4 results
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
Primary objective:Determine the incidence rate of inhibitor formation in PUPs with severe and moderately severe hemophilia A during the first 50 exposure days (EDs) to starting with a once weekly prophylactic regimen together with the minimization…
Primary objective: - To study the pharmacokinetics of acetaminophen and metabolites in morbidly obese patients and compare with normal weight patients. Secondary objectives: - To compare the pharmacokinetics of acetaminophen and metabolites in…
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…