4 results
Primary ObjectiveTo generate evidence demonstrating the domain specification (via modern psychometric methods), reliability, validity, and responsiveness (within-subject meaningful change) of the patient-reported outcome (PRO) endpoints Secondary…
Primary Objective* To compare the effect of a 30-week course of mavacamten with placebo on clinical response comprising of exercise capacity and clinical symptoms in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM)…
Primary Objectives: • To assess the efficacy of a 48-week course of mavacamten compared to placebo on patient- reported health status (symptoms and physical limitations)• To assess the efficacy of a 48-week course of mavacamten compared to placebo…
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…