2 results
Approved WMOWill not start
Primary objective: To determine the effect of hyperbilirubinemia on systemic activation of the innate immune response induced by a lipopolysaccharide (LPS) challenge. Secondary Objective(s): - To determine if hyperbilirubinemia shifts the pro-anti…
Not approvedWill not start
Primary:The purpose of this study is to evaluate the safety, tolerability, orally administered LMI070 in patients with Type 1 SMA.pharmacokinetics (PK), pharmacodynamics (PD) and efficacy; and to estimate the Maximum Tolerated Dose (MTD) and optimal…