2 results
Approved WMOCompleted
Primary objectives:To assess the bioequivalence of a single-dose TRI as a whole tablet (reference) compared to a crushed and suspended tablet (intervention I).To assess the bioequivalence of a single-dose TRI as a whole tablet (reference) compared…
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…