by the heart) and glomerular filtration rate (flow rate of…
Primary objectives of the study are:• Arm 1 and Arm 3 (PTPs): to characterize the long-term safety profile of rVIII SingleChain with respect to inhibitor development in PTPs.• Arm 2 (PUPs): • To characterize the safety with respect to inhibitor…
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…