3 results
Approved WMOWill not start
Primary Objective:* confirm the dose of nomacopan and sample size for Part B and evaluate the rank order of secondary endpointsOther Objectives:* compare nomacopan with adjunct OCS to placebo with adjunct OCS in achievement of Complete Remission of…
Approved WMOPending
Assess the neurological efficacy of ambroxol in adults and children with GD3.
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…