2 results
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…
Approved WMOPending
Primary objectives: • Determine the safety and tolerability • Define the recommended phase 2 dose regimen (RP2D-R) and/or the maximum tolerated dose-regimen (MTD-R) (phase 1 only) • Evaluate the preliminary anti-leukemic activity (primary for phase…