2 results
Approved WMOCompleted
• To evaluate the safety and efficacy of multiple doses of XPF-001 (400 mg bid) for relief of pain in patients with IEM.• To evaluate the efficacy of multiple doses of XPF-001 (400 mg bid) for relief of vasomotor signs in patient with IEM.• To…
Approved WMOCompleted
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…