5 results
Objectives: The following trial objectives for adult patients with plasma therapy-resistantAtypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:• Assess the effect of eculizumab to reduce thrombotic microangiopathy (TMA) asindicated by…
Objectives: The following trial objectives for adolescent patients (from 12 and up to 18 years of age) with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:* Assess the effect of eculizumab on TMA-Event Free status…
Objectives: The following trial objectives for adult patients with plasma therapy-resistantAtypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:• Assess the effect of eculizumab to reduce thrombotic microangiopathy (TMA) asindicated by…
To evaluate the effect of treatment with BoNT on psychogenic jerks.
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…