7 results
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
- to evaluate the FVIIa activity PK of 2 CSL689 dose levels in subjects with congenital FVII deficiency- to determine the PK characteristics of FVIIa activity of CSL689- to evaluate the safety and tolerability of intravenous administration of CSL689
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 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…
Primary Objective* To compare the effect of a 30-week course of mavacamten with placebo on clinical response comprising of exercise capacity and clinical symptoms in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM)…
primary objective:To assess the long-term safety and tolerability of mavacamten in participants with hypertrophic cardiomyopathy (HCM) previously enrolled in 1 of 2 placebo-controlled trials: MAVERICK-HCM (MYK-461-006) for non-obstructive HCM (nHCM…
Primary Objectives: • To assess the efficacy of a 48-week course of mavacamten compared to placebo on patient- reported health status (symptoms and physical limitations)• To assess the efficacy of a 48-week course of mavacamten compared to placebo…