6 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…
Primary: To determine the safety and tolerability of CK3773274 in patients with symptomatic HCMSecondary: Assess long-term effects of CK3773274 on left ventricular outflow tract gradient (LVOT-G) in patients with oHCM
The primary objective of this study is to examine the effectiveness of a mobile application, *The SUPPORT Coach*, in reducing traumatic stress symptoms among traumatized adults with traumatic stress. The secondary objective is to investigate the…
The aim of this study is to assess the validity and reliability of an FD-specific electronic patient-reported outcome measure (ePRO), based on the Experience Sampling Method-principle, for symptom assessment and identification of symptom triggers in…
Primary• To evaluate the effect of CK-3773274 on exercise capacity (VO2) in patients with symptomatic oHCMSecondary• To evaluate the effect of CK-3773274 on patient health status• To evaluate the effect of CK-3773274 on New York Heart Association (…