3 results
To evaluate the inter- and intra-subject variability in pharmacokinetics (PK) and pharmacodynamics (PD) of study drug following a two-period repeated single dose of 2 mg delivered by subcutaneous (SC) injection in healthy volunteers.
Primary objectives: 1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and2. To evaluate the safety and…
The purpose of this study is to compare event-free survival (EFS) in participants with Bacillus Calmette-Guerin (BCG)-naive high-risk non-muscle invasive bladder cancer (HR-NMIBC), including high-grade papillary Ta, any T1, or carcinoma in situ (CIS…