7 results
The primary objective of Part 1 is the primary objective of the entire study.Part 1The primary objective of Part 1 is:* To evaluate the effect of ZX008 0.8 mg/kg/day versus placebo as adjunctive therapy for the treatment of uncontrolled seizures in…
* To assess the long-term safety and tolerability of ZX008.
The primary objective is:* To evaluate RBC transfusion independence (RBC-TI) of luspatercept compared with placebo for the treatment of anemia due to IPSS-R very low, low, or intermediate risk MDS in subjects with ring sideroblasts (* 15%) who…
The primary efficacy objectives of this two-cohort study are:o To demonstrate that ZX008 is superior to placebo as adjunctive therapy in the treatment of symptoms of Dravet syndrome in children and young adults based on change in the frequency of…
Primary objective:• To evaluate the efficacy of luspatercept on RBC transfusion independence (RBC-TI for 12 weeks [84 days] with an associated concurrent mean hemoglobin increase >= 1.5 g/dL) compared with epoetin alfa for the treatment of…
PART A and PART DTo assess the safety and tolerability of TAK-994 following multiple oral doses in subjects with narcolepsy with or without cataplexy (NT1 or NT2).PART B and PART C To assess the efficacy of TAK-994 on reducing excessive daytime…
Objectives for Part 1: Primary (Efficacy): To demonstrate that the efficacy of fenfluramine (ZX008) 0.8 mg/kg/day is superior to placebo as an adjunctive therapy for pediatric and adult subjects with CDD Secondary (Safety): To characterize the…