3 results
The primary objectives are the following:- To assess the safety profile and determine the Optimal Biological Dose (OBD) or Maximum Tolerated Dose (MTD), whichever occurs first, of intravesically administered TMX-101. The secondary objectives are the…
Primary objective: 1. to establish the percentage of children with functional abdominal pain becoming pain free with laxative therapy, in comparison with placebo therapy.Secondary objective: 2. to establish the decrease of the pain intensity in…
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…